diff --git a/src/components/Calendar/cal-content.tsx b/src/components/Calendar/cal-content.tsx
index cdac87c5ed8b18c164b36a081068a87e9be21cf5..d03b5d2a6caa3ba18d1e6d93061ce9488be4d40b 100644
--- a/src/components/Calendar/cal-content.tsx
+++ b/src/components/Calendar/cal-content.tsx
@@ -171,7 +171,7 @@ let tabs = [
<p>Cyanobacteria blooms pose significant ecological threats, and current control methods are inadequate. The proposed solution involves using peptides from algae to prevent blooms while preserving ecosystems. Key pathways and targets were identified, emphasizing the need for conservation and biodiversity.</p>
<h3>Fourth Presentation: Cystic Fibrosis</h3>
- <p>Cystic fibrosis affects a significant number of individuals and is diagnosed through sweat tests and genetic screening. Current treatments focus on managing symptoms, but the goal is to develop long-term cures by targeting the genetic basis of the disorder. The team is collaborating with various specialists and organizations to develop educational materials and optimize treatment strategies.</p>
+ <p>Cystic Cystic Fibrosis affects a significant number of individuals and is diagnosed through sweat tests and genetic screening. Current treatments focus on managing symptoms, but the goal is to develop long-term cures by targeting the genetic basis of the disorder. The team is collaborating with various specialists and organizations to develop educational materials and optimize treatment strategies.</p>
<h3>Further Presentations</h3>
<p>Discussions included the potential of phages as an alternative to antibiotics and future presentations on related topics.</p>
diff --git a/src/contents/Contribution/safety-contribution.tsx b/src/contents/Contribution/safety-contribution.tsx
index 0128f6ae829cbd485c558f3ef8c828d835895634..4d2a843e0afdab05e699c519fff68870c045aaa8 100644
--- a/src/contents/Contribution/safety-contribution.tsx
+++ b/src/contents/Contribution/safety-contribution.tsx
@@ -10,7 +10,7 @@ export function SafetyContribution(){
<>
<Subesction title="Biosafety & Security" id="Our Contributions1" >
<H4 text="Primary Culture Safety Guideline"/>
- <p>In the early phases of our project, we encountered several challenges while working to bring it together. We quickly decided to focus on human biomaterials, specifically cultivating primary human nasal epithelial cells from both fibrosis (CF) patients and wild-type controls. To ensure compliance, we carefully evaluated the safety regulations of our institution and government. Our contribution includes three key elements: </p>
+ <p>In the early phases of our project, we encountered several challenges while working to bring it together. We quickly decided to focus on human biomaterials, specifically cultivating primary human nasal epithelial cells from both Cystic Fibrosis (CF) patients and wild-type controls. To ensure compliance, we carefully evaluated the safety regulations of our institution and government. Our contribution includes three key elements: </p>
<ol>
<li> A guideline outlining the proper handling of biomaterials according to BSL2 standards, along with additional safety measures to ensure secure experimental design. </li>
<li> A clinical trial-style questionnaire designed to assess the medical history of participants donating nasal epithelial cells. </li>
diff --git a/src/contents/Human Practices/Conclisuin.tsx b/src/contents/Human Practices/Conclisuin.tsx
index be9c38d4fc014263c0610a71a2e63a2788b6f1d6..dc2a2c6e9c1a2b1bd8a89eaae4017fe6d4d09eb1 100644
--- a/src/contents/Human Practices/Conclisuin.tsx
+++ b/src/contents/Human Practices/Conclisuin.tsx
@@ -10,7 +10,7 @@ export function HPconclusion(){
useTabNavigation();
return(
<>
- <p>Our project has evolved through a deeply collaborative and human-centered approach, integrating diverse feedback from patients, clinicians, researchers, and industry experts. These insights shaped not only the technical aspects of our gene therapy for fibrosis (CF) but also our commitment to addressing real-world patient needs, ethical considerations, and the disparities in CF treatment worldwide. From <a onClick={() => goToPagesAndOpenTab('maxfirst', '')}>Max Beckmann’s</a> patient perspective to expert guidance on technical and ethical issues, each stakeholder contributed to refining our solution, ensuring it is both innovative and empathetic. Our focus on gene therapy targeting CF’s complex mutations, integrating physiotherapy, and ensuring global accessibility demonstrates our holistic and inclusive vision for this project. Importantly, the collaboration with researchers in nanoparticle stability and gene therapy, along with the development of bilingual surveys and outreach materials, highlights our efforts to make science more accessible and transparent, bridging gaps in knowledge and care. </p>
+ <p>Our project has evolved through a deeply collaborative and human-centered approach, integrating diverse feedback from patients, clinicians, researchers, and industry experts. These insights shaped not only the technical aspects of our gene therapy for Cystic Fibrosis (CF) but also our commitment to addressing real-world patient needs, ethical considerations, and the disparities in CF treatment worldwide. From <a onClick={() => goToPagesAndOpenTab('maxfirst', '')}>Max Beckmann’s</a> patient perspective to expert guidance on technical and ethical issues, each stakeholder contributed to refining our solution, ensuring it is both innovative and empathetic. Our focus on gene therapy targeting CF’s complex mutations, integrating physiotherapy, and ensuring global accessibility demonstrates our holistic and inclusive vision for this project. Importantly, the collaboration with researchers in nanoparticle stability and gene therapy, along with the development of bilingual surveys and outreach materials, highlights our efforts to make science more accessible and transparent, bridging gaps in knowledge and care. </p>
<H5 text="Human Practices Integration "/>
<p>From the start, we prioritized engaging with CF patients, making sure that our project aligned with both their needs and scientific expectations. Early input from Max Beckmann, a CF patient and friend of our team, guided key design decisions, such as our focus on lung-targeted gene therapy. His insights also shaped aspects like hygiene protocols for immunocompromised patients and the portrayal of CF in our outreach materials. Max’s ongoing feedback provided invaluable emotional insight, helping us ground the project in the real-world experiences of CF patients.</p>
<H5 text="Stakeholder Engagement "/>
@@ -34,7 +34,7 @@ export function HPconclusion(){
<li><p><b>Expanded Global Outreach:</b></p> <p>Increase awareness and education on CF and gene therapy through multilingual platforms and collaboration with international CF communities, particularly in underrepresented regions.</p></li>
<li><p><b>Ethical and Legal Considerations:</b></p> <p>Finalize all bioethical protocols for patient sampling and data management to pave the way for safe, compliant future research, including the development of guidelines for future teams.</p></li>
</ol>
- <p>By continuing this patient-driven, scientifically rigorous, and globally conscious trajectory, we aim to advance fibrosis care and make a lasting impact on patient lives, ensuring that our project remains poised for clinical success.</p>
+ <p>By continuing this patient-driven, scientifically rigorous, and globally conscious trajectory, we aim to advance Cystic Fibrosis care and make a lasting impact on patient lives, ensuring that our project remains poised for clinical success.</p>
</>
@@ -270,7 +270,7 @@ function AnalyseBharti(){
function AnalyseIgnatova(){
return(
<Collapsible title="Prof. Dr. Ignatova – CF Expert & Researcher" id="ignatovaanalyseC">
- <p>Through our collaboration with <a href="https://2024.igem.wiki/hamburg/" title="iGEM Hamburg" > iGEM Hamburg</a>, we were introduced to Prof. Ignatova, a leading expert in fibrosis (CF) research. Initially, we consulted her to gain a deeper understanding of CF. Later, when the HEK cells from Leuven proved unsuitable for our tests, we reached out again to explore alternative cell models. </p>
+ <p>Through our collaboration with <a href="https://2024.igem.wiki/hamburg/" title="iGEM Hamburg" > iGEM Hamburg</a>, we were introduced to Prof. Ignatova, a leading expert in Cystic Fibrosis (CF) research. Initially, we consulted her to gain a deeper understanding of CF. Later, when the HEK cells from Leuven proved unsuitable for our tests, we reached out again to explore alternative cell models. </p>
<p>Prof. Ignatova provided access to the CFBE41o- cell line, immortalized CF cells derived from a CF patient, which we obtained with permission from Prof. Karl Kunzelmann at the University of Regensburg. This cell line offered us a new, reliable testing system, and we successfully cultivated the cells in our lab, although they required significant time to acclimate and grow. </p>
<div className="row align-items-center">
<div className="col">
@@ -569,7 +569,7 @@ function AnalyseJoshua(){
return(
<Collapsible title="Joshua – Vice president of CF Vest international, Father of a CF child" id="joshuaanalyseC">
<p>Through discussions with <a onClick={() => goToPagesAndOpenTab ('joshua', '/human-practices')}> Joshua </a>, we learned that CF statistics are inadequate, primarily representing the white population, which skews understanding of the disease's prevalence. We discovered that in Asian countries like Thailand, CF is underrepresented, leading to insufficient access to therapies and medications. </p>
- <p>This awareness sharpened our focus on the need for improved science communication and highlighted the ongoing issues of racism and discrimination within scientific research. In response, we are committed to enhancing the data landscape in Germany by creating <a onClick={() => goToPageAndScroll ('our-surveys-on--fibrosis-and-gene-therapy', '/human-practices')}> surveys </a> in both German and English to gather broader insights and increase outreach. </p>
+ <p>This awareness sharpened our focus on the need for improved science communication and highlighted the ongoing issues of racism and discrimination within scientific research. In response, we are committed to enhancing the data landscape in Germany by creating <a onClick={() => goToPageAndScroll ('our-surveys-on--Cystic Fibrosis-and-gene-therapy', '/human-practices')}> surveys </a> in both German and English to gather broader insights and increase outreach. </p>
<figure>
<img src="https://static.igem.wiki/teams/5247/photos/hp/joshua-zoom.webp" alt=""/>
@@ -647,7 +647,7 @@ function AnalyseMax(){
const {goToPagesAndOpenTab} = useNavigation();
return(
<Collapsible title="Max Beckmann – CF Patient and fellow student" id="maxanalyseC">
- <p>Max's input as a fibrosis (CF) patient directly influenced several key aspects of our project. After learning about the daily challenges of living with CF, we adapted our gene therapy approach to target the lungs, aligning our treatment with patient needs. His insights on the shortcomings of existing therapies strengthened our focus on developing a more effective solution.</p>
+ <p>Max's input as a Cystic Fibrosis (CF) patient directly influenced several key aspects of our project. After learning about the daily challenges of living with CF, we adapted our gene therapy approach to target the lungs, aligning our treatment with patient needs. His insights on the shortcomings of existing therapies strengthened our focus on developing a more effective solution.</p>
<p>Following Max's feedback, we implemented changes to our <a onClick={() => goToPagesAndOpenTab ('Patient MattersH', '/contribution')}> hygiene plan</a>, ensuring it meets the needs of immunocompromised individuals which we later presented to <a onClick={() => goToPagesAndOpenTab ('johannfunke', '/human-practices')}> Mr. Johannfunke</a>, contact person for students with disabilities and impairments at the university of bielefeld, and checked its feasibility. Max’s perspective also shaped the content of our outreach materials, helping us portray CF in a more realistic and respectful way during the whole project. </p>
<p>Additionally, Max contributed to the project by donating cells for our experiments, which allowed us to test our model systems effectively. Our ongoing communication with him has ensured that we stay patient-focused throughout, continually refining our approach based on his experiences. </p>
<div className="row">
diff --git a/src/contents/Human Practices/Feedback.tsx b/src/contents/Human Practices/Feedback.tsx
index 18e4ff1bbdd22e8e60dad5663933e3786c64139a..4ca88cb6aaeab8a084fe5247af9df9a70cec4108 100644
--- a/src/contents/Human Practices/Feedback.tsx
+++ b/src/contents/Human Practices/Feedback.tsx
@@ -14,11 +14,11 @@ export function HPFeedback(){
<div>
<p>Through our project, the insights and feedback from various stakeholders and experts played a crucial role in shaping and refining our approach. We actively integrated their input into the design, execution, and public engagement aspects of our work, ensuring a human-centered, scientifically sound solution. Below, we highlight key contributors and how their feedback impacted the project's development across multiple phases. </p>
- <H4 text="Our surveys on fibrosis and gene therapy"></H4>
- <p>From our outreach efforts, we learned that many people lack knowledge about fibrosis and desire more education on the subject. The same applies to gene therapy, with most individuals expressing openness to treatment options, which reinforces our commitment to pursuing this approach. </p>
+ <H4 text="Our surveys on Cystic Fibrosis and gene therapy"></H4>
+ <p>From our outreach efforts, we learned that many people lack knowledge about Cystic Fibrosis and desire more education on the subject. The same applies to gene therapy, with most individuals expressing openness to treatment options, which reinforces our commitment to pursuing this approach. </p>
<p>However, we recognize the importance of handling the public's trust and lack of knowledge responsibly. We aim to educate the community about safety and ethical considerations surrounding gene therapy. </p>
<p>In response, we have decided to implement feedback by creating informative materials such as flyers and utilizing platforms like <a onClick={() => goToPageWithTabAndScroll({tabId: 'mukomove', scrollToId: "cf-month" , path: '/human-practices'})}>mukoMOVE</a>, <a onClick={() => goToPageWithTabAndScroll({tabId: 'teutoruft', scrollToId: "teuroruft-heading" , path: '/human-practices'})}>â€Teuto ruft!â€</a>, <a onClick={() => goToPageWithTabAndScroll({tabId: 'akademie', scrollToId: "student-academy-heading" , path: '/human-practices'})}>SchülerInnenakademie</a>, and <a href='https://www.instagram.com/igem.bielefeld/?hl=de'>social media</a> to raise awareness and provide education. </p>
- <p><strong>Lack of knowledge about CF:</strong> Many people are unfamiliar with fibrosis and expressed a need for more education on the subject.</p>
+ <p><strong>Lack of knowledge about CF:</strong> Many people are unfamiliar with Cystic Fibrosis and expressed a need for more education on the subject.</p>
<p><strong>Gene therapy openness:</strong> Most individuals showed openness to gene therapy treatments, reinforcing our commitment to pursuing this therapeutic approach.</p>
@@ -30,7 +30,7 @@ export function HPFeedback(){
<div>
<Collapsible id="collapsible1" open={false} title="Full results of our surveys">
- <p> We are proud of our surveys on gene therapy and fibrosis (CF), which explore knowledge about the disease and willingness to embrace gene
+ <p> We are proud of our surveys on gene therapy and Cystic Fibrosis (CF), which explore knowledge about the disease and willingness to embrace gene
therapy as a potential treatment. Since we wanted to differentiate between the general public and affected CF patients, we created two different
surveys. 187 people partipipated in the survey for the general public and 185 people participated in the survey for patients and next of kin.</p>
<div className="row align-items-center">
@@ -47,7 +47,7 @@ export function HPFeedback(){
</div>
<div className="row ">
<div className="col">
- <p>The majority of respondents (62.70%) indicated that they or their relative require medical treatment or therapy daily. Weekly treatment was necessary for 14.59%, while 9.73% needed therapy several times per week. Only 6.49% reported needing treatment either monthly or rarely. The high frequency of daily treatments highlights the heavy burden of managing fibrosis and reinforces the potential appeal of gene therapy, which could reduce the need for constant medical intervention. </p>
+ <p>The majority of respondents (62.70%) indicated that they or their relative require medical treatment or therapy daily. Weekly treatment was necessary for 14.59%, while 9.73% needed therapy several times per week. Only 6.49% reported needing treatment either monthly or rarely. The high frequency of daily treatments highlights the heavy burden of managing Cystic Fibrosis and reinforces the potential appeal of gene therapy, which could reduce the need for constant medical intervention. </p>
</div>
<div className="col">
<p>A significant majority, 78.72%, indicated that they would be open to gene therapy if it significantly improved symptoms, while only 1.42% said no. This overwhelming support aligns with the hope patients have for less invasive and more effective treatments. This also reflects the possibility of gene therapy becoming a central treatment method, especially given the heavy therapeutic load CF patients already carry.</p>
@@ -102,7 +102,7 @@ function DetailedAnalysis(){
<p>56.76% of respondents reported that they are related to someone with CF, while 43.24% stated they are affected by CF themselves. This likely reflects the fact that many parents completed the survey on behalf of their children, as CF is typically diagnosed at a young age. The high involvement of parents underscores how the disease impacts not just the patients themselves but also their families, who are deeply involved in the day-to-day management of CF. This highlights the importance of considering both the perspectives of young patients and their families when discussing gene therapy and CF treatments, as parents often play a critical role in decision-making regarding new treatment options.</p>
</div>
<div className="col">
- <p>82.89% of respondents have heard of fibrosis, while 17.11% had not. The high level of awareness about CF suggests that the general public is relatively informed about the condition, possibly due to the visibility of the disease through media, health campaigns, or personal connections to affected individuals. However, the 17% unfamiliar with CF indicates that further outreach is necessary, particularly focusing on this demographic to spread knowledge about the disease and potential treatments, including gene therapy. </p>
+ <p>82.89% of respondents have heard of Cystic Fibrosis, while 17.11% had not. The high level of awareness about CF suggests that the general public is relatively informed about the condition, possibly due to the visibility of the disease through media, health campaigns, or personal connections to affected individuals. However, the 17% unfamiliar with CF indicates that further outreach is necessary, particularly focusing on this demographic to spread knowledge about the disease and potential treatments, including gene therapy. </p>
</div>
<div className="col">
<p>The majority of respondents (44.17%) learned about CF through media channels, such as television, news, or the internet. Other significant sources of information include family and friends (25.15%), as well as educational institutions (20.86%). Interestingly, only 3.68% of respondents learned about CF from healthcare providers, suggesting that the disease is more commonly understood through external sources rather than direct medical education. This reliance on media and personal connections highlights the importance of accurate and accessible information in the public domain, especially when considering the introduction of gene therapy as a treatment option. </p>
@@ -129,7 +129,7 @@ function DetailedAnalysis(){
</div>
<div className="col">
- <p>42.16% of respondents rated the impact of fibrosis on daily life as a 3 out of 5, indicating a moderate effect. Additionally, 32.97% rated the impact as a 2, while 12.43% rated it as a 4. Only 4.32% of respondents felt that CF had a very strong impact (rating it a 5), and 8.11% rated it a 1, suggesting little to no daily effect. These results indicate that for many patients and families, CF has a notable but varied impact on daily life, reinforcing the importance of treatments like gene therapy that could alleviate the burden. </p>
+ <p>42.16% of respondents rated the impact of Cystic Fibrosis on daily life as a 3 out of 5, indicating a moderate effect. Additionally, 32.97% rated the impact as a 2, while 12.43% rated it as a 4. Only 4.32% of respondents felt that CF had a very strong impact (rating it a 5), and 8.11% rated it a 1, suggesting little to no daily effect. These results indicate that for many patients and families, CF has a notable but varied impact on daily life, reinforcing the importance of treatments like gene therapy that could alleviate the burden. </p>
</div>
<div className="col">
@@ -155,7 +155,7 @@ function DetailedAnalysis(){
</div>
<div className="col">
- <p>The survey reveals that 36.79% of respondents identified emotional stress as the most stressful aspect of fibrosis, closely followed by physical symptoms at 32.78%. Social restrictions were noted by 17.73% of respondents, and financial burden was a concern for 11.37%. Only 1.34% cited other factors. These results show that emotional and physical challenges dominate the stressors for patients and families, highlighting the need for treatments like gene therapy that could reduce both the physical and emotional burdens of managing CF. </p>
+ <p>The survey reveals that 36.79% of respondents identified emotional stress as the most stressful aspect of Cystic Fibrosis, closely followed by physical symptoms at 32.78%. Social restrictions were noted by 17.73% of respondents, and financial burden was a concern for 11.37%. Only 1.34% cited other factors. These results show that emotional and physical challenges dominate the stressors for patients and families, highlighting the need for treatments like gene therapy that could reduce both the physical and emotional burdens of managing CF. </p>
</div>
</div>
</div>
diff --git a/src/contents/Human Practices/Further Engagement/Collaborations.tsx b/src/contents/Human Practices/Further Engagement/Collaborations.tsx
index 2c0899b1b461544da350c59e6e6e89bed85bec7a..c953c6a4df47c9097abd3ae0e8838e1762acc6f5 100644
--- a/src/contents/Human Practices/Further Engagement/Collaborations.tsx
+++ b/src/contents/Human Practices/Further Engagement/Collaborations.tsx
@@ -36,7 +36,7 @@ export function HPCollabs(){
<div id="coll-overview" className="ent-interview" style={{display: "block"}}>
<H4 id="ent-heading" text="Collaborations as part of a integrated human practice - but why?"/>
<p>Entrepreneurship is not only an interesting possibility but necessary to turn our ideas and results into a real product that can help people. </p>
- <p>That is why in this section we focus on the aspects of entrepreneurship that are crucial for the potential successful realisation of our project to develop new therapies for fibrosis. A pivotal moment was our interview with Nicole Friedlein, which gave us valuable insights into the challenges and opportunities in the field of biomedical innovation. The discussions in the interview encouraged us to look more closely at the regulatory requirements, which is why one team member completed a GxP course and subsequently trained the team in this area. In addition, we conducted further interviews in the area of entrepreneurship to gain a better understanding of the practical aspects of business development. These experiences not only enriched the scientific depth of our project, but also sharpened our perspective on the practical implementation and market launch of new therapies.
+ <p>That is why in this section we focus on the aspects of entrepreneurship that are crucial for the potential successful realisation of our project to develop new therapies for Cystic Fibrosis. A pivotal moment was our interview with Nicole Friedlein, which gave us valuable insights into the challenges and opportunities in the field of biomedical innovation. The discussions in the interview encouraged us to look more closely at the regulatory requirements, which is why one team member completed a GxP course and subsequently trained the team in this area. In addition, we conducted further interviews in the area of entrepreneurship to gain a better understanding of the practical aspects of business development. These experiences not only enriched the scientific depth of our project, but also sharpened our perspective on the practical implementation and market launch of new therapies.
</p>
</div>
diff --git a/src/contents/Human Practices/Further Engagement/Education.tsx b/src/contents/Human Practices/Further Engagement/Education.tsx
index a59829b78ab981ed45dde6622036c5994703c1b5..786ea5851f158d2e7bd0a3a4dc84a2304a908f88 100644
--- a/src/contents/Human Practices/Further Engagement/Education.tsx
+++ b/src/contents/Human Practices/Further Engagement/Education.tsx
@@ -40,24 +40,24 @@ export function HPEducation(){
<ul>
<li>To help people make <b>informed choices</b> and encourage <b>emancipation through education</b>.</li>
<li>Only informed participants allow for <b>ethical engagement</b>.</li>
- <li>To ensure <b>continuous learning</b> in order to secure the future of synthetic biology and fibrosis research.</li>
+ <li>To ensure <b>continuous learning</b> in order to secure the future of synthetic biology and Cystic Fibrosis research.</li>
<li>Only awareness and knowledge can <b>prevent misuse and misinformation</b>.</li>
</ul>
- <p>This is applicable to both fibrosis and synthetic biology in general.</p>
+ <p>This is applicable to both Cystic Fibrosis and synthetic biology in general.</p>
<p>Many people gravitate towards fields they are interested in. Awareness, exploration, and receiving new knowledge are necessary to cultivate an authentic
interest, which, together with positive social interaction, forms a promising foundation for a lasting interest<TabScrollLink tab="edu-overview" num="1" scrollId="desc-edu"/>. As future researchers and part of a
competition aiming for continuous innovation, we feel education is an important ascpect that should not be shrugged off under the guise of focusing on Human Practices.</p>
<H4 id="edu-why-heading" text="Our educational activities"/>
<p>In both "Der Teuto ruft!" and the CeBiTec Student Academy, our team focused on education through personal contact not only as way to spread
- awareness about fibrosis, but to spread the love we have for what we do. </p>
+ awareness about Cystic Fibrosis, but to spread the love we have for what we do. </p>
<p>We are glad to have had the possibility to work with such different audiences. While "Der Teuto ruft!" had a focus on families and required a creative
approach, the "Schüler*innen Akademie" and "MINT Sommer" allowed us to interact with aspiring researchers who may very well be our future classmates at
Bielefeld University.
</p>
<p>However, we came to realize that "Der Teuto ruft!" may have been the more impactful event for our personal growth. It took us out of the familiar
"science bubble" and into a space where we could interact with the general public—people who don’t necessarily have a scientific background. This
- experience reminded us how non-scientists perceive complex topics like gene therapy and fibrosis. It also highlighted the importance of not only
+ experience reminded us how non-scientists perceive complex topics like gene therapy and Cystic Fibrosis. It also highlighted the importance of not only
ethical responsibility but also social responsibility in communicating science. We gained and regained insight into the concerns, misconceptions, and
hopes that the public has regarding synthetic biology, allowing us to better understand what is not only scientifically sound but also socially
acceptable. We are confident that participating in "Der Teuto ruft!" very positively influenced our approach to further communication. </p>
@@ -87,11 +87,11 @@ Due to our collaboration with the Student Academy, we conducted the nanopore seq
<div id="teutoruft" className="edu-cycletab" style={{display: "none"}}>
<H4 id="teuroruft-heading" text="Educational city tour for young and old"/>
<H5 id="Der Teuto ruft!" text=" What is “Der Teuto ruft!†and why we participate"/>
-<p>"Der Teuto ruft!" is an outreach event located all over the city of Bielefeld where various local companies and institutions engage with the public to inform them about their work. Since we wanted to raise awareness for fibrosis and present our approach to developing an optimized gene therapy to combat this disease, our participation in the "Der Teuto ruft!" event in Bielefeld was the perfect opportunity to do so.</p>
+<p>"Der Teuto ruft!" is an outreach event located all over the city of Bielefeld where various local companies and institutions engage with the public to inform them about their work. Since we wanted to raise awareness for Cystic Fibrosis and present our approach to developing an optimized gene therapy to combat this disease, our participation in the "Der Teuto ruft!" event in Bielefeld was the perfect opportunity to do so.</p>
<H5 id="What was our strategy?" text="What is our strategy?"/>
<p>Our goal was to educate children about the challenges faced by CF patients, especially the ones with lung problems. The knowledge which we gained at the <a onClick={() => goToPageAndScroll ('commworkshop', '/contribution')}> Science Communication Workshop </a> as part of the <a onClick={() => goToPageAndScroll ('bfh-european-meetup', '/contribution')}> BFH Meetup </a> was the optimal basis to plan our outreach to the public. We engaged the children with activities like coloring lung images and conducting experiments to experience and understand lung related symptoms.
One such experiment involved creating a lung model from balloons and straws, demonstrating the difficulty patients have in breathing by having the children blow into the straws. Additionally, we set up a tank with a mixture of starch and water to simulate mucus and placed a ball on top. The children tried to blow the ball across the surface, illustrating how hard it is for air to move through mucus compared to water, where the ball moved much more easily.
-The very little ones could paint coloring pages which we designed and printed for them. For the adults, we provided information about our project and discussed the implications and potential of gene therapy for fibrosis. These conversations as well as the results of our <a onClick={() => goToPageAndScroll ('our-surveys-on--fibrosis-and-gene-therapy', '/human-practices')}> survey on CF and gene therapy </a> which was conducted events like these made it abundantly clear that degrees of knowledge on this topic widely vary throughout the public and we were happy to fill in the existing gaps in people's knowledge and exchange points of view on gene therapy.
+The very little ones could paint coloring pages which we designed and printed for them. For the adults, we provided information about our project and discussed the implications and potential of gene therapy for Cystic Fibrosis. These conversations as well as the results of our <a onClick={() => goToPageAndScroll ('our-surveys-on--Cystic Fibrosis-and-gene-therapy', '/human-practices')}> survey on CF and gene therapy </a> which was conducted events like these made it abundantly clear that degrees of knowledge on this topic widely vary throughout the public and we were happy to fill in the existing gaps in people's knowledge and exchange points of view on gene therapy.
Moreover, we connected with other institutions and participants at the event. We shared our booth at Bielefeld’s “Skulpturenpark†on the outside with <a href="https://bts-ev.de/bielefeld/" title="btS" > btS </a>, the life science student initiative from Bielefeld University, with whose members we had stimulating discussions as well. We were more than delighted when the city of Bielefeld featured us on their Instagram, highlighting our presence during "Der Teuto ruft!". This collaboration helped us reach a wider audience and raise awareness about our research efforts.</p>
<br/>
{/* <a href="https://unibielefeldde.sharepoint.com/sites/iGEM2024teams/_layouts/15/stream.aspx?id=%2Fsites%2FiGEM2024teams%2FFreigegebene%20Dokumente%2FGeneral%2FFotos%2C%20Videos%20und%20Co%2FTeuto%20ruft%2FVideo%20Insta%20Teuto%20Ruft%2Emov&ga=1&referrer=StreamWebApp%2EWeb&referrerScenario=AddressBarCopied%2Eview%2Ee4a43a55%2Dfff3%2D4b44%2Db081%2Dad26306f93e0" title="video Teuto ruft" > watch me</a>
@@ -100,7 +100,7 @@ Moreover, we connected with other institutions and participants at the event. We
<div className="row align-items-center">
<div className="col">
<H5 id="conclusion" text="What is our conclusion"/>
- <p>Despite the changeable weather, we could educate many people of Bielefeld's community about fibrosis, our therapeutic approach and gene therapy in general and had the opportunity to improve our science communication for the future as well so it was a successful event! </p>
+ <p>Despite the changeable weather, we could educate many people of Bielefeld's community about Cystic Fibrosis, our therapeutic approach and gene therapy in general and had the opportunity to improve our science communication for the future as well so it was a successful event! </p>
</div>
<div className="col">
<figure>
@@ -131,13 +131,13 @@ Moreover, we connected with other institutions and participants at the event. We
<img src="https://static.igem.wiki/teams/5247/photos/hp/mintsommerlogo.png" style={{width:"30%", height:"20%"}}/>
<div className="col">
<p>“MINT Summer 2024†is a comprehensive program designed primarily for high school graduates of the class of 2024, who are considering pursuing studies in STEM fields (Science, Technology, Engineering, and Mathematics, including teaching degrees). The program is perfect for those who are still uncertain if they want to study in STEM or which specific discipline aligns best with their interests.</p>
- <p>Our participation in <a href="https://www.uni-bielefeld.de/studium/studieninteressierte/mint-sommer/" title="Mint Summer" >MINT Summer </a> offered us the chance to raise awareness of fibrosis and showcase our cutting-edge approach to develop an optimized gene therapy to combat this disease. Through the event we engaged with potential future scientists and researchers, informing them about our project, iGEM and the importance of scientific research in advancing medical treatments. This program not only allows us to share our mission but also to inspire the next generation of STEM students by highlighting the real-world impact of their studies. </p>
+ <p>Our participation in <a href="https://www.uni-bielefeld.de/studium/studieninteressierte/mint-sommer/" title="Mint Summer" >MINT Summer </a> offered us the chance to raise awareness of Cystic Fibrosis and showcase our cutting-edge approach to develop an optimized gene therapy to combat this disease. Through the event we engaged with potential future scientists and researchers, informing them about our project, iGEM and the importance of scientific research in advancing medical treatments. This program not only allows us to share our mission but also to inspire the next generation of STEM students by highlighting the real-world impact of their studies. </p>
</div>
</div>
<H5 id="strategy summer" text="What was our strategy?"/>
- <p>Our objective at MINT Summer was to inform attendees, especially aspiring STEM students, about the unique challenges faced by fibrosis (CF) patients, with a particular focus on lung-related complications. We drew heavily on the insights gained from the Science Communication Workshop at the BFH Meetup, which provided us with the perfect framework to meticulously plan our outreach for this event. This foundation allowed us to craft engaging and educational activities that effectively conveyed the complexities of CF to our audience, ensuring our message was both impactful and accessible. </p>
+ <p>Our objective at MINT Summer was to inform attendees, especially aspiring STEM students, about the unique challenges faced by Cystic Fibrosis (CF) patients, with a particular focus on lung-related complications. We drew heavily on the insights gained from the Science Communication Workshop at the BFH Meetup, which provided us with the perfect framework to meticulously plan our outreach for this event. This foundation allowed us to craft engaging and educational activities that effectively conveyed the complexities of CF to our audience, ensuring our message was both impactful and accessible. </p>
<p>We took the opportunity to explain the iGEM competition and our project to participants. We shared how iGEM is a global competition that brings together student teams to solve real-world problems using synthetic biology. We discussed how our approach aims to correct the genetic mutation responsible for CF, potentially offering a more effective treatment. By engaging with attendees, we were able to highlight the significance of our research and the impact it could have on improving the lives of those affected by this challenging condition. They got the opportunity to contribute to our project by participating in our survey. </p>
- <p>Over the time of two weeks, we established meaningful connections with professors, students, and participants across various STEM fields during the event, like the student initiative btS and the Campusbrauerei Bielefeld. Sharing our project with the life science students was helpful, motivating and opened the door to engaging discussions that enriched our perspective and fostered collaboration. These interactions allowed us to connect with experts and students from different disciplines, enhancing our understanding of how our gene therapy research for fibrosis fits within the broader scientific landscape.</p>
+ <p>Over the time of two weeks, we established meaningful connections with professors, students, and participants across various STEM fields during the event, like the student initiative btS and the Campusbrauerei Bielefeld. Sharing our project with the life science students was helpful, motivating and opened the door to engaging discussions that enriched our perspective and fostered collaboration. These interactions allowed us to connect with experts and students from different disciplines, enhancing our understanding of how our gene therapy research for Cystic Fibrosis fits within the broader scientific landscape.</p>
<H5 id="conclusion summer" text="What is our conclusion?"/>
<p>The experience allowed us to refine our science communication skills and connect with a broad range of STEM professionals and students. Overall, the event was a valuable opportunity for both education and professional growth, making it a rewarding and impactful experience for our team. </p>
</div>
diff --git a/src/contents/Human Practices/Further Engagement/Entrepreneurship.tsx b/src/contents/Human Practices/Further Engagement/Entrepreneurship.tsx
index f7aca6d64177df57ff97acbf9ed3463b6d61604d..748350a89c18c3dabe33d5730b93bae232801478 100644
--- a/src/contents/Human Practices/Further Engagement/Entrepreneurship.tsx
+++ b/src/contents/Human Practices/Further Engagement/Entrepreneurship.tsx
@@ -23,17 +23,17 @@ export function HPEntrepreneur(){
<div id="ent-overview" className="ent-interview" style={{display: "block"}}>
<H4 id="ent-heading" text="Entrepreneurship as part of a integrated human practice - but why?"/>
<p>Entrepreneurship is not only an interesting possibility but necessary to turn our ideas and results into a real product that can help people. </p>
- <p>That is why in this section we focus on the aspects of entrepreneurship that are crucial for the potential successful realisation of our project to develop new therapies for fibrosis. A pivotal moment was our interview with Nicole Friedlein, which gave us valuable insights into the challenges and opportunities in the field of biomedical innovation. The discussions in the interview encouraged us to look more closely at the regulatory requirements, which is why one team member completed a GxP course and subsequently trained the team in this area. In addition, we conducted further interviews in the area of entrepreneurship to gain a better understanding of the practical aspects of business development. These experiences not only enriched the scientific depth of our project, but also sharpened our perspective on the practical implementation and market launch of new therapies.
+ <p>That is why in this section we focus on the aspects of entrepreneurship that are crucial for the potential successful realisation of our project to develop new therapies for Cystic Fibrosis. A pivotal moment was our interview with Nicole Friedlein, which gave us valuable insights into the challenges and opportunities in the field of biomedical innovation. The discussions in the interview encouraged us to look more closely at the regulatory requirements, which is why one team member completed a GxP course and subsequently trained the team in this area. In addition, we conducted further interviews in the area of entrepreneurship to gain a better understanding of the practical aspects of business development. These experiences not only enriched the scientific depth of our project, but also sharpened our perspective on the practical implementation and market launch of new therapies.
</p>
<H4 id="ent-heading" text="Our Entrepreneurship"/>
- <p>In conclusion, the entrepreneurial journey of developing RNA-based gene therapy for fibrosis, as outlined in our experiences and interviews with industry founders, demonstrates that entrepreneurship is not only an interesting possibility but a necessary avenue to transform scientific innovation into real-world solutions. Our approach has been shaped by the challenges and opportunities in the biotech field, from understanding regulatory frameworks like GxP to navigating complex market dynamics and funding challenges. </p>
+ <p>In conclusion, the entrepreneurial journey of developing RNA-based gene therapy for Cystic Fibrosis, as outlined in our experiences and interviews with industry founders, demonstrates that entrepreneurship is not only an interesting possibility but a necessary avenue to transform scientific innovation into real-world solutions. Our approach has been shaped by the challenges and opportunities in the biotech field, from understanding regulatory frameworks like GxP to navigating complex market dynamics and funding challenges. </p>
<p>Through key interviews, such as the one with Nicole Friedlein, we have gained insights into the pivotal role of regulatory standards in scaling our project. The completion of GxP training by one team member reflects our commitment to ensuring compliance with Good Laboratory Practice (GLP) and Good Manufacturing Practice (GMP), both of which are essential for advancing from proof-of-concept to clinical trials. This foundation is crucial for building investor confidence and meeting regulatory requirements.</p>
- <p>Additionally, market evaluations reveal a significant opportunity for our therapy, particularly targeting the unmet needs of fibrosis patients who do not respond to current treatments like CFTR modulators. The growing gene therapy market presents a strong case for our innovation, although we are aware of the competitive landscape dominated by companies like Vertex Pharmaceuticals. Our unique value lies in providing a more permanent solution for patients not served by existing treatments. </p>
+ <p>Additionally, market evaluations reveal a significant opportunity for our therapy, particularly targeting the unmet needs of Cystic Fibrosis patients who do not respond to current treatments like CFTR modulators. The growing gene therapy market presents a strong case for our innovation, although we are aware of the competitive landscape dominated by companies like Vertex Pharmaceuticals. Our unique value lies in providing a more permanent solution for patients not served by existing treatments. </p>
<p>Interviews with founders from companies such as PlasmidFactory and RNhale have provided valuable lessons on transitioning from research to commercialization. The importance of building networks, securing diverse funding sources, and maintaining flexibility to adapt to market feedback are key takeaways that will guide our next steps. Establishing strategic partnerships and seeking early engagement with regulatory bodies will be essential as we prepare for clinical trials and eventual market entry.</p>
- <p>To align our long-term vision of revolutionizing fibrosis treatment with immediate milestones, we will continue optimizing our lipid nanoparticle delivery system, pursuing regulatory compliance, and engaging with the fibrosis community to refine our product. Our focus on both the scientific and business aspects ensures that we are building a strong foundation for success in bringing this innovative therapy to market, improving the lives of patients with fibrosis. </p>
+ <p>To align our long-term vision of revolutionizing Cystic Fibrosis treatment with immediate milestones, we will continue optimizing our lipid nanoparticle delivery system, pursuing regulatory compliance, and engaging with the Cystic Fibrosis community to refine our product. Our focus on both the scientific and business aspects ensures that we are building a strong foundation for success in bringing this innovative therapy to market, improving the lives of patients with Cystic Fibrosis. </p>
</div>
<div id="ent-interview" className="ent-interview" style={{display: "none"}}>
@@ -47,7 +47,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>RNhale validated their idea by seeking feedback from both the scientific community and industry professionals at conferences and networking events. They noticed growing interest in RNA therapeutics, particularly for lung delivery. The challenges surrounding delivery systems, especially highlighted during the COVID-19 pandemic, confirmed that there was a strong market demand for their technology, which motivated them to move forward with commercialization.</p>
<H5 text="Learnings and Implications for our project "/>
- <p>For our project, a concrete next step would be to actively seek feedback from fibrosis research communities and biotech conferences. We should continue to present our RNA-based gene therapy approach to experts in gene editing and delivery systems, specifically asking for input on our delivery mechanism using lipid nanoparticles (LNPs). This early engagement could help identify whether our approach addresses a real unmet need in fibrosis treatment and refine our product to better meet clinical and patient needs.
+ <p>For our project, a concrete next step would be to actively seek feedback from Cystic Fibrosis research communities and biotech conferences. We should continue to present our RNA-based gene therapy approach to experts in gene editing and delivery systems, specifically asking for input on our delivery mechanism using lipid nanoparticles (LNPs). This early engagement could help identify whether our approach addresses a real unmet need in Cystic Fibrosis treatment and refine our product to better meet clinical and patient needs.
</p>
<H4 id="ent-expert-heading" text="Question 2: Proof-of-Concept"/>
<H5 text="What we asked the Founders"/>
@@ -69,7 +69,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>For RNhale, the biggest challenge was securing sufficient funding, as transitioning from university-based research to the private sector requires a strategic approach to bridging this gap. They also mentioned that developing a clear business model earlier on could have sped up the process. Another challenge was forming partnerships with industry at an earlier stage, which might have eased both the funding process and commercialization efforts.</p>
<H5 text="Learnings and Implications for our project "/>
- <p>Both founders emphasized the challenge of securing funding and building a clear business model. At Bielefeld University, we should consider exploring partnerships with industry early, such as biotech firms or pharmaceutical companies. A concrete next step could be identifying relevant funding programs like EXIST or EU grants, which could help bridge the gap between our university research and commercialization. Developing a business model tailored to RNA-based therapeutics for fibrosis will also be critical to attract investors. </p>
+ <p>Both founders emphasized the challenge of securing funding and building a clear business model. At Bielefeld University, we should consider exploring partnerships with industry early, such as biotech firms or pharmaceutical companies. A concrete next step could be identifying relevant funding programs like EXIST or EU grants, which could help bridge the gap between our university research and commercialization. Developing a business model tailored to RNA-based therapeutics for Cystic Fibrosis will also be critical to attract investors. </p>
<H4 text="Question 4: Funding "/>
@@ -81,7 +81,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>RNhale initially relied on public funding from university grants and government programs such as GrowBio and EXIST, which provided crucial pre-seed support. As they transitioned into a private company, they secured additional funding through the European Union’s EIC Transition grant. They also attracted venture capital from firms specializing in biotech, such as the Hightech-Gründerfonds and international investors like Karma Fund and Wellington, who understood the long timelines and high costs associated with biotech development.</p>
<H5 text="Learnings and Implications for our project "/>
- <p>Both founders highlighted the importance of securing diverse funding sources early on. A concrete next step could be collaborating with the university’s startup support services to identify potential investors, especially those with biotech experience. Additionally, exploring non-traditional sources such as industry-sponsored research collaborations could provide crucial initial funding to support the development of our fibrosis gene therapy. </p>
+ <p>Both founders highlighted the importance of securing diverse funding sources early on. A concrete next step could be collaborating with the university’s startup support services to identify potential investors, especially those with biotech experience. Additionally, exploring non-traditional sources such as industry-sponsored research collaborations could provide crucial initial funding to support the development of our Cystic Fibrosis gene therapy. </p>
<H4 text="Question 5: Team Building "/>
<H5 text="What we asked the Founders"/>
@@ -127,7 +127,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>RNhale secured their intellectual property through university licensing and strategic patent filings. Early work was patented by the university, and they secured exclusive rights to use the technology for commercialization through a licensing agreement. For later developments, they took a strategic approach, filing priority patents to protect novelty and expanding patent claims within the 12-month window to cover commercially relevant aspects. They emphasized the importance of negotiating IP agreements early, especially when working with universities, and planning a robust patent strategy.</p>
<H5 text="Learnings and Implications for our project "/>
- <p>Both founders emphasized the importance of securing IP early, especially when working with universities or external partners. For our project, we should develop a clear patent strategy for our RNA-based fibrosis therapy. A concrete next step would be to consult with IP experts to ensure our technology is well protected. Negotiating early IP agreements with the university or external collaborators is crucial to safeguard our innovations while allowing room for future developments. </p>
+ <p>Both founders emphasized the importance of securing IP early, especially when working with universities or external partners. For our project, we should develop a clear patent strategy for our RNA-based Cystic Fibrosis therapy. A concrete next step would be to consult with IP experts to ensure our technology is well protected. Negotiating early IP agreements with the university or external collaborators is crucial to safeguard our innovations while allowing room for future developments. </p>
<H4 text="Question 9: Pivoting "/>
<H5 text="What we asked the Founders"/>
@@ -138,7 +138,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>RNhale had to adapt their original idea several times. One significant pivot was shifting from providing a service for lipid nanoparticle formulation to developing their own proprietary therapeutic product for severe asthma. Feedback from investors and participation in startup bootcamps revealed a stronger market demand for a product-driven approach with a clear exit strategy. This led them to revise their business model while still leveraging their core technology.</p>
<H5 text="Learnings and Implications for our project "/>
- <p>Both founders discussed the importance of remaining adaptable to feedback and market needs. For our project, we must be open to making strategic adjustments based on the feedback we receive from clinical trials, investors, or partners. A concrete next step would be to establish a flexible business plan that allows for pivots, such as focusing on specific subtypes of fibrosis patients or adjusting our lipid nanoparticle delivery system to meet evolving technological or regulatory requirements. </p>
+ <p>Both founders discussed the importance of remaining adaptable to feedback and market needs. For our project, we must be open to making strategic adjustments based on the feedback we receive from clinical trials, investors, or partners. A concrete next step would be to establish a flexible business plan that allows for pivots, such as focusing on specific subtypes of Cystic Fibrosis patients or adjusting our lipid nanoparticle delivery system to meet evolving technological or regulatory requirements. </p>
<H4 text="Question 10: Long-term Vision "/>
<H5 text="What we asked the Founders"/>
@@ -149,7 +149,7 @@ export function HPEntrepreneur(){
<p><b>RNhale (Benjamin Winkeljann)</b></p>
<p>RNhale’s long-term vision was to develop RNA-based therapeutics, particularly for respiratory diseases. They reconciled this vision with short-term goals by breaking their vision into actionable milestones, such as developing a lead candidate for severe asthma. Alongside their core therapeutic focus, they offered small-scale manufacturing services to generate revenue and build credibility. This dual approach helped them maintain momentum while working towards their larger goal of establishing a pipeline of RNA therapeutics. </p>
<H5 text="Learnings and Implications for our project "/>
- <p>Both founders highlighted the importance of aligning short-term goals with a long-term vision. For our project, we must ensure that while focusing on immediate milestones, such as demonstrating the efficacy of our RNA-based therapy, we maintain sight of our broader goal: revolutionizing fibrosis treatment. A concrete next step would be to break down our long-term vision into actionable short-term goals, such as optimizing our delivery system and securing regulatory approvals, while building a sustainable pipeline for future RNA therapeutics. </p>
+ <p>Both founders highlighted the importance of aligning short-term goals with a long-term vision. For our project, we must ensure that while focusing on immediate milestones, such as demonstrating the efficacy of our RNA-based therapy, we maintain sight of our broader goal: revolutionizing Cystic Fibrosis treatment. A concrete next step would be to break down our long-term vision into actionable short-term goals, such as optimizing our delivery system and securing regulatory approvals, while building a sustainable pipeline for future RNA therapeutics. </p>
</div>
@@ -157,7 +157,7 @@ export function HPEntrepreneur(){
<div id="ent-next" className="ent-interview" style={{display: "none"}}>
<H4 id="ent-course-heading" text="GXP in the context of clinical trials "/>
<H5 text="Role of GXP in Scaling and Proof-of-Concept"/>
- <p>To take our RNA-based gene therapy for fibrosis closer to clinical trials and potential market entry, investors and regulatory authorities need
+ <p>To take our RNA-based gene therapy for Cystic Fibrosis closer to clinical trials and potential market entry, investors and regulatory authorities need
confidence in the quality and reliability of our work. While the current iGEM proof-of-concept demonstrates feasibility, investors typically expect a
more sophisticated validation, especially in <b>In-Vivo models</b>. GXP would be fundamental in achieving this next step: </p>
<ul>
@@ -181,7 +181,7 @@ export function HPEntrepreneur(){
symptomatic care [4]. Our RNA-based gene therapy could address this unmet need, specifically targeting the Delta F508 mutation for which many patients have
limited options. </p>
<H5 text="Market Size and Growth Potential"/>
- <p><b>Market Size: </b> The global fibrosis treatment market was valued at USD 9.41 billion in 2023 and is expected to grow to USD 29.19 billion by
+ <p><b>Market Size: </b> The global Cystic Fibrosis treatment market was valued at USD 9.41 billion in 2023 and is expected to grow to USD 29.19 billion by
2032, with a compound annual growth rate (CAGR) of 13.4% [5]. This growth is driven by advancements in gene therapy and increased research funding. Gene
therapy targeting the F508del mutation, the most common CF mutation, presents a significant market opportunity within this larger CF treatment market[6]. </p>
<p><b>Growth Drivers:</b> The increase in CF patient lifespan due to improved treatments, alongside ongoing innovation in RNA-based therapies, offers
@@ -190,7 +190,7 @@ export function HPEntrepreneur(){
patients do not benefit from these therapies [9]. Our RNA-based therapy has the potential to capture this segment of the market, addressing an unmet
clinical need.</p>
<H5 text="3. Competitive Landscape "/>
- <p><b>Current Competitors:</b>The fibrosis treatment space is dominated by pharmaceutical giants such as Vertex Pharmaceuticals, which has developed
+ <p><b>Current Competitors:</b>The Cystic Fibrosis treatment space is dominated by pharmaceutical giants such as Vertex Pharmaceuticals, which has developed
CFTR modulators like Kaftrio/Trikafta. These modulators are currently the gold standard for treating CF patients with the F508del mutation [10].
Other key players in the market include Novartis, Gilead Sciences, and AbbVie, all of whom are active in CF drug development[11].</p>
<p><b>Gene Therapy Competitors:</b>While CFTR modulators have been highly successful, several companies are exploring gene therapies aimed at addressing the
@@ -202,7 +202,7 @@ export function HPEntrepreneur(){
<H5 text="4. Barriers to Entry "/>
<p><b>Regulatory Hurdles:</b>One of the biggest challenges in bringing a gene therapy to market is navigating the complex regulatory environment.
Compliance with Good Manufacturing Practice (GMP) and Good Laboratory Practice (GLP) is essential for obtaining approvals from bodies like the FDA and EMA.
- Securing approval for RNA-based gene therapies, particularly those targeting rare diseases like fibrosis, can involve lengthy and expensive clinical
+ Securing approval for RNA-based gene therapies, particularly those targeting rare diseases like Cystic Fibrosis, can involve lengthy and expensive clinical
trials[13][14].</p>
<p><b>High R&D Costs:</b> Developing gene therapies involves significant upfront costs, from research and development to clinical trials. For a small
biotech startup, securing the necessary funding can be a barrier, especially when competing against established pharmaceutical companies with larger R&D
@@ -216,17 +216,17 @@ export function HPEntrepreneur(){
<H5 text="5. Go-to-Market Strategy"/>
<p><b>Initial Focus on Clinical Partnerships:</b> The first step in bringing our RNA-based gene therapy to market will be partnering with academic
institutions and clinical research centers to conduct initial clinical trials. Establishing credibility through collaborations with key opinion leaders
- in fibrosis treatment will help build trust and validate the efficacy of our therapy [18][19].</p>
- <p><b>Early Adopters: </b>Our focus will be on targeting early adopters, such as specialized fibrosis clinics and hospitals that are familiar
+ in Cystic Fibrosis treatment will help build trust and validate the efficacy of our therapy [18][19].</p>
+ <p><b>Early Adopters: </b>Our focus will be on targeting early adopters, such as specialized Cystic Fibrosis clinics and hospitals that are familiar
with cutting-edge gene therapies. These institutions are more likely to adopt novel treatments and provide us with real-world data to further refine our
therapy[20].</p>
<p><b>Partnerships with Biotech and Pharmaceutical Companies:</b> Partnering with established biotech or pharmaceutical companies could help accelerate
commercialization by providing access to distribution channels, regulatory expertise, and additional funding. Licensing agreements or co-development
deals with companies specializing in gene therapy could be key to scaling production[21].</p>
<p><b>Regulatory Strategy:</b>Navigating the regulatory environment will be a priority, and early engagement with the FDA, EMA, and other regulatory
- bodies will help ensure a smoother approval process. Focusing on orphan drug designation or fast-track approvals for rare diseases like fibrosis
+ bodies will help ensure a smoother approval process. Focusing on orphan drug designation or fast-track approvals for rare diseases like Cystic Fibrosis
could expedite the regulatory timeline[22].</p>
- <p><b>Long-Term Vision:</b> After initial success in treating fibrosis, our RNA-based therapy could be expanded to treat other genetic disorders.
+ <p><b>Long-Term Vision:</b> After initial success in treating Cystic Fibrosis, our RNA-based therapy could be expanded to treat other genetic disorders.
The modular nature of our technology allows us to adapt the therapy for other rare diseases, providing a broader market potential in the future[23].</p>
</div>
diff --git a/src/contents/Human Practices/Further Engagement/Outreach.tsx b/src/contents/Human Practices/Further Engagement/Outreach.tsx
index 2a0709d89b8eda60c9422b44f1f47cb0bff9cd29..f03c4aecc8b20b5caea82a27d687e41859d674a3 100644
--- a/src/contents/Human Practices/Further Engagement/Outreach.tsx
+++ b/src/contents/Human Practices/Further Engagement/Outreach.tsx
@@ -25,7 +25,7 @@ export function HPOutreach(){
<H4 id="out-why-heading" text="Public engament as part of a integrated human practice - but why?"/>
<p>While many of our efforts in science communication were educational, we also recognized the importance of public engagement through activities
that were not focused on formal education but rather on raising awareness. Initiatives like MUKOmove and our waffle sale were essential in bringing
- fibrosis (CF) into public focus and showing a visible commitment to the cause.</p>
+ Cystic Fibrosis (CF) into public focus and showing a visible commitment to the cause.</p>
<p>Public enganegment and outreach serves as a reminder that science does not happen in isolation - it is rooted
in real-world problems that impact individuals and communities. Establishing presence allows diverting interest to our project and our cause which in turn
paves the way to edcuate interested people and to lay the groundwork for a deeper connection between us and our project and the general public. </p>
@@ -33,7 +33,7 @@ export function HPOutreach(){
<p>Our public engagement served both as a form of spreading awareness and to establish first contacts. By inviting other people in Bielefeld to join our team
for MUKOmove, we were able to reach a wider audience and foster connections that extended beyond our university.</p>
<p>In addition to our in-person events, we used social media as a tool to keep the community engaged and updated. We shared our progress in
- MUKOmove, promoted our events, and posted educational content about fibrosis and gene therapy. Our social media presence helped us
+ MUKOmove, promoted our events, and posted educational content about Cystic Fibrosis and gene therapy. Our social media presence helped us
stay connected with a broader audience, ensuring that even those who could not attend our events could still follow along and support our mission.</p>
<p>
Through these efforts, we also made valuable connections, resulting in an interview with the "Muko Dino" <a onClick={() => goToPagesAndOpenTab('dino', '/human-practices')}>Thomas Malenke</a>. This highlighted the power
@@ -45,8 +45,8 @@ export function HPOutreach(){
<H4 text="Waffle Sale"></H4>
<div className="row">
<div className="col">
- <p>To support our research project and raise funds for our iGEM team at Bielefeld University, we decided to organize a waffle sale in the main hall of the university. This initiative was aimed at raising awareness about our project and collecting funds for our research into fibrosis. </p>
- <p>The sale took place in fibrosis awareness month May in the Great Hall of our University. As people passed by, we engaged them by introducing our research group and explaining our project’s objectives. We shared information about fibrosis and why we are raising money. Our goal was to not only just to sell waffles, but also to educate the university community about our research and its’ potential impact. The response has been overwhelmingly positive. Many were genuinely interested in our work and asked for more details about our research and the goals of our project. This enthusiasm strengthened our commitment to the project and highlighted the importance of community involvement in scientific research. </p>
+ <p>To support our research project and raise funds for our iGEM team at Bielefeld University, we decided to organize a waffle sale in the main hall of the university. This initiative was aimed at raising awareness about our project and collecting funds for our research into Cystic Fibrosis. </p>
+ <p>The sale took place in Cystic Fibrosis awareness month May in the Great Hall of our University. As people passed by, we engaged them by introducing our research group and explaining our project’s objectives. We shared information about Cystic Fibrosis and why we are raising money. Our goal was to not only just to sell waffles, but also to educate the university community about our research and its’ potential impact. The response has been overwhelmingly positive. Many were genuinely interested in our work and asked for more details about our research and the goals of our project. This enthusiasm strengthened our commitment to the project and highlighted the importance of community involvement in scientific research. </p>
<p>The waffle sale was a great success, both in terms of raising funds and increasing awareness about our work within the university. It was a collaborative effort that brought our team closer together and demonstrated the power of community support in advancing scientific research. </p>
</div>
<div className="col-4">
@@ -58,17 +58,17 @@ export function HPOutreach(){
</div>
<div id="mukomove" className="out-cycletab" style={{display: "none"}}>
- <H4 id="cf-month" text="Cystic fibrosis awareness month"/>
+ <H4 id="cf-month" text="Cystic Cystic Fibrosis awareness month"/>
<div className="row">
<div className="full-small col-3">
<img className="max400" src="https://static.igem.wiki/teams/5247/photos/for-wiki-texts/po-mukomove/wir-plakat-mukomove.jpeg"></img>
</div>
<div className="col">
<h3>What is MUKOmove?</h3>
- <p>As part of the fibrosis awareness month May, we took part in this year's <a href="https://www.muko.info/mukomove">MUKOmove</a> from May 8th to May
+ <p>As part of the Cystic Fibrosis awareness month May, we took part in this year's <a href="https://www.muko.info/mukomove">MUKOmove</a> from May 8th to May
12th under the team name iGEM Bielefeld. MUKOmove is a participatory sports initiative organized by
<a href="https://www.muko.info/"> Mukoviszidose e.V.</a>, the German Cystic Fibrosis Association, aimed at raising awareness and funds
- for fibrosis research and support. This annual event encourages individuals and teams to
+ for Cystic Fibrosis research and support. This annual event encourages individuals and teams to
engage in various physical activities, track their movement, and share their experiences online. </p>
<button>Spenden</button>
</div>
@@ -80,8 +80,8 @@ export function HPOutreach(){
<p>While MUKOmove was not a scientific or educational event, it played an important role in demonstrating our presence in the broader CF
community.</p>
<p>We did not stop at our participation itself - we wanted to make other people from our university and city
- aware of the event and collect sport hours for fibrosis with them by inviting them to join our team.
- Our survey about fibrosis awareness and our discussions with <a onClick={() => goToPagesAndOpenTab('InvWesthoff', '/human-practices?tab=')}>Kathrin Westhoff</a>, the head of a
+ aware of the event and collect sport hours for Cystic Fibrosis with them by inviting them to join our team.
+ Our survey about Cystic Fibrosis awareness and our discussions with <a onClick={() => goToPagesAndOpenTab('InvWesthoff', '/human-practices?tab=')}>Kathrin Westhoff</a>, the head of a
practice for physiotherapy in Gütersloh who is also treating young CF patients, showed us how little known
this illness still is. Especially the interview with the physiotherapist made us realize how important
exercise is for everyone and how it brings a community together - we wanted to establish MUKOmove in
@@ -107,7 +107,7 @@ export function HPOutreach(){
<br/>
<h3>What did we achieve?</h3>
<p>We are happy to announce that both movement goals were surpassed: Team iGEM Bielefeld was able to collect 358 sport hours, and everyone who took part in MUKOmove together collected 36,542 sport hours! </p>
- <p>The MUKOmove was therefore a complete success, and we take pride in having contributed to the goal while raising more awareness for fibrosis in our region and Germany as a whole.</p>
+ <p>The MUKOmove was therefore a complete success, and we take pride in having contributed to the goal while raising more awareness for Cystic Fibrosis in our region and Germany as a whole.</p>
</div>
</div>
);
diff --git a/src/contents/Human Practices/Further Engagement/Partnerships.tsx b/src/contents/Human Practices/Further Engagement/Partnerships.tsx
index fc8329382486e292240f1c2479f0945a48857a7b..535fb143cc02fd0f63e4a73cfe8141d0dba9655b 100644
--- a/src/contents/Human Practices/Further Engagement/Partnerships.tsx
+++ b/src/contents/Human Practices/Further Engagement/Partnerships.tsx
@@ -6,8 +6,8 @@ export function HPPartnerships(){
return(
<div className="col">
<H4 text="CF Vests"></H4>
- <p>CF Vests Worldwide is a dedicated charity organization committed to providing life-saving vest equipment to those in need, regardless of their financial situation. But they can't do it alone — they need your support. Help us make a difference! By donating to CFVWW, you can directly impact the lives of fibrosis patients, giving them the chance to breathe easier and live fuller lives. Every contribution counts.</p>
- <p><strong>Join us in the fight against fibrosis.</strong> <a href="https://donorbox.org/igem"> Donate today</a> and help us bring hope, one vest at a time! Together, we can change lives.</p>
+ <p>CF Vests Worldwide is a dedicated charity organization committed to providing life-saving vest equipment to those in need, regardless of their financial situation. But they can't do it alone — they need your support. Help us make a difference! By donating to CFVWW, you can directly impact the lives of Cystic Fibrosis patients, giving them the chance to breathe easier and live fuller lives. Every contribution counts.</p>
+ <p><strong>Join us in the fight against Cystic Fibrosis.</strong> <a href="https://donorbox.org/igem"> Donate today</a> and help us bring hope, one vest at a time! Together, we can change lives.</p>
</div>
)
}
\ No newline at end of file
diff --git a/src/contents/Human Practices/HP svgs/swots.tsx b/src/contents/Human Practices/HP svgs/swots.tsx
index a84aa18547057f0a5da2115a011a9849a87e1a95..8580912f7f76edf2bdca946413cfb146b2c868f0 100644
--- a/src/contents/Human Practices/HP svgs/swots.tsx
+++ b/src/contents/Human Practices/HP svgs/swots.tsx
@@ -255,7 +255,7 @@ export function SWOTone(){
textDecoration="none"
transform="matrix(1 0 0 1 640.685 5388)"
>
- {"of fibrosis"}
+ {"of Cystic Fibrosis"}
</text>
<text
fill="#000000"
diff --git a/src/contents/Human Practices/HP-abstract.tsx b/src/contents/Human Practices/HP-abstract.tsx
index f9c9f069011a561ab1564c800b618c7a0a32d02d..84a5459d444405241878320604c8234d0536b64a 100644
--- a/src/contents/Human Practices/HP-abstract.tsx
+++ b/src/contents/Human Practices/HP-abstract.tsx
@@ -5,7 +5,7 @@ export function HPAbstract(){
useTabNavigation();
return(
<Section title="Abstract" id="Abstract">
- <p>As the iGEM Bielefeld-CeBiTec team, we adopted a human-centered approach and delivered an extensive framework of <strong>eight reglementations</strong> for future iGEM teams, establishing <strong>eight frameworks</strong> that were validated through our project. Our methodology was shaped by over <strong>80 interviews with stakeholders and institutions</strong> , creating a comprehensive and multidimensional perspective on the complex challenge of gene therapy for fibrosis (CF). Through <strong>33 key interviews</strong> , we navigated <strong>seven thematic areas</strong> simultaneously, incorporating <strong>five iterative feedback loops</strong> to ensure the project’s refinement at every stage.
+ <p>As the iGEM Bielefeld-CeBiTec team, we adopted a human-centered approach and delivered an extensive framework of <strong>eight reglementations</strong> for future iGEM teams, establishing <strong>eight frameworks</strong> that were validated through our project. Our methodology was shaped by over <strong>80 interviews with stakeholders and institutions</strong> , creating a comprehensive and multidimensional perspective on the complex challenge of gene therapy for Cystic Fibrosis (CF). Through <strong>33 key interviews</strong> , we navigated <strong>seven thematic areas</strong> simultaneously, incorporating <strong>five iterative feedback loops</strong> to ensure the project’s refinement at every stage.
</p>
<p> Our approach offers future iGEM teams and the synthetic biology community a valuable blueprint for responsible and ethical project development. By actively engaging with CF patients, clinicians, researchers, and regulatory bodies, we ensured that real-world needs and societal values were central to our project's evolution. Our efforts culminated in a thoughtful, scientifically sound solution, setting new standards for accessibility, patient focus, and regulatory compliance. Future teams can leverage our documented methodologies to further their own impact, ensuring that innovation remains aligned with the needs of those it serves.
</p>
diff --git a/src/contents/Human Practices/IHP.tsx b/src/contents/Human Practices/IHP.tsx
index 02254461670d6f0f51de99023928ece773657931..a1d0c4bb693270125e75067332e510e33dab4554 100644
--- a/src/contents/Human Practices/IHP.tsx
+++ b/src/contents/Human Practices/IHP.tsx
@@ -64,7 +64,7 @@ export function HPIntegrated(){
<p>To clearly communicate to our stakeholders how they can support the <PreCyse/> project and the specific areas requiring
their input, we developed a stakeholder management framework highlighting four key areas. Starting at the bottom right of the
figure and moving clockwise, the first area needing feedback is <b>implementation</b>. It is crucial for our team to ensure that the project
- has all necessary components for realistic application in healthcare settings, focusing on the gene therapy treatment for fibrosis.
+ has all necessary components for realistic application in healthcare settings, focusing on the gene therapy treatment for Cystic Fibrosis.
This includes both the technical design and the appropriate business model to ensure successful implementation.
</p>
<p>The second key area is <b>sustainability and social impact.</b> We seek to understand how our project aligns with current healthcare initiatives and facilities in Germany and Europe, but also in the globally, and how it interacts with social efforts related to CF awareness and gene therapy strategies.
@@ -116,9 +116,9 @@ export function HPIntegrated(){
<p>
Close support and extensive feedback are necessary to successfully implement <PreCyse/>.
Target groups include experts and physicians with expertise in gene therapy and treatment
- strategies for fibrosis. The project has a reasonable, responsible, and future-oriented
+ strategies for Cystic Fibrosis. The project has a reasonable, responsible, and future-oriented
significance for the world. Furthermore, scientific-technological knowledge and biosafety are
- crucial. Collaboration with specialists in the field of gene therapy, fibrosis, and different
+ crucial. Collaboration with specialists in the field of gene therapy, Cystic Fibrosis, and different
treatment strategies ensures the quality and effectiveness of the solutions developed.
</p>
@@ -178,7 +178,7 @@ export function HPIntegrated(){
<p><b>Patients and patient organizations</b></p>
<p>
- Patients suffering from fibrosis and the organizations that support them have a high interest in
+ Patients suffering from Cystic Fibrosis and the organizations that support them have a high interest in
new therapies that could improve their quality of life. However, their power to influence the project
is limited.
</p>
@@ -771,7 +771,7 @@ export function HPIntegrated(){
</div>
<H5 text="Agenda"/>
<p>
- The central goal of the <PreCyse/> project is to develop an innovative gene therapy solution for fibrosis (CF) that is not only technically effective but also socially acceptable and ethically justifiable. The primary questions include:
+ The central goal of the <PreCyse/> project is to develop an innovative gene therapy solution for Cystic Fibrosis (CF) that is not only technically effective but also socially acceptable and ethically justifiable. The primary questions include:
</p>
<ul>
<li>How can the project improve the lives of CF patients?</li>
diff --git a/src/contents/Human Practices/Introduction.tsx b/src/contents/Human Practices/Introduction.tsx
index 4e98cd6b27a67b4cd720a6f017a58df61a3cd6a4..b17ca428afa01a4a15e2fdfbf9a9f161f6bb7c84 100644
--- a/src/contents/Human Practices/Introduction.tsx
+++ b/src/contents/Human Practices/Introduction.tsx
@@ -33,13 +33,13 @@ export function HPIntroduction(){
<p>This year, we at iGEM Bielefeld-CeBiTec have consciously chosen a <b>human-centered project design</b>. At the heart of our iGEM project is
one key pillar: <b>Human Practice</b>. Our goal is to understand the impact of our project on society, the scientific community and the world
as whole. This is not just about the technical effectiveness of our parts, but also about how the solution is embraced in everyday
- practice, and the potential long-term impact it could have for fibrosis patients and their families all over the world.
+ practice, and the potential long-term impact it could have for Cystic Fibrosis patients and their families all over the world.
</p>
<p>With our human-centered approach, we aim to address fundamental iGEM Human Practice questions and beyond:
</p>
<p style={{textAlign: "center"}}> <b>How does our project affect the world around us?</b></p>
<p style={{textAlign: "center"}}> <b>How does the world around us influence our project? </b></p>
- <p>From the very beginning, it was our priority to identify various stakeholders and meet people affected by fibrosis early on to <b>actively involve</b> them throughout the planning and development process. This collaborative approach allowed us to ensure that our project
+ <p>From the very beginning, it was our priority to identify various stakeholders and meet people affected by Cystic Fibrosis early on to <b>actively involve</b> them throughout the planning and development process. This collaborative approach allowed us to ensure that our project
addresses real needs and contributes to solutions for as many different people as possible. Without the critical advice, varied perspectives
and input from our stakeholders, it would have been impossible to identify and reflect on all aspects of our project. We made every
effort to <b>deeply understand</b> their values and backgrounds, allowing us to integrate their feedback into our solutions.
@@ -47,12 +47,12 @@ export function HPIntroduction(){
</div>
<div className="col intro-cycletab" id="mission" style={{display: "none"}}>
<p>We view Human Practice as an opportunity to <b>go beyond practical lab work and traditional science</b> and to learn about
- the needs of people affected by fibrosis. It’s a chance for us to creatively engage with different aspects of our project
+ the needs of people affected by Cystic Fibrosis. It’s a chance for us to creatively engage with different aspects of our project
while developing an awareness of the responsibilities that come with it.
</p>
<p>
As part of our <PreCyse/> project, we performed intensive brainstorming sessions and expert consultations. We conducted comprehensive
- <a onClick={() => goToPageAndScroll("our-surveys-on--fibrosis-and-gene-therapy", "/human-practices")}>surveys</a> among the public and people with fibrosis and their relatives. We focused on critical aspects such as the <b>needs of our
+ <a onClick={() => goToPageAndScroll("our-surveys-on--Cystic Fibrosis-and-gene-therapy", "/human-practices")}>surveys</a> among the public and people with Cystic Fibrosis and their relatives. We focused on critical aspects such as the <b>needs of our
target groups, safety, ethics, design, implementation, and business</b> — each guided by the core values of our team. Based on these interactions
and the recommendations of the Human Practice committee, we have developed an optimal strategy for our project, ensuring that our work is not
only innovative but also mindful of its broader impact on society.
@@ -61,7 +61,7 @@ export function HPIntroduction(){
</p>
</div>
<div className="col intro-cycletab" id="approach" style={{display: "none"}}>
- <p>It was important to us as a team to not only offer technical solutions, but to show that our project can contribute to the larger context of ongoing initiatives and movements to optimize health care. We wanted to really understand the feedback and insights of the stakeholders to gain a better understanding of how our project fits into the overall picture of living with fibrosis, the current state of research and how it can be used to reduce the health care gap.
+ <p>It was important to us as a team to not only offer technical solutions, but to show that our project can contribute to the larger context of ongoing initiatives and movements to optimize health care. We wanted to really understand the feedback and insights of the stakeholders to gain a better understanding of how our project fits into the overall picture of living with Cystic Fibrosis, the current state of research and how it can be used to reduce the health care gap.
</p>
<p>Our strategy includes:</p>
<ul>
@@ -75,7 +75,7 @@ export function HPIntroduction(){
</ul>
<p>With this approach and the support of our stakeholders, our ultimate goals are to:</p>
<ul>
- <li>Improve care for fibrosis patients</li>
+ <li>Improve care for Cystic Fibrosis patients</li>
<li>Optimize the availability of essential medications</li>
</ul>
diff --git a/src/contents/description.tsx b/src/contents/description.tsx
index d597eeaa769be89837d1a8b1667fee8b888fc2f6..ff01eec140f0eb352d153917721779eb83f34973 100644
--- a/src/contents/description.tsx
+++ b/src/contents/description.tsx
@@ -26,7 +26,7 @@ export function Description() {
<div className="col">
<Section title="Abstract" id="Abstract">
<p id="obenindescription">We are proud to present <PreCyse/>, a next-generation Prime Editing technology, as innovative gene therapy approach for Cystic Fibrosis (CF)
- specifically targeting the most common mutation <b>F508del</b> of the CFTR gene. Cystic fibrosis is a severe disorder that primarily affects the lungs
+ specifically targeting the most common mutation <b>F508del</b> of the CFTR gene. Cystic Cystic Fibrosis is a severe disorder that primarily affects the lungs
and for which only short-term symptomatic treatments exist. PreCyse aims to provide long-term relief by delivering a small genetic payload with speed
and precision. Our approach integrates <b>PrimeGuide</b>, a highly optimized Prime Editing system, with <b>AirBuddy</b>, a novel lipid nanoparticle
(LNP) delivery platform. The <b>SORT LNPs</b> used in AirBuddy are optimized for pulmonary delivery, offering precise organ targeting and structural
@@ -162,7 +162,7 @@ export function Description() {
</Subesction>
<Subesction title="Diagnosis" id="Cystic Fibrosis5">
<p>With Cystic Fibrosis being a hereditary disease, the diagnostic methods have evolved significantly <SupScrollLink label="56"/> <SupScrollLink label="57"/> . Early diagnosis is crucial, as it allows for timely interventions that can improve the quality of life and longevity for CF patients <SupScrollLink label="56"/><sup>,</sup><SupScrollLink label="58"/>. With advancements in screening and diagnostic tools, many individuals are diagnosed shortly after birth, enabling early management of the disease <SupScrollLink label="56"/> <SupScrollLink label="61"/> .</p>
- <p>Cystic fibrosis can be diagnosed through a variety of methods, often starting in infancy or even before birth <SupScrollLink label="56"/> <SupScrollLink label="60"/> . The most common diagnostic test is the newborn screening, which involves a blood test that checks for elevated levels of a protein called immunoreactive trypsinogen (IRT) <SupScrollLink label="59"/> <SupScrollLink label="61"/> . Elevated IRT levels can indicate potential CF, prompting further testing <SupScrollLink label="61"/> . </p>
+ <p>Cystic Cystic Fibrosis can be diagnosed through a variety of methods, often starting in infancy or even before birth <SupScrollLink label="56"/> <SupScrollLink label="60"/> . The most common diagnostic test is the newborn screening, which involves a blood test that checks for elevated levels of a protein called immunoreactive trypsinogen (IRT) <SupScrollLink label="59"/> <SupScrollLink label="61"/> . Elevated IRT levels can indicate potential CF, prompting further testing <SupScrollLink label="61"/> . </p>
<Collapsible id="newborn-screening-collapsible" title="Newborn screening">
<p>Newborn screening for Cystic Fibrosis (CF) has been a major advancement in early detection and management, leading to significantly improved patient outcomes. This practice, which started in the late 1960s, became more widespread in the 1970s. The screening typically involves a blood test within the first few days of life, measuring immunoreactive trypsinogen (IRT), a marker that is elevated in newborns with CF. Elevated IRT levels prompt further genetic testing to identify CFTR mutations <SupScrollLink label="62"/> . If mutations are found, a sweat chloride test is often conducted to confirm the diagnosis. </p>
<p>Many countries, including the United States, Canada, the United Kingdom, Australia, and several European nations, have implemented newborn screening programs for CF. However, a survey of CF screening in Europe revealed that the implementation of such programs varies widely, with some countries adopting more comprehensive protocols than others <SupScrollLink label="63"/> . Early diagnosis through screening offers significant benefits, such as improved growth, better lung function, and overall enhanced health outcomes <SupScrollLink label="64"/> . The discovery of the CFTR gene has further refined diagnostic techniques and underscored the crucial role of newborn screening in the early detection and management of CF. </p>
@@ -175,7 +175,7 @@ export function Description() {
</Collapsible>
</Subesction>
<Subesction title="Treatment" id="Cystic Fibrosis6">
- <p>Current Cystic fibrosis treatments focus on managing symptoms, slowing disease progression, and improving quality of life <SupScrollLink label="66"/> . Since there is still no cure for CF, treatment is typically lifelong and involves multiple approaches, including medications, physical therapy, and dietary adjustments <SupScrollLink label="66"/> <SupScrollLink label="67"/> . </p>
+ <p>Current Cystic Cystic Fibrosis treatments focus on managing symptoms, slowing disease progression, and improving quality of life <SupScrollLink label="66"/> . Since there is still no cure for CF, treatment is typically lifelong and involves multiple approaches, including medications, physical therapy, and dietary adjustments <SupScrollLink label="66"/> <SupScrollLink label="67"/> . </p>
<p>The primary goal of CF treatment is to clear the thick mucus from the lungs to prevent infections and improve breathing <SupScrollLink label="68"/> . Airway clearance techniques, such as chest physiotherapy, are often used alongside inhaled medications, like bronchodilators and mucolytics, to thin the mucus and open the airways <SupScrollLink label="69"/> <SupScrollLink label="70"/> . Antibiotics are frequently prescribed to treat or prevent lung infections caused by trapped bacteria in the airways <SupScrollLink label="71"/> .</p>
<p>One of the most significant advances in CF treatment has been the development of CFTR modulators, which target the underlying protein dysfunction caused by mutations in the CFTR gene <SupScrollLink label="70"/> <SupScrollLink label="72"/> . These drugs, such as ivacaftor, lumacaftor, and elexacaftor, work by improving the function of the defective CFTR protein, particularly in patients with specific mutations like F508del <SupScrollLink label="23"/> <SupScrollLink label="24"/> . While CFTR modulators can dramatically improve lung function and overall health in many patients, they are not effective for all CFTR mutations and often are very expensive <SupScrollLink label="73"/> .</p>
<p>Digestive enzyme supplements are essential for CF patients who suffer from pancreatic insufficiency, helping them to absorb nutrients from food <SupScrollLink label="68"/> . Additionally, high-calorie diets and vitamins are recommended to support growth and maintain body weight <SupScrollLink label="68"/> .</p>
diff --git a/src/data/drug-data.tsx b/src/data/drug-data.tsx
index 31942fed14f3d1ae2c5c3b3e170cb7a1795489c5..d179b7c68ec744c42cfd73c74f4aa4d7f9816c3c 100644
--- a/src/data/drug-data.tsx
+++ b/src/data/drug-data.tsx
@@ -88,7 +88,7 @@ export const drugdata: (Array<DrugDatensatz>) = [
{
name: "Digestive enzymes and diet", //ein beispiel
picture: "https://static.igem.wiki/teams/5247/scientific-figures/diet.svg",
- introduction: <> The digestive process is impaired in 80% of patients with fibrosis (CF), as a result of pancreatic insufficiency, which in turn leads to difficulties in digesting food and absorbing nutrients. Enzyme supplements like Creon® are therefore essential [16]. Moreover CF patients are also advised to eat a balanced and energy-rich diet, as the increased work of breathing and increased coughing, as well as infections, fever and diarrhoea, consume more energy than a healthy person. <SupScrollLink label="74"/> It is also an option for patients to use nutritional supplements. Electrolyte preparations are also used in this context to compensate for the increased need for fluids and the required salts.</>,
+ introduction: <> The digestive process is impaired in 80% of patients with Cystic Fibrosis (CF), as a result of pancreatic insufficiency, which in turn leads to difficulties in digesting food and absorbing nutrients. Enzyme supplements like Creon® are therefore essential [16]. Moreover CF patients are also advised to eat a balanced and energy-rich diet, as the increased work of breathing and increased coughing, as well as infections, fever and diarrhoea, consume more energy than a healthy person. <SupScrollLink label="74"/> It is also an option for patients to use nutritional supplements. Electrolyte preparations are also used in this context to compensate for the increased need for fluids and the required salts.</>,
examples: [
{
title: "Creon", //quelle 17
diff --git a/src/data/hptimelinedata.tsx b/src/data/hptimelinedata.tsx
index 2b63eabed756165431afb259f4ffb96d348dea3a..af932884d9ca9c5d37c041402f45fae2e89763aa 100644
--- a/src/data/hptimelinedata.tsx
+++ b/src/data/hptimelinedata.tsx
@@ -182,7 +182,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
cardtext: "",
quoteNachname: "Köhler, Teammember",
quoteVorname: "Vera",
- quote: "Firstly, we discussed various project ideas, including the use of magnetic microswimmers for targeted medical applications, gene editing approaches for fibrosis, treatments for muscular dystrophy and combating cyanobacteria with algae.",
+ quote: "Firstly, we discussed various project ideas, including the use of magnetic microswimmers for targeted medical applications, gene editing approaches for Cystic Fibrosis, treatments for muscular dystrophy and combating cyanobacteria with algae.",
type: "meta",
summary: [<p>During our initial discussions about project ideas, the team explored several innovative concepts before honing in on <b> Cystic Fibrosis</b>. Each project presented unique scientific challenges and potential impacts.</p>,
<p>One idea involved the development of <b>magnetic microswimmers</b> designed for targeted cancer therapy, particularly for ovarian cancer. The proposal aimed to overcome the limitations of traditional treatments, such as radiotherapy and cisplatin, which often damage healthy cells and result in numerous side effects. The microswimmers would be biodegradable algae-based vehicles, magnetically guided to deliver drugs directly to cancerous cells while minimizing damage to surrounding tissue. This approach offered a novel, non-invasive targeting mechanism, leveraging the potential of magnetic fields to direct the swimmers precisely to the affected areas.</p>,
@@ -226,7 +226,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
language: "de",
quoteNachname:"Beckmann, Patient",
quoteVorname: "Max",
- quote: "A friend of mine with fibrosis recently got a fungal infection that he can't get rid of. His situation really struck me; it showed how quickly a seemingly minor issue can worsen a person's life, especially for someone like us. It’s a strong reminder of how fragile our health is and how fast things can change without warning.",
+ quote: "A friend of mine with Cystic Fibrosis recently got a fungal infection that he can't get rid of. His situation really struck me; it showed how quickly a seemingly minor issue can worsen a person's life, especially for someone like us. It’s a strong reminder of how fragile our health is and how fast things can change without warning.",
aimofcontact: [<p>When CF came up as a possible topic, we reached out to a teammate's friend Max in the hopes of getting insights into the needs of CF patients and current treatments to verify the need for further treatment options.
Since he was much more enthusiastic and open for discussion than we dared to hope, we extended our exchanges into the realms of the reality of life for CF patients, possible progressions, organizations and doctors in our area and his personal perspectives and values.
The interest in meeting him grew in the whole team and we invited him to one of our meetings. </p>],
@@ -332,12 +332,12 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteVorname: "Prof. Dr. Kristiann",
quote: "AAVs have been widely used in gene therapy, but their scalability and immunogenicity pose challenges, particularly when re-dosing is required. In contrast, LNPs offer a promising alternative, with a higher packaging capacity and lower immunogenicity. While AAVs excel in targeted delivery, LNPs could become a more scalable and cost-effective solution for delivering gene-editing tools in the future.",
aimofcontact: [<p>The aim of our interview with Prof. Dr. Kristian Müller was to gain expert insights into the potential of prime editing technology and its application in gene therapy, particularly for treating CF. We sought to understand how innovations in prime editing, combined with optimized delivery systems, could enhance therapeutic outcomes. Prof. Müller’s expertise in molecular biology and gene editing provided valuable perspectives on the opportunities and challenges in developing precise, efficient, and safe treatment strategies for genetic disorders.</p>],
- insights: [<p>During the interview, Prof. Dr. Müller highlighted the growing importance of prime editing as a powerful tool for gene therapy. Unlike traditional CRISPR-Cas systems, which often result in double-strand DNA breaks, prime editing allows for precise single-strand cuts, minimizing off-target effects and enabling more specific genetic corrections. This technology opens up new possibilities for treating diseases with known mutations, such as fibrosis.
+ insights: [<p>During the interview, Prof. Dr. Müller highlighted the growing importance of prime editing as a powerful tool for gene therapy. Unlike traditional CRISPR-Cas systems, which often result in double-strand DNA breaks, prime editing allows for precise single-strand cuts, minimizing off-target effects and enabling more specific genetic corrections. This technology opens up new possibilities for treating diseases with known mutations, such as Cystic Fibrosis.
Prof. Dr. Kristian Müller emphasized the critical role of delivery systems in the success of gene therapies, particularly in the context of CF treatment. Two primary delivery mechanisms were discussed: AAVs (Adeno-associated viruses) and LNPs (Lipid nanoparticles), each with distinct advantages and limitations.
- AAVs are a well-established vehicle in gene therapy, having been used successfully in various approved treatments. They are highly efficient at delivering genetic material to target cells, especially in well-characterized diseases like CF. One of their key strengths is their ability to precisely target specific tissues, making them particularly valuable for lung delivery in fibrosis. However, AAVs come with notable challenges, primarily their limited packaging capacity (approximately 4.5 kilobases), which constrains the size of the genetic payload they can carry. Additionally, AAVs can elicit immune responses, particularly when multiple doses are required, posing a barrier to their long-term use.
+ AAVs are a well-established vehicle in gene therapy, having been used successfully in various approved treatments. They are highly efficient at delivering genetic material to target cells, especially in well-characterized diseases like CF. One of their key strengths is their ability to precisely target specific tissues, making them particularly valuable for lung delivery in Cystic Fibrosis. However, AAVs come with notable challenges, primarily their limited packaging capacity (approximately 4.5 kilobases), which constrains the size of the genetic payload they can carry. Additionally, AAVs can elicit immune responses, particularly when multiple doses are required, posing a barrier to their long-term use.
On the other hand, LNPs offer a scalable and re-dosable alternative. LNPs have the advantage of a larger packaging capacity, allowing them to carry more complex genetic instructions or larger gene-editing tools, such as prime editors. They are also easier and cheaper to produce on a large scale, making them an attractive option for widespread clinical applications. A significant benefit of LNPs is their lower immunogenicity, which reduces the risk of adverse immune reactions upon repeated dosing. However, LNPs currently face challenges in specific targeting compared to AAVs. AAVs have a higher precision in targeting specific tissues, while LNPs still need optimization for targeted delivery to areas like the lungs.</p>],
implementation: [<p>Prof. Müller’s insights directly inform the implementation of our iGEM project, where we aim to design novel prime editors that are small enough to be delivered efficiently, while also exploring LNPs{/* [LINK Cycle Delivery] */} as a scalable and re-dosable alternative to AAVs. By tailoring our approach to address the specific challenges of CF, such as mucus penetration and lung cell targeting, we can enhance the precision and efficacy of gene therapy. These innovations have the potential to set new standards in the field and contribute to broader research on genetic disease treatment.</p>],
- summary: "In our interview with Prof. Dr. Kristian Müller, we explored the revolutionary potential of prime editing as a next-generation gene editing technology. Prof. Müller highlighted the advantages of prime editing over traditional CRISPR-Cas systems, particularly its ability to make precise genetic modifications without double-strand breaks, thus reducing off-target effects. He emphasized the importance of optimizing delivery systems, such as AAV and LNPs, and discussed the ethical considerations and biosafety measures crucial for advancing gene therapy. The interview underscored the significance of fibrosis as a model disease, given its prevalence and the potential for impactful treatments through targeted genetic corrections.",
+ summary: "In our interview with Prof. Dr. Kristian Müller, we explored the revolutionary potential of prime editing as a next-generation gene editing technology. Prof. Müller highlighted the advantages of prime editing over traditional CRISPR-Cas systems, particularly its ability to make precise genetic modifications without double-strand breaks, thus reducing off-target effects. He emphasized the importance of optimizing delivery systems, such as AAV and LNPs, and discussed the ethical considerations and biosafety measures crucial for advancing gene therapy. The interview underscored the significance of Cystic Fibrosis as a model disease, given its prevalence and the potential for impactful treatments through targeted genetic corrections.",
months: "April",
interview:<><iframe title="Bielefeld-CeBiTec: Interview Müller AAV vs LNP (2024) [English]" width="560" height="315" src="https://video.igem.org/videos/embed/0613b6b8-7755-4373-9d86-9910fe30781f" frameBorder="0" allowFullScreen={true} sandbox="allow-same-origin allow-scripts allow-popups allow-forms"></iframe><p>This interview was recorded on video at a later date.</p></>,
},
@@ -348,19 +348,19 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
job: "Team iGEM",
affiliation: "Bielfeld CeBiTec 2024",
tag: "Milestone",
- heading: "Identifying key experts in fibrosis and prime editing",
+ heading: "Identifying key experts in Cystic Fibrosis and prime editing",
interviewtabid: "experts",
cardtext: "",
- quote: "Focusing on gene therapy for fibrosis has been a meaningful journey for our team. Collaborating with experts has enriched our understanding and helped us refine our approach, especially in exploring prime editing. We're eager to turn our plans into reality and make a real impact",
+ quote: "Focusing on gene therapy for Cystic Fibrosis has been a meaningful journey for our team. Collaborating with experts has enriched our understanding and helped us refine our approach, especially in exploring prime editing. We're eager to turn our plans into reality and make a real impact",
quoteNachname:"Kanthak, Teammember",
quoteVorname: "Kai",
type: "meta",
- summary: [<p>After our team came together and thoroughly explored various project ideas, we decided to focus on gene therapy for fibrosis, largely due to a personal connection with a close friend affected by the condition. Up until that point, we had not yet developed a concrete concept, so we sought to engage with experts in order to broaden our understanding of the latest advancements in gene therapy.
- In addition to grasping the importance of a functional gene therapy, we delved into different strategies regarding the underlying mechanisms and the best delivery methods for the therapy. While the general topic of our project was clear, we now faced the challenge of working out the details. At this stage, we decided to consult further experts in the field of fibrosis to deepen our knowledge and refine our approach.
- Through connections with the University Hospital Münster and our local hospital, we aimed to gain a comprehensive overview of the clinical applications of gene therapy and the current research in fibrosis. These consultations with specialists allowed us to acquire valuable insights into different therapeutic options and laid the groundwork for our own exploration of potential strategies, particularly in the area of prime editing as a promising treatment avenue.</p>,
+ summary: [<p>After our team came together and thoroughly explored various project ideas, we decided to focus on gene therapy for Cystic Fibrosis, largely due to a personal connection with a close friend affected by the condition. Up until that point, we had not yet developed a concrete concept, so we sought to engage with experts in order to broaden our understanding of the latest advancements in gene therapy.
+ In addition to grasping the importance of a functional gene therapy, we delved into different strategies regarding the underlying mechanisms and the best delivery methods for the therapy. While the general topic of our project was clear, we now faced the challenge of working out the details. At this stage, we decided to consult further experts in the field of Cystic Fibrosis to deepen our knowledge and refine our approach.
+ Through connections with the University Hospital Münster and our local hospital, we aimed to gain a comprehensive overview of the clinical applications of gene therapy and the current research in Cystic Fibrosis. These consultations with specialists allowed us to acquire valuable insights into different therapeutic options and laid the groundwork for our own exploration of potential strategies, particularly in the area of prime editing as a promising treatment avenue.</p>,
<ul>
<li>
- <b>Team Formation & Research:</b> Chose gene therapy for fibrosis and explored mechanisms and delivery strategies.
+ <b>Team Formation & Research:</b> Chose gene therapy for Cystic Fibrosis and explored mechanisms and delivery strategies.
</li>
<li>
<b>Expert Engagement:</b> Consulted with specialists to refine approach, focusing on prime editing.
@@ -409,7 +409,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
cardtext: "",
language: "en",
quote: "This is also an innovative step that you have developed yourselves. That's the part that's really new and significant. A clever and exciting approach with great potential.",
- aimofcontact: [<p>The aim of the interview was to gain expert insights on optimizing the delivery of CFTR-mRNA via lung-targeted lipid nanoparticles (LNPs) for fibrosis (CF) treatment.
+ aimofcontact: [<p>The aim of the interview was to gain expert insights on optimizing the delivery of CFTR-mRNA via lung-targeted lipid nanoparticles (LNPs) for Cystic Fibrosis (CF) treatment.
Specifically, the goal was to explore potential cell targets, challenges in delivery mechanisms, and technical tools for assessing the effectiveness of mRNA therapies like the Ussing chamber system. </p>],
insights: [<p>The experts highlighted the potential of targeting ionocytes, given their key role in CFTR expression, but emphasized the difficulty in accessing them due to their basal position in the respiratory epithelium.
While Prof. Weber found ionocytes to be an intriguing target, Dr. Große-Onnebrink pointed out that there is still limited understanding of their exact role in CF pathology. Both stressed the challenge of penetrating the
@@ -442,11 +442,11 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
language: "de",
quoteNachname: "Olariu, Clinical Physician and CF Expert",
quoteVorname: "Dr. Cristian-Gabriel",
- quote: "For most families, it’s a shock. Cystic fibrosis still has a strong association with being a life-threatening disease, despite the fact that we now have good treatments, and many patients can live healthy lives. The diagnosis puts a huge psychological strain on the family, especially when dealing with very young children.",
+ quote: "For most families, it’s a shock. Cystic Cystic Fibrosis still has a strong association with being a life-threatening disease, despite the fact that we now have good treatments, and many patients can live healthy lives. The diagnosis puts a huge psychological strain on the family, especially when dealing with very young children.",
aimofcontact: "To gain a deeper insight into the path to diagnosis, we invited pediatrician Dr. Cristian-Gabriel Olariu from the University Department of Pediatrics and Adolescent Medicine to share his experiences with CF patients with us. We interviewed him because of his expertise in the effects of diagnosis on the patient and the family members, but also on daily life. Additionally, we want to close the gap and create a bridge between academic research and clinical applications. Therefore, Dr. Olariu gave us insights about the clinical perspectives on CF patients.",
- insights: [<p>We invited Max, our CF patient contact, to join Dr. Olariu in discussing the intersection of academic research, clinical application, and patient needs. Through our connection with <a href="https://www.cfvww.org" >CF Vests Worldwide</a>, an organization dedicated to providing life-saving therapy vests to fibrosis patients globally, we gained insights into the challenges faced by CF patients, particularly in regions like Thailand, where access to advanced treatments and medical devices is limited. The conversation highlighted the critical role of early diagnosis and intervention, as well as the quality-of-life challenges many patients endure due to conventional treatments that may not be effective for everyone. Innovative approaches, such as our SORT LNP (lipid nanoparticle) delivery system, present promising alternatives for CF therapy. This system, which allows for RNA encapsulation and administration via dry spray inhalation, could revolutionize treatment by targeting lung cells more effectively, particularly in resource-limited settings. Dr. Olariu underscored the need for psychological support and coordinated care for CF patients, emphasizing that novel therapies like LNP-based gene treatments have the potential to improve treatment efficacy and accessibility, ultimately reducing the lifelong burden of care for patients and their families. </p>,
+ insights: [<p>We invited Max, our CF patient contact, to join Dr. Olariu in discussing the intersection of academic research, clinical application, and patient needs. Through our connection with <a href="https://www.cfvww.org" >CF Vests Worldwide</a>, an organization dedicated to providing life-saving therapy vests to Cystic Fibrosis patients globally, we gained insights into the challenges faced by CF patients, particularly in regions like Thailand, where access to advanced treatments and medical devices is limited. The conversation highlighted the critical role of early diagnosis and intervention, as well as the quality-of-life challenges many patients endure due to conventional treatments that may not be effective for everyone. Innovative approaches, such as our SORT LNP (lipid nanoparticle) delivery system, present promising alternatives for CF therapy. This system, which allows for RNA encapsulation and administration via dry spray inhalation, could revolutionize treatment by targeting lung cells more effectively, particularly in resource-limited settings. Dr. Olariu underscored the need for psychological support and coordinated care for CF patients, emphasizing that novel therapies like LNP-based gene treatments have the potential to improve treatment efficacy and accessibility, ultimately reducing the lifelong burden of care for patients and their families. </p>,
,
- <p>We have considered the extent to which an early diagnosis is always an advantage, as some parents perceive an early diagnosis as an additional burden and would prefer to experience the first years of their child's life without constant medical intervention. Especially when there are cases in which patients only show a clear clinical picture at an advanced age. The psychological burden also lies with the children, who often experience medical trauma because they are involved in such intensive medical care from birth. Additionally, the treatment of fibrosis is very expensive, and the costs are covered by health insurance companies to varying degrees. In some countries, such as the USA, Ukraine or Developing countries, many families cannot afford the necessary treatments. But Dr. Olariu also drew our attention to another problem in the treatment of fibrosis. Infections, especially with bacteria such as Pseudomonas spcc., are difficult to treat and often lead to long hospital stays. Max, our patients’ representative, who knows Dr. Olariu through his treatment, shared his experience with Pseudomonas spcc infections, illustrating the reality of an invisible danger that determines a patient's everyday life. Strict hygiene measures are required to prevent infections, such as wearing face masks in hospital and careful handling of potential sources of infection. The clinics where fibrosis patients are treated work closely with a multidisciplinary team of doctors, psychologists, physiotherapists and nutritionists to ensure that patients receive holistic care. </p>,
+ <p>We have considered the extent to which an early diagnosis is always an advantage, as some parents perceive an early diagnosis as an additional burden and would prefer to experience the first years of their child's life without constant medical intervention. Especially when there are cases in which patients only show a clear clinical picture at an advanced age. The psychological burden also lies with the children, who often experience medical trauma because they are involved in such intensive medical care from birth. Additionally, the treatment of Cystic Fibrosis is very expensive, and the costs are covered by health insurance companies to varying degrees. In some countries, such as the USA, Ukraine or Developing countries, many families cannot afford the necessary treatments. But Dr. Olariu also drew our attention to another problem in the treatment of Cystic Fibrosis. Infections, especially with bacteria such as Pseudomonas spcc., are difficult to treat and often lead to long hospital stays. Max, our patients’ representative, who knows Dr. Olariu through his treatment, shared his experience with Pseudomonas spcc infections, illustrating the reality of an invisible danger that determines a patient's everyday life. Strict hygiene measures are required to prevent infections, such as wearing face masks in hospital and careful handling of potential sources of infection. The clinics where Cystic Fibrosis patients are treated work closely with a multidisciplinary team of doctors, psychologists, physiotherapists and nutritionists to ensure that patients receive holistic care. </p>,
<p>Pros of Early Diagnosis and Treatment</p>,
<ol>
<li>Timely Intervention: Prevents severe organ damage and improves long-term outcomes.</li>
@@ -463,12 +463,12 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<li>Over-medicalization: Continuous focus on treatment can overwhelm families, affecting the quality of early childhood experiences.</li>
</ol>,
],
- implementation: [<p>In summary, our project greatly benefited from the conversation with Dr. Olariu. His insights into the complexities of fibrosis treatment, particularly the significance of early diagnosis, were invaluable and we considered early treatment methods. Max’s personal experiences added a human perspective, illustrating the medical and psychological challenges he faces, including infections with <i>Pseudomonas spp.</i> Dr. Olariu highlightend the importance of a multidisciplinary approach, involving not just medical professionals but also psychologists, physiotherapists, and nutritionists for holistic care. This discussion helped us appreciate the balance between timely interventions and the emotional burden on patients and their families, guiding us to develop a more empathetic understanding of living with fibrosis.</p>],
+ implementation: [<p>In summary, our project greatly benefited from the conversation with Dr. Olariu. His insights into the complexities of Cystic Fibrosis treatment, particularly the significance of early diagnosis, were invaluable and we considered early treatment methods. Max’s personal experiences added a human perspective, illustrating the medical and psychological challenges he faces, including infections with <i>Pseudomonas spp.</i> Dr. Olariu highlightend the importance of a multidisciplinary approach, involving not just medical professionals but also psychologists, physiotherapists, and nutritionists for holistic care. This discussion helped us appreciate the balance between timely interventions and the emotional burden on patients and their families, guiding us to develop a more empathetic understanding of living with Cystic Fibrosis.</p>],
interview: <>
- <QaBox q="Could you please tell us about the journey that parents go through with their CF-sick children from the first visit to diagnosis and treatment?" a="Since 2016, fibrosis (CF) diagnosis has been part of newborn screening. This means that we receive many children right after birth whose screening results were abnormal. These children are then sent to us for further clarification. Not every child with an abnormal screening result is sick, so we perform a sweat test, and about one-third of the children are diagnosed with the disease. The advantage of early diagnosis is that we can intervene and start treatment early to prevent organ damage. However, there are also rare mutations where the course of the disease is difficult to predict." />
- <QaBox q="What are the pros and cons of newborn screening for fibrosis?" a="From a medical point of view, it’s beneficial that we can catch many of these cases early, allowing us to act swiftly. There are even medications for small babies, and early intervention can protect organs, preventing conditions that would require transplants later on. On the downside, because of the wide variety of genetic mutations, some cases we identify may not show significant symptoms until adulthood. This creates a dilemma, as we can’t predict how their condition will progress, but we still start treatments early, which can be stressful for families." />
+ <QaBox q="Could you please tell us about the journey that parents go through with their CF-sick children from the first visit to diagnosis and treatment?" a="Since 2016, Cystic Fibrosis (CF) diagnosis has been part of newborn screening. This means that we receive many children right after birth whose screening results were abnormal. These children are then sent to us for further clarification. Not every child with an abnormal screening result is sick, so we perform a sweat test, and about one-third of the children are diagnosed with the disease. The advantage of early diagnosis is that we can intervene and start treatment early to prevent organ damage. However, there are also rare mutations where the course of the disease is difficult to predict." />
+ <QaBox q="What are the pros and cons of newborn screening for Cystic Fibrosis?" a="From a medical point of view, it’s beneficial that we can catch many of these cases early, allowing us to act swiftly. There are even medications for small babies, and early intervention can protect organs, preventing conditions that would require transplants later on. On the downside, because of the wide variety of genetic mutations, some cases we identify may not show significant symptoms until adulthood. This creates a dilemma, as we can’t predict how their condition will progress, but we still start treatments early, which can be stressful for families." />
<QaBox q="Can you give us an example of how this stress impacts families?" a="Yes, I’ve been caring for a patient from birth who is now five years old and doing very well. However, from the beginning, she had to undergo physiotherapy, regular check-ups, and blood tests, even though she hasn’t shown any major symptoms. Her mother once told me she wasn't sure if she would make the same decision again, as the early intervention caused a lot of stress. She wondered if she might have enjoyed the first year of her child’s life more if things had been more relaxed. Now, at age five, nothing significant has changed in her condition, and they’ve decided against starting modulator therapy for the time being." />
- <QaBox q="How do families typically react when a CF diagnosis is confirmed?" a="For most families, it’s a shock. Cystic fibrosis still has a strong association with being a life-threatening disease, despite the fact that we now have good treatments and many patients can live healthy lives. The diagnosis puts a huge psychological strain on the family, especially when dealing with very young children. The most important factor in managing this, aside from medical treatments, is the support from the medical team. It’s critical to have a team that works well together, not just a single doctor calling all the shots. Families often need much more psychological and nutritional support early on than medical intervention, and this is where having a multidisciplinary team becomes essential." />
+ <QaBox q="How do families typically react when a CF diagnosis is confirmed?" a="For most families, it’s a shock. Cystic Cystic Fibrosis still has a strong association with being a life-threatening disease, despite the fact that we now have good treatments and many patients can live healthy lives. The diagnosis puts a huge psychological strain on the family, especially when dealing with very young children. The most important factor in managing this, aside from medical treatments, is the support from the medical team. It’s critical to have a team that works well together, not just a single doctor calling all the shots. Families often need much more psychological and nutritional support early on than medical intervention, and this is where having a multidisciplinary team becomes essential." />
<QaBox q="What is the process for diagnosing and treating older patients who haven’t been through newborn screening?" a="Older patients who come to us with complaints may not have undergone newborn screening, so they are diagnosed based on their symptoms. These complaints can range from mild to severe and are often non-specific, like chronic cough or failure to thrive. When the cause of these symptoms isn’t immediately clear, we do a sweat test. Once diagnosed, we can start treatment, which often involves working with a psychologist to help the family process the news." />
<QaBox q="How do you support families during the initial shock of diagnosis?" a="When the diagnosis is particularly difficult for families to process, we sometimes have the patients stay in the hospital for up to a week. This gives us time to meet with them daily, answer questions, and provide guidance. During the first consultation, families often fall into a state of shock, and no matter how carefully the doctor explains things, it’s hard for them to absorb all the information. Meeting with them again over the following days helps, and we have specialists in hygiene, physiotherapy, and social counseling on the team to offer holistic support." />
<QaBox q="What happens if a child gets infected with Pseudomonas or another bacterial culture in the lungs?" a="Pseudomonas is one of the most feared infections for CF patients. It’s a common environmental bacterium that is difficult for CF patients to clear from their lungs. Once we detect it, we treat the patient with specific antibiotics, often through intravenous delivery over two weeks in the hospital. After the initial treatment, patients may continue with inhaled antibiotics for several months to prevent further infection. It’s a very intensive process, taking a lot of time and energy, and even though we may get rid of the infection a few times, eventually the germ can become resistant and stay in the body." />
@@ -484,7 +484,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
</>,
pictureurl_aim: "https://static.igem.wiki/teams/5247/photos/hp/interview-olariu.svg",
pictureurl_interview: "https://static.igem.wiki/teams/5247/photos/hp/olario-abbildung1.svg",
- summary: "We interviewed Dr. Cristian-Gabriel Olariu to gain insights into the diagnosis and treatment of fibrosis (CF). He highlighted the importance of early diagnosis through newborn screening, which allows for timely intervention but can also be perceived as a burden by families, especially when symptoms may not manifest until later. Dr. Olariu emphasized the emotional and financial challenges families face, particularly regarding costly treatments and insurance variability. Patient contact Max shared his experiences with infections like Pseudomonas spp., which complicate care and necessitate a multidisciplinary approach involving medical professionals, psychologists, and nutritionists. Overall, the discussion underscored the need to balance medical interventions with the emotional well-being of patients and families, guiding us toward a more compassionate understanding of living with CF.",
+ summary: "We interviewed Dr. Cristian-Gabriel Olariu to gain insights into the diagnosis and treatment of Cystic Fibrosis (CF). He highlighted the importance of early diagnosis through newborn screening, which allows for timely intervention but can also be perceived as a burden by families, especially when symptoms may not manifest until later. Dr. Olariu emphasized the emotional and financial challenges families face, particularly regarding costly treatments and insurance variability. Patient contact Max shared his experiences with infections like Pseudomonas spp., which complicate care and necessitate a multidisciplinary approach involving medical professionals, psychologists, and nutritionists. Overall, the discussion underscored the need to balance medical interventions with the emotional well-being of patients and families, guiding us toward a more compassionate understanding of living with CF.",
months: "may"
},
{
@@ -502,18 +502,18 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteVorname: "Liliana",
type: "meta",
summary: [<p>
- After receiving valuable feedback from both clinical and academic experts, we decided to focus on optimizing Prime Editing strategies for fibrosis treatment. Both experts not only encouraged us in our approach but also provided insightful feedback, which we will integrate into our future project design. Through these discussions, we learned that current treatment strategies are urgently needed in real life but are limited in precision and efficiency.
- Additionally, we gained key insights into lung-specific delivery methods, which inspired us to pursue lung-specific correction of the CFTR gene, a critical aspect of fibrosis therapy.
- At this stage, we are eager to expand our perspectives by seeking input from industry and business professionals, while also striving to increase our local impact. To evaluate this impact, we plan to develop a survey aimed at understanding the interest in gene therapy and the community’s knowledge of fibrosis within our local area. This will help us gauge awareness and ensure our project addresses both scientific and societal needs effectively.</p>,
+ After receiving valuable feedback from both clinical and academic experts, we decided to focus on optimizing Prime Editing strategies for Cystic Fibrosis treatment. Both experts not only encouraged us in our approach but also provided insightful feedback, which we will integrate into our future project design. Through these discussions, we learned that current treatment strategies are urgently needed in real life but are limited in precision and efficiency.
+ Additionally, we gained key insights into lung-specific delivery methods, which inspired us to pursue lung-specific correction of the CFTR gene, a critical aspect of Cystic Fibrosis therapy.
+ At this stage, we are eager to expand our perspectives by seeking input from industry and business professionals, while also striving to increase our local impact. To evaluate this impact, we plan to develop a survey aimed at understanding the interest in gene therapy and the community’s knowledge of Cystic Fibrosis within our local area. This will help us gauge awareness and ensure our project addresses both scientific and societal needs effectively.</p>,
<ul>
<li>
<b>Expert Feedback Integration:</b> Refined the project focus on optimizing Prime Editing strategies and lung-specific gene delivery based on clinical and academic insights.
</li>
<li>
- <b>Focus on Lung-Specific Correction:</b> Shifted toward lung-specific CFTR gene correction for fibrosis treatment.
+ <b>Focus on Lung-Specific Correction:</b> Shifted toward lung-specific CFTR gene correction for Cystic Fibrosis treatment.
</li>
<li>
- <b>Community Engagement Plan:</b> Initiated plans for a local survey to assess awareness of fibrosis and interest in gene therapy, aiming to increase local impact.
+ <b>Community Engagement Plan:</b> Initiated plans for a local survey to assess awareness of Cystic Fibrosis and interest in gene therapy, aiming to increase local impact.
</li>
</ul>
],
@@ -526,7 +526,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
affiliation: "Independent",
pictureurl: pics['westhoff'],
tag: "Medical Professional",
- heading: "Interview with a specialized physiotherapist regarding breathing therapy for fibrosis patients",
+ heading: "Interview with a specialized physiotherapist regarding breathing therapy for Cystic Fibrosis patients",
interviewtabid: "westhoffinv",
cardtext: "",
language: "de",
@@ -535,9 +535,9 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quote: "The more we know, the more opportunities we have.",
aimofcontact: "The objective of the contact was to gain in-depth insights into the treatment and care of children with CF. The therapist's expertise was intended to help develop a better understanding of the challenges and necessary measures in the treatment of this chronic disease. In addition, the aim was to ascertain how the therapy is implemented in everyday life and which specific approaches and methods are particularly effective.",
insights: "The interview yielded valuable insights into the regular implementation of the therapy, the use of aids and the adaptation of exercises to the individual needs of the patients. It was notable that the therapy has improved over the last years, considerably thanks to better medication and adapted exercises, with a concomitant increase in life expectancy for children affected by CF. Of particular interest was the emphasis on the importance of sport and exercise, which should not only be therapeutically effective, but also increase quality of life. ",
- implementation: "The following statement by Katrin Westhoff had a particular impact on our project: ‘The more we know, the more options we have’. We learnt from the interview that the current medication is already helping many patients very well, but that there is still great potential for improvement. Successful gene therapy would significantly improve the quality of life of CF patients. We implemented the findings from this interview in our participation in MukoMove - we also actively took part in fibrosis awareness month and learnt even more about the importance of physiotherapy.",
+ implementation: "The following statement by Katrin Westhoff had a particular impact on our project: ‘The more we know, the more options we have’. We learnt from the interview that the current medication is already helping many patients very well, but that there is still great potential for improvement. Successful gene therapy would significantly improve the quality of life of CF patients. We implemented the findings from this interview in our participation in MukoMove - we also actively took part in Cystic Fibrosis awareness month and learnt even more about the importance of physiotherapy.",
pictureurl_interview: "https://static.igem.wiki/teams/5247/photos/hp/katrin-westhoff-zoom.webp",
- summary: [<p>The objective of our discussion with a therapist was to gain a comprehensive understanding of the treatment and care of children with fibrosis. The interview provided invaluable insights into the therapy's implementation, highlighting the significant advancements in medication and tailored exercises that have led to improved patient outcomes and increased life expectancy. A key takeaway was the emphasis on the role of sports and exercise, not just for therapeutic efficacy but also for enhancing overall quality of life. It let to our participation in the CF awarness month and the outreach project mukomove {/* [Link mukomove] */}</p>],
+ summary: [<p>The objective of our discussion with a therapist was to gain a comprehensive understanding of the treatment and care of children with Cystic Fibrosis. The interview provided invaluable insights into the therapy's implementation, highlighting the significant advancements in medication and tailored exercises that have led to improved patient outcomes and increased life expectancy. A key takeaway was the emphasis on the role of sports and exercise, not just for therapeutic efficacy but also for enhancing overall quality of life. It let to our participation in the CF awarness month and the outreach project mukomove {/* [Link mukomove] */}</p>],
months: "May",
interview:<>
<QaBox q="From what age do the patients come to you? How long do they stay? How many patients do you treat?" a="The patients come to us at a very early age. A definite diagnosis is made after 6 weeks at the latest. Once diagnosed, the whole family is genetically tested, and children are sent for physiotherapy, often starting in the hospital. Currently, we have 8 children with CF in our practice, which is relatively small compared to other diseases. We have slightly more CF patients because we specialize in it."/>
@@ -611,7 +611,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<b>Expansion of Biosafety Standards:</b> Extending biosafety protocols to mimic preclinical trial conditions reinforced the commitment to safe, ethical research practices and laid the groundwork for clinical relevance, demonstrating responsibility toward future patients.
</li>
<li>
- <b>Local and International Community Engagement:</b> Efforts to raise awareness at both local and global levels ensured that the project was not only scientifically sound but also socially responsible, with a focus on educating and involving the public in the conversation around fibrosis and gene therapy.
+ <b>Local and International Community Engagement:</b> Efforts to raise awareness at both local and global levels ensured that the project was not only scientifically sound but also socially responsible, with a focus on educating and involving the public in the conversation around Cystic Fibrosis and gene therapy.
</li>
</ul>
],
@@ -631,7 +631,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteNachname: "Susat, Teammember",
quoteVorname: "Kathleen",
quote: "It was amazing to see how movement can bring people together. I had a great time.",
- summary: [<p>MUKOmove{/* [LINK unten mukomove] */} is a sports initiative by Mukoviszidose e.V., the German Cystic Fibrosis Association, to raise awareness and funds for fibrosis. Team iGEM Bielefeld participated from May 8th to May 12th, promoting the event at their university and city, encouraging others to join and collect sport hours. They organized a team event at their university, involving sports games to promote community engagement. Their efforts helped surpass their goal of 240 hours, with the team achieving 358 sport hours, while the entire event gathered over 36,000 sport hours. The initiative successfully raised awareness about fibrosis and promoted physical activity as a means of community building.</p>],
+ summary: [<p>MUKOmove{/* [LINK unten mukomove] */} is a sports initiative by Mukoviszidose e.V., the German Cystic Fibrosis Association, to raise awareness and funds for Cystic Fibrosis. Team iGEM Bielefeld participated from May 8th to May 12th, promoting the event at their university and city, encouraging others to join and collect sport hours. They organized a team event at their university, involving sports games to promote community engagement. Their efforts helped surpass their goal of 240 hours, with the team achieving 358 sport hours, while the entire event gathered over 36,000 sport hours. The initiative successfully raised awareness about Cystic Fibrosis and promoted physical activity as a means of community building.</p>],
months: "May"
},
{
@@ -676,7 +676,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
implementation: [<p>The inputs given by Mattijs directly impacted our design choices for multiple parts of the project. For the pegRNA design, we decided to use the same 3’ motif as Mattijs had used and also, like he suggested, checked our spacer candidates for predicted cleavage efficiency. Also we used HEK cells for screening our pegRNAs. We looked further into PE systems that influence cellular mismatch repair (such as PE4) and tried to include these into our design.</p>],
interview: <>
<QaBox q="We have prepared some questions for you. The first question is: You mentioned that it was quite challenging to target the F508 delta mutation. Could you provide more detailed reasons for why this is the case or explain why this mutation is particularly difficult to target compared to others?"
- a="Yes, that's the million-dollar question. First of all, let me clarify: our group has never directly worked on that mutation because we immediately focused on the drug-refractory mutations, such as nonsense mutations where the protein is not formed, indel mutations, or severe missense mutations that do not respond to modulator therapies. Of course, we know several groups in the field who either work on gene editing or focus on fibrosis (CF). We've heard from some of them who attempted to target the F508 delta mutation. For example, some collaborators really tried to design different guides but were unable to find anything above the detection limit.
+ a="Yes, that's the million-dollar question. First of all, let me clarify: our group has never directly worked on that mutation because we immediately focused on the drug-refractory mutations, such as nonsense mutations where the protein is not formed, indel mutations, or severe missense mutations that do not respond to modulator therapies. Of course, we know several groups in the field who either work on gene editing or focus on Cystic Fibrosis (CF). We've heard from some of them who attempted to target the F508 delta mutation. For example, some collaborators really tried to design different guides but were unable to find anything above the detection limit.
F508del is probably one of the most logical mutations to try to correct, not just for CF but for the entire gene-editing field. If you look at the frequencies of mutations that cause genetic diseases, the F508 delta mutation is by far the most common deletion mutation causing a severe disease. This is because CF, along with sickle cell disease, is one of the most common deadly inherited diseases, and it's overrepresented within CF. So, it makes sense that they would have been trying to target it from the beginning.
Interestingly, if you read the Prime Editing paper by Anzalone, F508 delta is mentioned in the introduction in connection with CF. So, it's somewhat surprising that after all this time—it's been almost five years now—they haven't published or released anything on F508 delta.
However, last weekend, there was an online seminar where David Liu gave a talk, and he showed some unpublished data indicating that they managed to achieve quite good Prime Editing efficiency on F508 delta. It's worth noting that David Liu rarely presents unpublished data unless the publication is either accepted or very close to acceptance. So, we all kind of expect that the paper will be published soon, perhaps within the next week or at least within a month. From what I saw, it appears they leveraged many of the approaches available today to enhance Prime Editing.
@@ -754,21 +754,21 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
cardtext: "",
quoteNachname: "Köhler, Teammember",
quoteVorname: "Vera",
- quote: "I’m excited about our partnership with Mukoviszidose e.V. Deutschland to raise awareness in our community. Educating people about fibrosis and gene therapy is essential, and I believe our scientific advancements will have a broader impact beyond just this condition.",
+ quote: "I’m excited about our partnership with Mukoviszidose e.V. Deutschland to raise awareness in our community. Educating people about Cystic Fibrosis and gene therapy is essential, and I believe our scientific advancements will have a broader impact beyond just this condition.",
type: "meta",
summary: [<p>
- During the early stages of our project, we discovered through our survey that while many participants were open to trying gene therapies, they lacked adequate knowledge about them. Additionally, most respondents were unfamiliar with fibrosis, highlighting the need for greater awareness. Driven by a desire to educate the people of Bielefeld, we collaborated with Mukoviszidose e.V. Deutschland to support the Muko Move campaign, a successful initiative aimed at raising awareness about fibrosis. On the scientific front, we elevated our project to a new level. With valuable feedback from Mattijs Bulcaen at the University of Leuven, we incorporated a novel RNA structural element into our Prime Editing complex, significantly improving its efficiency. After successfully optimizing the pegRNA, we moved forward with enhancing the Prime Editing protein complex. Our goal is to make Prime Editing not only safer but also easier to apply, so that our research can benefit more than just fibrosis patients, ultimately broadening the impact of our work.
+ During the early stages of our project, we discovered through our survey that while many participants were open to trying gene therapies, they lacked adequate knowledge about them. Additionally, most respondents were unfamiliar with Cystic Fibrosis, highlighting the need for greater awareness. Driven by a desire to educate the people of Bielefeld, we collaborated with Mukoviszidose e.V. Deutschland to support the Muko Move campaign, a successful initiative aimed at raising awareness about Cystic Fibrosis. On the scientific front, we elevated our project to a new level. With valuable feedback from Mattijs Bulcaen at the University of Leuven, we incorporated a novel RNA structural element into our Prime Editing complex, significantly improving its efficiency. After successfully optimizing the pegRNA, we moved forward with enhancing the Prime Editing protein complex. Our goal is to make Prime Editing not only safer but also easier to apply, so that our research can benefit more than just Cystic Fibrosis patients, ultimately broadening the impact of our work.
</p>,
<ul>
<li>
- <b>Survey Insights:</b> Many participants were motivated to try gene therapies but lacked knowledge about them and fibrosis, highlighting the need for better public education.
+ <b>Survey Insights:</b> Many participants were motivated to try gene therapies but lacked knowledge about them and Cystic Fibrosis, highlighting the need for better public education.
</li>
<li>
- <b>Awareness Campaign:</b> Partnered with Mukoviszidose e.V. Deutschland to support the Muko Move campaign, raising awareness about fibrosis in the local community.
+ <b>Awareness Campaign:</b> Partnered with Mukoviszidose e.V. Deutschland to support the Muko Move campaign, raising awareness about Cystic Fibrosis in the local community.
</li>
<li>
- <b>Scientific Advancements:</b> Improved the efficiency of Prime Editing by incorporating a novel RNA structural element, with further efforts to optimize the Prime Editing protein complex for broader applicability beyond fibrosis.
+ <b>Scientific Advancements:</b> Improved the efficiency of Prime Editing by incorporating a novel RNA structural element, with further efforts to optimize the Prime Editing protein complex for broader applicability beyond Cystic Fibrosis.
</li>
</ul>],
months: "june"
@@ -785,7 +785,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteNachname: "Sahami Moghaddam, Teammember",
quoteVorname: "Asal",
quote: "I had a great time educating the kids about gene therapy and CF in a playful way, and there were some very interesting conversations with the parents.",
- summary: [<p>"Der Teuto ruft!"{/* [LInk zu Teuto unten] */} is a community event in Bielefeld where iGEM Bielefeld participated to raise awareness about fibrosis (CF) and gene therapy. They engaged children through interactive experiments, such as creating lung models and simulating mucus to help them understand the challenges faced by CF patients. Adults were informed about their gene therapy project and had meaningful discussions about the implications of CF treatment. Collaborating with other institutions like the life science student initiative btS, the team expanded their outreach. Despite changeable weather, the event was a success in educating the public and improving science communication.</p>],
+ summary: [<p>"Der Teuto ruft!"{/* [LInk zu Teuto unten] */} is a community event in Bielefeld where iGEM Bielefeld participated to raise awareness about Cystic Fibrosis (CF) and gene therapy. They engaged children through interactive experiments, such as creating lung models and simulating mucus to help them understand the challenges faced by CF patients. Adults were informed about their gene therapy project and had meaningful discussions about the implications of CF treatment. Collaborating with other institutions like the life science student initiative btS, the team expanded their outreach. Despite changeable weather, the event was a success in educating the public and improving science communication.</p>],
months: "June"
},
{
@@ -814,14 +814,14 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
dealing with CF and we were able to discuss the situation for patients in Germany in comparison to other countries better in later <HPLinktoOtherHPTab tab="joshua" text="interviews" />. </p>],
interview: <>
<QaBox q="Can you tell us a bit about your family? How old are your children and yourselves?" a="I’m 37, my husband is 44, and our daughter is six, turning seven soon. We also have a son who’s about a year and a half." />
- <QaBox q="Does your son also have fibrosis?" a="No, he doesn’t." />
- <QaBox q="When was your daughter diagnosed with fibrosis?" a="Right after birth. She was transferred to a bigger hospital due to an intestinal blockage and had surgery. After about two to three weeks in intensive care, the fibrosis diagnosis came through newborn screening. At that time, the results took longer to process than they do now." />
+ <QaBox q="Does your son also have Cystic Fibrosis?" a="No, he doesn’t." />
+ <QaBox q="When was your daughter diagnosed with Cystic Fibrosis?" a="Right after birth. She was transferred to a bigger hospital due to an intestinal blockage and had surgery. After about two to three weeks in intensive care, the Cystic Fibrosis diagnosis came through newborn screening. At that time, the results took longer to process than they do now." />
<QaBox q="That intestinal issue can happen for many reasons, right?" a="Yes, it was all new to us. The beginning was difficult, but things have gotten better since then, and we’re very grateful." />
<QaBox q="How did you feel when you first heard the diagnosis?" a="It felt like our world was falling apart. I still remember the moment—it was like being in a movie. We were told in a separate room, and it felt overwhelming. One doctor even suggested we go home to think about it in peace, but all I could think about was returning to my child. It was a lot to take in, especially thinking about how we’d tell our family." />
<QaBox q="That sounds incredibly hard. How did you handle it as time passed?" a="It was tough, but we were fortunate to have a doctor who really understood what we were going through, as he had a disabled child himself. He never scared us unnecessarily and guided us step by step, which made a big difference. We know many families who live in constant fear, but since those first months, we’ve learned to manage the situation without being overwhelmed by fear." />
<QaBox q="Did any particular support help your family adjust to the diagnosis?" a="Yes, the rehab program we attended was a huge help. It was a family-oriented program, so my husband could be there too, which was important since I manage most things day-to-day. It really helped our daughter realize she’s not alone—she met other kids with similar conditions, which was a huge comfort." />
<QaBox q="How did you explain the illness to your daughter?" a="We try to give it as little attention as possible in daily life. She’s been inhaling medication since she was eight weeks old, and it’s just part of her routine now. Thankfully, she doesn’t fight it or question it much, and her school and kindergarten haven’t made a big deal of it either, which is what we wanted." />
- <QaBox q="Does she ever ask about her illness compared to her younger brother, who doesn’t have fibrosis?" a="She does sometimes ask why she’s sick and he’s not, but she’s not upset by it. We’ve made sure not to give her any special treatment because of her illness, which can be hard at times, but we want her to understand that her illness doesn’t define her." />
+ <QaBox q="Does she ever ask about her illness compared to her younger brother, who doesn’t have Cystic Fibrosis?" a="She does sometimes ask why she’s sick and he’s not, but she’s not upset by it. We’ve made sure not to give her any special treatment because of her illness, which can be hard at times, but we want her to understand that her illness doesn’t define her." />
<QaBox q="That sounds like a good balance. What about medications—did she start on any special treatments?" a="Yes, she started on Orkambi at around three years old but had to stop briefly due to high liver values. Now she’s on Kaftrio, which she started shortly before her sixth birthday, and it’s been going well." />
<QaBox q="Did you face any issues with the health insurance for covering these medications?" a="Fortunately, no. We have statutory health insurance, and they’ve covered everything without any issues. We’ve heard it can be more complicated for those with private insurance." />
<QaBox q="Have you ever had difficulties with access to medication?" a="Yes, there have been times when we’ve had to wait a few days for certain medications, like Kreon or antibiotics, especially in the winter. But we always plan ahead and keep a buffer, so we’ve never been without what we need." />
@@ -873,13 +873,13 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
affiliation: "University Potsdam",
pictureurl: pics['nicole'],
tag: "Academia",
- heading: "Discussion on how health insurance companies manage fibrosis patients and gene therapy treatments",
+ heading: "Discussion on how health insurance companies manage Cystic Fibrosis patients and gene therapy treatments",
interviewtabid: "nicole",
cardtext: "",
language: "de",
quote: "Public health insurance operates under an economic efficiency principle, meaning the most cost-effective treatments are preferred. But if gene therapies become the only treatment option for certain conditions, they will likely have to be included in the coverage, which could be a challenge for the system.",
- aimofcontact: "The main objective of the contact was to learn from the discussion on issues related to fibrosis (CF), gene therapy, health insurance processes and regulatory pathways. In particular, we wanted to understand the real-world challenges and technical aspects of gene editing, especially prime editing, as well as the complexities of approval and reimbursement of gene therapies for CF patients.",
- insights: "The regulatory approval process, particularly by the European Medicines Agency (EMA) for advanced medical devices, has highlighted the bureaucratic hurdles that gene therapies must overcome. We learned that such therapies for fibrosis have to navigate complex European and German regulatory systems. The discussion on the AMNOG process was crucial. We learnt that the additional benefit of a therapy is assessed for reimbursement by the statutory health insurance funds. We implemented this insight in our project by considering the long-term regulatory and economic effects as important milestones for therapy development. We also gained insight into how public and private health insurers may differ in their reimbursement of such therapies. Public insurers have stricter guidelines, while private insurers can be more flexible, but both require strict justification, especially for rare diseases such as fibrosis. Information on newborn screening and genetic counselling covered by public health insurance was crucial to understanding how preventive measures for CF are managed. This underlines the importance of early intervention and diagnosis in our project. Atypical forms of CF, where health insurance companies do not cover treatment due to non-standardised test results, were identified as a key problem. This helped us to recognise the need for more adaptable insurance policies and clearer pathways for the treatment of atypical cases in our project plans. The debate about whether healthcare systems can afford the high costs of gene therapies highlighted an important issue in the current medical landscape. We have incorporated this insight into our project by discussing possible cost-effective alternatives and the need for thorough cost-benefit analysis in the development of treatments.",
+ aimofcontact: "The main objective of the contact was to learn from the discussion on issues related to Cystic Fibrosis (CF), gene therapy, health insurance processes and regulatory pathways. In particular, we wanted to understand the real-world challenges and technical aspects of gene editing, especially prime editing, as well as the complexities of approval and reimbursement of gene therapies for CF patients.",
+ insights: "The regulatory approval process, particularly by the European Medicines Agency (EMA) for advanced medical devices, has highlighted the bureaucratic hurdles that gene therapies must overcome. We learned that such therapies for Cystic Fibrosis have to navigate complex European and German regulatory systems. The discussion on the AMNOG process was crucial. We learnt that the additional benefit of a therapy is assessed for reimbursement by the statutory health insurance funds. We implemented this insight in our project by considering the long-term regulatory and economic effects as important milestones for therapy development. We also gained insight into how public and private health insurers may differ in their reimbursement of such therapies. Public insurers have stricter guidelines, while private insurers can be more flexible, but both require strict justification, especially for rare diseases such as Cystic Fibrosis. Information on newborn screening and genetic counselling covered by public health insurance was crucial to understanding how preventive measures for CF are managed. This underlines the importance of early intervention and diagnosis in our project. Atypical forms of CF, where health insurance companies do not cover treatment due to non-standardised test results, were identified as a key problem. This helped us to recognise the need for more adaptable insurance policies and clearer pathways for the treatment of atypical cases in our project plans. The debate about whether healthcare systems can afford the high costs of gene therapies highlighted an important issue in the current medical landscape. We have incorporated this insight into our project by discussing possible cost-effective alternatives and the need for thorough cost-benefit analysis in the development of treatments.",
implementation: [<p>After the interview, we further tailored our project to focus on a simple delivery method to reduce the therapeutic effort. To gain an overview of the regulatory requirements and to better deliver the project, one of our team members attended a <HPLinktoOtherHPTab tab="gxpcourse" text="GxP course"/> to ensure we met all the necessary standards. To deepen our knowledge of entrepreneurship, we conducted further interviews with start-ups and <HPgoToPageAndScroll id="Further Engagement3H" path="\human-practices" text="industrial companies" />, which gave us important insights into practical implementation. These steps ensure that our project is not only based on scientific research, but also takes into account the practical, regulatory and social aspects that are crucial to bringing new CF therapies to the market. We are currently developing strategies to successfully implement our ideas and the project in the future.</p>],
interview: <>
<QaBox q="To start with this interview. Do you have any questions about this project?"
@@ -888,9 +888,9 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
a="Are you writing a formal paper?" />
<QaBox q="We’re not writing a formal text-based paper, but everything will be available on a website. We will document most of our work on the website, with sub-pages detailing lab work, interviews, and research."
a="What exactly is Prime Editing, and how does it differ from altering the germline? Where in the genome does this therapy act?" />
- <QaBox q="Our current plan is to deliver the therapy via a lipid nanoparticle system, which will be inhaled and go into the lungs. While fibrosis (CF) affects all mucus membranes, the lungs are the most critical area, so we’re focusing on that. The therapy will only target surface cells in the lungs, not the basal cells responsible for producing new lung cells."
+ <QaBox q="Our current plan is to deliver the therapy via a lipid nanoparticle system, which will be inhaled and go into the lungs. While Cystic Fibrosis (CF) affects all mucus membranes, the lungs are the most critical area, so we’re focusing on that. The therapy will only target surface cells in the lungs, not the basal cells responsible for producing new lung cells."
a="Thank you for giving me insights into your project." />
- <QaBox q="Do you know how fibrosis (CF) approval works in terms of health insurance and regulatory processes?"
+ <QaBox q="Do you know how Cystic Fibrosis (CF) approval works in terms of health insurance and regulatory processes?"
a="The approval process for gene therapies is primarily done through the EMA (European Medicines Agency) under specific EU regulations for Advanced Medical Products, including gene therapies. There is also a national approval process in Germany for individualized treatments, but large-scale therapies must go through the EU process." />
<QaBox q="Can you share more about the approval and reimbursement processes for CF treatment?"
a="The approval process is separate from reimbursement by public health insurance. CF is considered a rare disease if it affects fewer than five out of 10,000 people, and treatments for rare diseases often face special reimbursement challenges. If more than five out of 10,000 people are affected, the disease is relatively common, and approval and reimbursement go through a different procedure known as the AMNOG process. For more common diseases, an additional benefit (Zusatznutzen) must be demonstrated during the approval process." />
@@ -921,9 +921,9 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<QaBox q="Do biologists or medical professionals develop gene therapies?"
a="In terms of development, it’s mainly biologists and biotechnologists. Medical professionals get involved primarily in clinical trials. Some doctors do research, but they’re often needed in hospitals, so hands-on development is mostly handled by molecular biologists or biotechnologists." />
<QaBox q="Does research in genome medicine and gene therapies come from biology, medicine, or both?"
- a="It’s mainly interdisciplinary. A lot of funding comes from industry, like BioNTech, or foundations like Mukoviszidose e.V., which funds research on fibrosis. But in terms of practical research, it’s usually biologists or biotechnologists. Without industry support, research can struggle due to a lack of funding, so having backing is essential." />
+ a="It’s mainly interdisciplinary. A lot of funding comes from industry, like BioNTech, or foundations like Mukoviszidose e.V., which funds research on Cystic Fibrosis. But in terms of practical research, it’s usually biologists or biotechnologists. Without industry support, research can struggle due to a lack of funding, so having backing is essential." />
</>,
- summary: "Our discussion addressed the complexities of fibrosis (CF) treatments, focusing on gene therapy and health insurance processes. We learned about the regulatory challenges gene therapies face, particularly regarding the European Medicines Agency (EMA) and the AMNOG process for reimbursement assessments. Public insurers impose stricter guidelines than private insurers, emphasizing the importance of early intervention in CF and the need for adaptable policies for atypical cases. We recognized the high costs associated with gene therapies and incorporated cost-benefit analysis into our project planning. Following the interview, we refined our approach to include straightforward delivery methods and attended a GxP course for regulatory compliance. Engaging with start-ups further informed our practical implementation strategies, ensuring our project aligns with both scientific and regulatory needs.",
+ summary: "Our discussion addressed the complexities of Cystic Fibrosis (CF) treatments, focusing on gene therapy and health insurance processes. We learned about the regulatory challenges gene therapies face, particularly regarding the European Medicines Agency (EMA) and the AMNOG process for reimbursement assessments. Public insurers impose stricter guidelines than private insurers, emphasizing the importance of early intervention in CF and the need for adaptable policies for atypical cases. We recognized the high costs associated with gene therapies and incorporated cost-benefit analysis into our project planning. Following the interview, we refined our approach to include straightforward delivery methods and attended a GxP course for regulatory compliance. Engaging with start-ups further informed our practical implementation strategies, ensuring our project aligns with both scientific and regulatory needs.",
months: "june",
pictureurl_interview: "https://static.igem.wiki/teams/5247/photos/hp/zoom-nicole.webp",
},
@@ -965,7 +965,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
language: "de",
quote: "Children are the world's best “mucus hidersâ€.",
aimofcontact: [<a>During the last interview with <HPLinktoOtherHPTab tab="westhoffinv" text="Katrin Westhoff" />, she invited us to join a few physiotherapy sessions – not just as spectators but as participants. We gladly accepted and visited her in her practice. Over a few hours, we took part in four sessions with different children – not all of them CF patients. </a>],
- insights: "During the sessions, we could ask Katrin as well as the respective parents and children questions. We learned that breathing therapy is also useful for other illnesses and that you can easily do some of the exercises yourself. Despite having fibrosis, the children were better at the breathing exercises than we and Katrin were! The sessions take 30 to 60 minutes and include both manual therapy and playful elements to help engage the children. Most older children range from mildly unhappy to enthusiastic, but babies often cry during the treatments as it feels uncomfortable. This is often hard on the parents even though the treatment brings good results. A lot of children tend to hide that they have mucus sitting in their lungs by suppressing coughs. Especially with young children, it is important to stay on top of it and do regular breathing therapy even if it seems like it is currently not necessary. We also learned about the various informational material aimed at children to help explain therapies and symptoms to them and what accessories for breathing therapy there are. For example, a flutter is to train breathing out forcefully by breathing against a small weight and a binder can be worn at night to promote deep breathing. ",
+ insights: "During the sessions, we could ask Katrin as well as the respective parents and children questions. We learned that breathing therapy is also useful for other illnesses and that you can easily do some of the exercises yourself. Despite having Cystic Fibrosis, the children were better at the breathing exercises than we and Katrin were! The sessions take 30 to 60 minutes and include both manual therapy and playful elements to help engage the children. Most older children range from mildly unhappy to enthusiastic, but babies often cry during the treatments as it feels uncomfortable. This is often hard on the parents even though the treatment brings good results. A lot of children tend to hide that they have mucus sitting in their lungs by suppressing coughs. Especially with young children, it is important to stay on top of it and do regular breathing therapy even if it seems like it is currently not necessary. We also learned about the various informational material aimed at children to help explain therapies and symptoms to them and what accessories for breathing therapy there are. For example, a flutter is to train breathing out forcefully by breathing against a small weight and a binder can be worn at night to promote deep breathing. ",
implementation: "The most important thing was that both Katrin and the parents agreed that the children were able to inhale at an early age and that there were generally no physical problems with inhalation in general. This reinforced our decision to work towards delivery by inhalation. It was very interesting to see the different ways children deal with their exercises and hear about the progress they made. ",
text: [<ol>
<li>
@@ -985,7 +985,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<p>Chrissi takes modulators and will soon take a trip to a water park with some friends. Katrin teaches us that when the children do not breathe out properly, air stays in the lungs and causes hyperinflation – with which it is actually harder to float in water! After the manual drainage, Katrin gets all of us glasses with water and dish soap and straws. Blowing bubbles is a playful way to train how to properly breathe out by either trying to blow bubbles as long as possible or trying to make an existing bubble as big as possible!</p>
</li>
</ol>,],
- summary: "In the visit with Katrin Westhoff, we participated in physiotherapy sessions for children, including those with fibrosis (CF). We observed that breathing therapy is beneficial for various illnesses and learned techniques that can be practiced at home. Sessions last 30 to 60 minutes, combining manual therapy with playful elements. While older children engaged well, infants often found the exercises uncomfortable. Importantly, both Katrin and parents noted that children could inhale without issues from an early age, reinforcing our focus on inhalation delivery methods for therapies.",
+ summary: "In the visit with Katrin Westhoff, we participated in physiotherapy sessions for children, including those with Cystic Fibrosis (CF). We observed that breathing therapy is beneficial for various illnesses and learned techniques that can be practiced at home. Sessions last 30 to 60 minutes, combining manual therapy with playful elements. While older children engaged well, infants often found the exercises uncomfortable. Importantly, both Katrin and parents noted that children could inhale without issues from an early age, reinforcing our focus on inhalation delivery methods for therapies.",
pictureurl_interview:"https://static.igem.wiki/teams/5247/photos/hp/besuch-westhoff/untitled-design.png",
months: "june"
},
@@ -1100,18 +1100,18 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
of care depends on the healthcare system, as we already touched on during the interview with <HPLinktoOtherHPTab tab="nicole" text="Nicole Friedlein" />,. On the parenting level, Joshua brought in many perspectives contrary to what we previously heard. In the interview with <HPLinktoOtherHPTab tab="maxfirst" text="Max" />,, we learned he vehemently avoids ponding water while Joshua’s daughter is allowed to roam around with no such restrictions. Neither have chronic infections.</p>],
implementation: [<p>The interview with Josh made us realize we too needed to look at the reason why we chose F508del. Did we, too, fall for bias?
Despite a change of target not being feasible anymore, we looked into it and traced back our steps that led to our decision. We did not find as much
- information about other mutations when first researching fibrosis, especially in the context of prime editing. Mattijs Bulceans's paper on
- targeting the mutations L227R and N1303K <TabScrollLink tab="joshua" scrollId="desc-1" num="1" /> was one of few papers. After explicitly searching for fibrosis records for specific countries and
+ information about other mutations when first researching Cystic Fibrosis, especially in the context of prime editing. Mattijs Bulceans's paper on
+ targeting the mutations L227R and N1303K <TabScrollLink tab="joshua" scrollId="desc-1" num="1" /> was one of few papers. After explicitly searching for Cystic Fibrosis records for specific countries and
regions, we uncovered a moderate number of papers examining CF in Asia and other regions we previously did not know much about. The very first article
supported Joshua's hypotheses and painted a sad picture: Among other things, it describes the case of a four-month-old boy who was diagnosed with
- fibrosis. Nothing unusual in itself, but the circumstances are depressing. Two of the three siblings born before him died within months of birth and had
- previously presented with symptoms of fibrosis. He was the first to be diagnosed. A sweat test aimed at fibrosis was not available at the
+ Cystic Fibrosis. Nothing unusual in itself, but the circumstances are depressing. Two of the three siblings born before him died within months of birth and had
+ previously presented with symptoms of Cystic Fibrosis. He was the first to be diagnosed. A sweat test aimed at Cystic Fibrosis was not available at the
hospital, so one was improvised. Later on, a genetic test revealed the presence of 508del. <TabScrollLink tab="joshua" scrollId="desc-2" num="2" /> We found ourselves and our lack of knowledge in good
company as we found papers as new as from 2020 (14 years after the previously mentioned paper) containing statements such as “recent reports suggest
- that CF does occur in Asia†<TabScrollLink tab="joshua" scrollId="desc-3" num="3" />. Fortunately, there is a rising number of fibrosis experts for Asia and other previously overlooked regions
+ that CF does occur in Asia†<TabScrollLink tab="joshua" scrollId="desc-3" num="3" />. Fortunately, there is a rising number of Cystic Fibrosis experts for Asia and other previously overlooked regions
such as Africa. <TabScrollLink tab="joshua" scrollId="desc-4" num="4" /> We chose to not only look at the scientific data but also into anecdotal evidence. To find the latter, we searched official
and private websites and chatrooms for information and experiences of patients. In the end, we found narratives from most ethnic backgrounds
- about being dismissed and often misdiagnosed. Of course, this is not an occurrence unique to fibrosis. Our conclusion is that yes,
+ about being dismissed and often misdiagnosed. Of course, this is not an occurrence unique to Cystic Fibrosis. Our conclusion is that yes,
we did fall for bias. But regardless of ethnicity, 508del occurs and is overall the most prevalent mutation as was confirmed in our interview
with CF expert Sriram .... This experience was uncomfortable as we felt the pressure to be thorough and deliver a perfect project. What would
have been more devastating than realizing we made a wrong choice at the very core? We made the conscious decision to invest our resources into
@@ -1135,11 +1135,11 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
cardtext: "",
quoteNachname: "Wiesner, Teammember",
quoteVorname: "Lisa",
- quote: "I’m thrilled with the progress we’re making in optimizing our delivery strategies. The collaboration with industry experts has opened our eyes to new possibilities, and I believe our work will significantly impact fibrosis treatment.",
+ quote: "I’m thrilled with the progress we’re making in optimizing our delivery strategies. The collaboration with industry experts has opened our eyes to new possibilities, and I believe our work will significantly impact Cystic Fibrosis treatment.",
type: "meta",
summary: [
<p>
- After successfully conducting multiple experiments, we delved deeper into delivery strategies for our Prime Editing system. Our stakeholders provided crucial feedback that helped us overcome experimental failures and improve our methodology. Marco Raduvic offered key tips for effectively utilizing lipid nanoparticles, allowing us to successfully transfect them into HEK and CFTR-specific cells. Additionally, we replicated the proof of concept established by David Liu et al. in 2019, gaining valuable insights into how a Prime Editing system should function. During this process, we identified a gap in measuring the efficiency of our Prime Editing constructs. To address this, we developed a fluorescence-based system with high selectivity, allowing us to perform precise efficiency analyses. Beyond lab work, we connected with various industry members, not only to secure sponsorships but to emphasize collaboration and exchange with leading market players. Through this network, we not only received feedback on the importance of physiological therapies but also gained insights into the insurance-related challenges associated with a fundamental disease like fibrosis. This motivated us to take our project to the next level. Our collaboration with the medical faculty of our university, along with national feedback, enabled us to integrate new and advanced validation methods. In the next phase of our studies, we decided to optimize lipid nanoparticles (LNPs) to make them suitable for a novel, lung-specific delivery strategy. Additionally, we prepared to explore enzyme engineering by engaging with stakeholders experienced in nickase development and yeast experimental design, further refining our Prime Editing system.
+ After successfully conducting multiple experiments, we delved deeper into delivery strategies for our Prime Editing system. Our stakeholders provided crucial feedback that helped us overcome experimental failures and improve our methodology. Marco Raduvic offered key tips for effectively utilizing lipid nanoparticles, allowing us to successfully transfect them into HEK and CFTR-specific cells. Additionally, we replicated the proof of concept established by David Liu et al. in 2019, gaining valuable insights into how a Prime Editing system should function. During this process, we identified a gap in measuring the efficiency of our Prime Editing constructs. To address this, we developed a fluorescence-based system with high selectivity, allowing us to perform precise efficiency analyses. Beyond lab work, we connected with various industry members, not only to secure sponsorships but to emphasize collaboration and exchange with leading market players. Through this network, we not only received feedback on the importance of physiological therapies but also gained insights into the insurance-related challenges associated with a fundamental disease like Cystic Fibrosis. This motivated us to take our project to the next level. Our collaboration with the medical faculty of our university, along with national feedback, enabled us to integrate new and advanced validation methods. In the next phase of our studies, we decided to optimize lipid nanoparticles (LNPs) to make them suitable for a novel, lung-specific delivery strategy. Additionally, we prepared to explore enzyme engineering by engaging with stakeholders experienced in nickase development and yeast experimental design, further refining our Prime Editing system.
</p>,
<ul>
<li>
@@ -1152,7 +1152,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<b>Development of Fluorescence-Based Efficiency Measurement:</b> Created a highly selective fluorescence-based system to accurately measure the efficiency of Prime Editing constructs.
</li>
<li>
- <b>Industry Collaboration:</b> Established connections with industry members for sponsorships, collaboration, and feedback on broader challenges such as insurance issues tied to fibrosis treatment.
+ <b>Industry Collaboration:</b> Established connections with industry members for sponsorships, collaboration, and feedback on broader challenges such as insurance issues tied to Cystic Fibrosis treatment.
</li>
<li>
<b>Optimization of LNPs for Lung-Specific Delivery:</b> Began optimizing lipid nanoparticles for lung-specific delivery, while preparing for enzyme engineering to enhance the Prime Editing system.
@@ -1519,8 +1519,8 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
language: "de",
quoteVorname: "Dr. Benjamin",
quoteNachname: "Winkeljann, Expert for LNPs und Ceo von RNhale",
- quote: "Spray-drying LNPs is a groundbreaking approach that enhances stability and enables efficient pulmonary delivery of mRNA, paving the way for innovative therapies for conditions like fibrosis.",
- aimofcontact: [<p>As part of our development process of an innovative, effective pulmonary delivery of therapeutic mRNA to fight fibrosis,
+ quote: "Spray-drying LNPs is a groundbreaking approach that enhances stability and enables efficient pulmonary delivery of mRNA, paving the way for innovative therapies for conditions like Cystic Fibrosis.",
+ aimofcontact: [<p>As part of our development process of an innovative, effective pulmonary delivery of therapeutic mRNA to fight Cystic Fibrosis,
we conducted an interview with Dr. Benjamin Winkeljann, who is the Co-Founder of <a href="https://rnhale.com/">RNhale</a>. Dr. Benjamin
Winkeljann has a wealth of experience in the field of RNA therapeutics and nanotechnology. His background includes extensive research in the
development of lipid-based delivery systems, focusing on optimizing stability and efficacy for therapeutic applications. Winkeljann’s work
@@ -1599,7 +1599,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteVorname:" Dr. Katharina",
quoteNachname: "Kolonko, LNP Specialist with focus on chitosan and CF therapies ",
quote: "Wow, you’re already further along than I was! That’s a really good approach, especially since dry powder formulations can help with stability.",
- aimofcontact: [<p>Our goal in reaching out to Dr. Katharina Kolonko, who earned her PhD working on chitosan-based nanoparticles for delivering nucleic acids to human respiratory cells in the context of fibrosis, was to seek her advice on the design, stability, and application of nanoparticles. We wanted to learn from her experience with chitosan-capsaicin nanoparticles, especially the challenges she encountered, and apply her insights to improve our own project. Specifically, we aimed to better understand nanoparticle stability, transfection methods, and how to effectively design our experiments.
+ aimofcontact: [<p>Our goal in reaching out to Dr. Katharina Kolonko, who earned her PhD working on chitosan-based nanoparticles for delivering nucleic acids to human respiratory cells in the context of Cystic Fibrosis, was to seek her advice on the design, stability, and application of nanoparticles. We wanted to learn from her experience with chitosan-capsaicin nanoparticles, especially the challenges she encountered, and apply her insights to improve our own project. Specifically, we aimed to better understand nanoparticle stability, transfection methods, and how to effectively design our experiments.
</p>],
insights: [<p>Dr. Kolonko provided us with valuable insights into working with nanoparticles, particularly emphasizing the advantages of nanocapsules. She highlighted that nanocapsules are more stable than nano-complexes, which is crucial for experiments involving complex environments and high shear forces. Additionally, her use of capsaicin wasn’t aimed at improving transfection efficiency but was part of a broader strategy targeting specific channels. She also shared practical methods for measuring particle stability and cytotoxicity, giving us clear guidance on tools and techniques that we can apply to our project.
Furthermore, Dr. Kolonko discussed the use of chitosan as a component in nanoparticle formulations. Chitosan, with its positive charge, can interact with mRNA, potentially enhancing the stability of the cargo. As an outlook, we plan to explore modifications using chitosan to improve the stability and performance of our mRNA delivery system. This approach may provide a more robust solution for optimizing nanoparticle formulations in future experiments. </p>],
@@ -1615,7 +1615,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
</ul>
] ,
interview:<>
- <QaBox q="How did you approach the design of Lipid Nanoparticles (LNPs)? What were the first steps you took at that time? Were you already familiar with LNPs, or was that a completely new experience for you?" a="I started working with nanoparticles during my bachelor’s thesis. I continued with nanoparticles into my master’s thesis, working on a project related to fibrosis. Initially, I worked with nano-complexes, but later switched to nano-capsules due to their stability, especially in cell culture media." />
+ <QaBox q="How did you approach the design of Lipid Nanoparticles (LNPs)? What were the first steps you took at that time? Were you already familiar with LNPs, or was that a completely new experience for you?" a="I started working with nanoparticles during my bachelor’s thesis. I continued with nanoparticles into my master’s thesis, working on a project related to Cystic Fibrosis. Initially, I worked with nano-complexes, but later switched to nano-capsules due to their stability, especially in cell culture media." />
<QaBox q="Since you’re focused on stability and applying high shear forces, could you explain why nano-capsules are more stable than complexes in this context?" a="Nano-capsules are generally more stable in cell culture media compared to nano-complexes, which often react with additives and proteins. However, I didn’t explore shear forces much further. My main goal was to stabilize the particles in cell culture media for testing on cells." />
<QaBox q="What kind of cell culture medium did you use for these experiments?" a="For transfection, we used Optimem as the medium, after removing antibiotics from the culture medium 24 hours prior to transfection." />
<QaBox q="How long did it take you to get to the point where you used nano-capsules?" a="I only started working with nano-capsules towards the end of my PhD. I spent much of my time with nano-complexes, but when I visited a lab in Leeds, I shifted to nano-capsules. This transition happened quite late, just months before I finished my thesis." />
@@ -1675,10 +1675,10 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
I have acquired the ability to create standard operating procedures (SOPs) that guarantee the transparent and traceable documentation of each stage of the research process. This not only facilitates internal organisation but is also crucial for subsequent approvals and audits by regulatory authorities.
It is of paramount importance to ensure the reproducibility of our experiments by maintaining accurate protocols and meticulously documenting all variables. This is of particular importance should the intention be to pursue clinical research at a later stage, as the reproducibility of experiments is a crucial factor in the validity of the results.
I acquired knowledge of techniques for risk assessment, including Failure Mode and Effects Analysis (FMEA). This process enables the identification of potential risks in a project at an early stage, thus facilitating the development of strategies to minimise them. This approach allows us to identify and address potential sources of error before they lead to significant issues.
- This knowledge is crucial as we think about the future of our project, particularly if we aim to move our gene therapy approach for fibrosis closer to clinical trials and real-world applications. My participation in the GXP training has equipped me with the necessary tools to potentially guide our team through the complex regulatory landscape, ensuring our work remains aligned with industry standards and ready for the next steps in development.
+ This knowledge is crucial as we think about the future of our project, particularly if we aim to move our gene therapy approach for Cystic Fibrosis closer to clinical trials and real-world applications. My participation in the GXP training has equipped me with the necessary tools to potentially guide our team through the complex regulatory landscape, ensuring our work remains aligned with industry standards and ready for the next steps in development.
One of the key speakers during the GXP course was <HPLinktoOtherHPTab tab="gxpexpert" text="Dr. Marcus Berger" />, whose expertise was invaluable to me and the entire team. After the course, I had the opportunity to ask Dr. Berger some questions, further deepening my understanding of the practical applications of GXP in research. The connection with Dr. Berger has been highly beneficial, as his insights helped shape key aspects of our project’s development and compliance with industry standards. His guidance will continue to be a valuable resource for our team moving forward.
Through this training, I feel better positioned to contribute to the team’s efforts, ensuring our project adheres to global safety and ethical guidelines. This experience has strengthened our approach and set a solid foundation for future progress, ensuring that our research, public engagement, and potential clinical applications continue to meet the highest regulatory standards. </p>],
- summary: "Kaya, a member of the iGEM Bielefeld 2024 team, completed an intensive one-week GXP (Good Practice) training, which covered Good Laboratory Practice (GLP), Good Clinical Practice (GCP), and Good Manufacturing Practice (GMP). The training provided valuable insights into maintaining high standards of quality, safety, and ethics throughout the research process. Kaya learned crucial skills, such as documenting research processes for reproducibility, creating standard operating procedures (SOPs), and conducting risk assessments using techniques like Failure Mode and Effects Analysis (FMEA). This knowledge is essential for advancing their fibrosis gene therapy project toward clinical trials and ensuring compliance with regulatory standards. Dr. Marcus Berger, a key speaker in the course, provided additional guidance, offering valuable insights that will continue to benefit the team.",
+ summary: "Kaya, a member of the iGEM Bielefeld 2024 team, completed an intensive one-week GXP (Good Practice) training, which covered Good Laboratory Practice (GLP), Good Clinical Practice (GCP), and Good Manufacturing Practice (GMP). The training provided valuable insights into maintaining high standards of quality, safety, and ethics throughout the research process. Kaya learned crucial skills, such as documenting research processes for reproducibility, creating standard operating procedures (SOPs), and conducting risk assessments using techniques like Failure Mode and Effects Analysis (FMEA). This knowledge is essential for advancing their Cystic Fibrosis gene therapy project toward clinical trials and ensuring compliance with regulatory standards. Dr. Marcus Berger, a key speaker in the course, provided additional guidance, offering valuable insights that will continue to benefit the team.",
months: "august",
pictureurl_implementation: "https://static.igem.wiki/teams/5247/photos/for-wiki-texts/gxp/gxp-course-kaya.webp",
pictureurl_interview:"https://static.igem.wiki/teams/5247/photos/for-wiki-texts/gxp/gxp-course-group.webp",
@@ -1693,11 +1693,11 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
heading: "Building Connections: The Power of Collaboration and Communication",
interviewtabid: "connect",
cardtext: "",
- quote: "Sharing our project updates and ideas with a diverse audience has been enlightening. The feedback we receive drives our innovation and reinforces the value of collaboration in tackling the challenges of fibrosis.",
+ quote: "Sharing our project updates and ideas with a diverse audience has been enlightening. The feedback we receive drives our innovation and reinforces the value of collaboration in tackling the challenges of Cystic Fibrosis.",
quoteNachname: "Susat, Teammember",
quoteVorname: "Kathleen",
type: "meta",
- summary: [<p>During our project journey, we made a concerted effort to connect and share insights with a wide array of individuals, leveraging social media and university networks. Engaging with peers, faculty, and industry professionals not only enriched our understanding but also fostered a collaborative spirit. Through platforms like LinkedIn and university forums, we initiated discussions that brought fresh perspectives on gene therapy and fibrosis.
+ summary: [<p>During our project journey, we made a concerted effort to connect and share insights with a wide array of individuals, leveraging social media and university networks. Engaging with peers, faculty, and industry professionals not only enriched our understanding but also fostered a collaborative spirit. Through platforms like LinkedIn and university forums, we initiated discussions that brought fresh perspectives on gene therapy and Cystic Fibrosis.
Our social media outreach allowed us to share our findings and progress with a broader audience, promoting awareness and sparking interest in our work. We actively participated in online discussions and webinars, exchanging ideas and feedback with experts from various fields. This exchange of knowledge has proven invaluable, guiding us in refining our methodologies and enhancing our project’s impact.
We also engaged with different community organizations and advocacy groups, which helped us grasp the societal implications of our research. Through these connections, we garnered support for our initiatives and increased visibility for our cause. This collaborative approach not only strengthened our project but also highlighted the importance of community involvement in scientific research.
</p>,
@@ -1707,7 +1707,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<b>Community Engagement:</b> Connected with community organizations and advocacy groups to understand the societal implications of our research and garner support.
</li>
<li>
- <b>Social Media Outreach:</b> Utilized platforms like LinkedIn to share progress and findings, promoting awareness of fibrosis and gene therapy.
+ <b>Social Media Outreach:</b> Utilized platforms like LinkedIn to share progress and findings, promoting awareness of Cystic Fibrosis and gene therapy.
</li>
<li>
<b>Collaborative Discussions:</b> Engaged in discussions with peers, faculty, and industry professionals, exchanging insights and refining methodologies.
@@ -1737,7 +1737,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
implementation: [<p>The next phase of developing a new hygiene concept is to maintain contact with Mr. Johannfunke in order to continue to advance the hygiene concept in collaboration. The strategic approach entails the incremental implementation of measures, exemplified by the establishment of the inaugural toilet facility within the main building. It is of the utmost importance to ensure the uninterrupted implementation of the hygiene concept. In order to achieve this, it is essential to draw upon the existing plans and measures that have already been implemented in new buildings. We are working on advancing the plans at a higher level and are in regular dialogue with the Central contact point Barrier-free in order to overcome bureaucratic hurdles and actively promote the topic. Furthermore, it is necessary to intensify lobbying work in order to gain greater support for this issue at both the university and political levels. </p>],
language: "de",
interview: <>
- <QaBox q="What do you think of our hygiene concept and our plan?" a="This is a very acute problem. It particularly affects students with disabilities and immune-compromised staff, such as those with cancer or fibrosis, who are forced to work from home. The problem is: Employees can work from home, but students cannot. There is a great need for hygiene measures, as contact must be avoided to minimise the risk of infection." />
+ <QaBox q="What do you think of our hygiene concept and our plan?" a="This is a very acute problem. It particularly affects students with disabilities and immune-compromised staff, such as those with cancer or Cystic Fibrosis, who are forced to work from home. The problem is: Employees can work from home, but students cannot. There is a great need for hygiene measures, as contact must be avoided to minimise the risk of infection." />
<QaBox q="What are the challenges in implementing the hygiene concept?" a="There is a lack of strategic development, although your hygiene concept is well developed. It is necessary to proceed in small steps, e.g. starting with a toilet in the main building. However, bureaucracy is a major obstacle. To be implemented, an application has to be submitted to the rectorate, and these processes are often lengthy and complicated." />
<QaBox q="What is the current situation at our university?" a="While some progress has been made with the installation of additional toilets and disabled-friendly toilets in new buildings, there is as yet no overarching strategy in place to guide future developments. Furthermore, the lack of clarity regarding the mission statement and objectives leaves room for ambiguity. The duty of care that employers have towards employees is established, yet the situation is regulated differently with regards to students. The possibility of receiving compensation for disadvantages is open, but is frequently seen as inadequate." />
<QaBox q="What are the next steps in implementing the hygiene concept?" a="It is essential that the concept be implemented in small, strategic steps. At the same time, it is vital that the rectorate and other decision-makers be consulted on a regular basis to ensure that this matter remains at the forefront of discussions. Furthermore, it is of great importance to engage in political lobbying to secure additional support for this issue." />
@@ -1760,26 +1760,26 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteNachname: "Vaidyanathan, Innovative leader in pediatric research",
quoteVorname: "Dr. Sriram",
quote: "I think you're thinking about it the right way.[...] I would have talked to all of the exact people that you have already spoken with.",
- aimofcontact: [<p>The objective of this contact was to gather further information about fibrosis (CF) in Asia, with a particular focus on understanding potential data biases, identifying common mutations, exploring the available medications, and assessing the diagnostic practices in the region. </p>],
- insights: [<p>The talk with Sriram revealed that, although fibrosis (CF) is relatively uncommon in Asia compared to other disease like sickle cell disease, it nevertheless exhibits considerable genetic diversity. The identification of different mutations in the CFTR gene across the region has revealed that the F508del mutation is the most common, a finding that aligns with global patterns. However, in Asian populations, other rare mutations are also prevalent, which presents unique challenges in diagnosis and treatment.
+ aimofcontact: [<p>The objective of this contact was to gather further information about Cystic Fibrosis (CF) in Asia, with a particular focus on understanding potential data biases, identifying common mutations, exploring the available medications, and assessing the diagnostic practices in the region. </p>],
+ insights: [<p>The talk with Sriram revealed that, although Cystic Fibrosis (CF) is relatively uncommon in Asia compared to other disease like sickle cell disease, it nevertheless exhibits considerable genetic diversity. The identification of different mutations in the CFTR gene across the region has revealed that the F508del mutation is the most common, a finding that aligns with global patterns. However, in Asian populations, other rare mutations are also prevalent, which presents unique challenges in diagnosis and treatment.
Additionally, it was found that environmental factors, such as air pollution, serve to exacerbate the symptoms of CF, particularly in densely populated regions, thereby further complicating the management of the disease. This emphasises the necessity for further research on CF that is specifically tailored to the needs of different regions, including improvements in diagnostic techniques and the development of treatments that are more closely aligned with the characteristics of the populations in question. </p>],
implementation: [<p>The data were incorporated by confirming that the F508del mutation is not only the most common in Europe but also globally, including in Asia, highlighting a broader perspective and contributing to a significant horizon expansion in understanding the mutation's worldwide prevalence. This finding lends support to the idea that existing therapies targeting the F508del mutation will be effective for many patients worldwide, thereby providing a solid foundation for treatment. As a starting point, this is promising, but future efforts will focus on adapting therapies to address other, rarer mutations found in specific populations.
</p>],
- summary: "The contact provided valuable insights into fibrosis (CF) in Asia and confirmed that the F508del mutation is the most common, as it is globally. However, the genetic diversity observed in Asia, together with the exacerbation of symptoms by environmental factors such as air pollution, highlights the need for more region-specific research. Future efforts will focus on refining treatments for rarer mutations and improving diagnostic accuracy in Asian populations.",
+ summary: "The contact provided valuable insights into Cystic Fibrosis (CF) in Asia and confirmed that the F508del mutation is the most common, as it is globally. However, the genetic diversity observed in Asia, together with the exacerbation of symptoms by environmental factors such as air pollution, highlights the need for more region-specific research. Future efforts will focus on refining treatments for rarer mutations and improving diagnostic accuracy in Asian populations.",
months: "September",
interview:<>
<QaBox q="Should we explain our project a little bit in the beginning?" a="That would be great."/>
- <QaBox q="Okay. As I wrote to you in the email, we are part of the Bielefeld-CeBiTec item team in Germany this year, and we have a project called Precyse, where we want to develop a platform for next-generation prime editing. With that, we aim to tackle CF mutations, starting with the F508del mutation. The complex should be adaptable for different mutations with minor changes. Essentially, we want to operate like Cas-based projects using prime editing tools but may transition to using a fanzor nickase in the future. Currently, we are working on a delivery system utilizing lipid nanoparticles to transport our complex into the lungs. These nanoparticles will be loaded with mRNA and designed for inhalation, similar to how asthma medications are delivered. Our goal is to advance personalized medicine and improve the standard of living for those affected by the disease, especially since a friend of one of our team members has fibrosis and shares insights on the challenges faced by those with the condition." a="That's a very nice summary. I have a couple of clarifying questions. Are you undergraduates?" />
+ <QaBox q="Okay. As I wrote to you in the email, we are part of the Bielefeld-CeBiTec item team in Germany this year, and we have a project called Precyse, where we want to develop a platform for next-generation prime editing. With that, we aim to tackle CF mutations, starting with the F508del mutation. The complex should be adaptable for different mutations with minor changes. Essentially, we want to operate like Cas-based projects using prime editing tools but may transition to using a fanzor nickase in the future. Currently, we are working on a delivery system utilizing lipid nanoparticles to transport our complex into the lungs. These nanoparticles will be loaded with mRNA and designed for inhalation, similar to how asthma medications are delivered. Our goal is to advance personalized medicine and improve the standard of living for those affected by the disease, especially since a friend of one of our team members has Cystic Fibrosis and shares insights on the challenges faced by those with the condition." a="That's a very nice summary. I have a couple of clarifying questions. Are you undergraduates?" />
<QaBox q="No, we are overgraduates at the University of Bielefeld." a="Oh, great! So, how are you optimizing prime editing, and who is helping you with this process?" />
<QaBox q="We tried to reach out to Dr. Liu, but he hasn't responded yet." a="You should also contact Marianne Carlon in Leuven, Belgium. They didn't work on Delta F508, but Mattijs is a graduate student there and could be helpful." />
<QaBox q="We’ve already been in touch with Mattijs; he’s been very nice." a="Perfect! You're reaching out to the right people. Are you aware of the Liu lab's Nature Biomedical Engineering paper that optimizes prime editing?" />
<QaBox q="Yes, we are aware of it." a="Great! How can I help you further?" />
- <QaBox q="We have some questions about fibrosis (CF) in Asia, specifically about its perceived rarity and its impact on diagnosis and research efforts." a="I can share some insights. While CF is often considered rare in Asia, it's essential to recognize that it affects various populations. In my lab, we analyze CF-causing mutations across different populations using data from Nomad, which focuses on genetic ancestry rather than self-identified race. CF is most prevalent in individuals of European origin, but it remains common across many populations worldwide, particularly in South Asia and other regions, although East Asia has lower prevalence rates." />
+ <QaBox q="We have some questions about Cystic Fibrosis (CF) in Asia, specifically about its perceived rarity and its impact on diagnosis and research efforts." a="I can share some insights. While CF is often considered rare in Asia, it's essential to recognize that it affects various populations. In my lab, we analyze CF-causing mutations across different populations using data from Nomad, which focuses on genetic ancestry rather than self-identified race. CF is most prevalent in individuals of European origin, but it remains common across many populations worldwide, particularly in South Asia and other regions, although East Asia has lower prevalence rates." />
<QaBox q="That’s helpful. Can you explain how the perceived rarity of CF impacts diagnosis and research in Asia?" a="The perception of CF as a 'white people disease' leads to underdiagnosis and fewer research efforts in Asian populations. For instance, in India, physicians are more familiar with conditions like sickle cell disease and thalassemia, leading to limited awareness of CF. Early diagnosis is challenging due to the lack of screening programs for CF, and currently, many regions are only beginning to implement sweat chloride testing." />
<QaBox q="Thank you! What mutations are more common in Asia?" a="Delta F508 is still the most frequent variant across different populations, but its frequency decreases in non-European groups. While there are several other common variants, they do not respond to modulator therapeutics, which complicates treatment options. Focusing on Delta F508 is sensible, but you might face challenges in finding individuals with other variants due to the limited awareness and screening for CF in those populations." />
<QaBox q="Okay. But you would say that the other mutations are a bit under-researched, right?" a="They are completely under-researched, right? Like I have not seen anybody work on L218X. I didn't even know L218X existed until I started looking into that group. And it seems like L218X, I've not seen it in any other population that frequently. So a lot of these variants seem to be unique. I don't know if they're completely unique to South Asians or if you go to Iran or something, whether you're going to start finding these people or whether if you go to Southeast Asia, then you're going to start finding some of these people, right? So we don't know that, but I think it will be really hard for you to find cells with these variants."/>
- <QaBox q="What do you believe, what steps should be done in the scientific community to ensure better representation of Asian populations, or in general, not only Western populations, in the genetic studies for fibrosis?" a="Ah, it's a very good question; it's also a question that doesn't have a very straightforward answer, right? In the sense that one, you know, you have to raise awareness and ensure that people are getting diagnosed, that's step number one, right. A lot of the numbers they showed you are based on prediction; they're not based on a registry in any country with some of these populations, right. India doesn't have a registry, China doesn't have a registry. There are efforts to make a registry, but you know it's a little hard to get started when you know if you go and argue that there are 6,000 children with CF that are being born in a year in India, and then the response that I've received before is well, there are about 60,000 kids dying of diarrhea, and you know so the priority from public health is often different. It's starting to change. So one focus would be diagnosis and raising awareness, and if you come across groups that are trying to improve their ability to diagnose then you try to help them, right. What's your other question in terms of treatment and research? I think being aware of it if you do end up finding samples that have some of these unique variants. Right then, trying to form a strategy around it is not bad; I've seen people identify variants of CF that are unique to different populations. I think even Marianne Carlon's group picked up variants that are unique to Belgium and then tried prime editing on that right, so those sort of strategies help quite a bit, right? And this is partially the reason you know I continue to focus on mutation-agnostic strategies; I've not really branched out into the Prime Editing world or the Base Editing world primarily because this seems to be a pretty challenging issue, but that said, you want as many shots at goal as possible. So, you know, if you're able to use prime editing to fix Delta 508, that still helps the most number of people with CF. And then maybe, you know, you could use that platform that's really well validated at that point, then go start targeting individual variants. The other idea with prime editing is that there's hope that you can probably at least try to replace one exon at a time. And that sort of makes things a little bit more manageable, right? So if you, you know, it's a little bit more, like if you have to target every single variant, then you're thinking about hundreds of different medical products. But if you're thinking of replacing every exon, that's 27, that's a countable number. And I would argue that you don't even have to do all 27. You start with the hotspots, right? You start with exon 11, you start with exon 12. But then as part of the conversation, I think we should just be thinking about exons that might be hotspots for variants in other populations and at least, you know, keeping them somewhere in the packing order so that as you go through the most frequent variants, right? And you're starting to develop strategies for the most frequent variants. Those are on the list so that we eventually get to them."/>
- <QaBox q="Yeah. Okay. And what do you think? Are there any healthcare challenges managing fibrosis in Asian countries?" a="Oh, absolutely. Right. I think, you know, if you're in a high-income country like Japan, I think, you know, the biggest challenge is awareness and getting diagnosed. If you're in low- and middle-income countries, right, then the challenge becomes access to drugs. So modulators are not available in most of Asia, or at least in the lower and middle-income countries in Asia, that's not available, like, it's not available for sale. Primarily, partly because they think that those people, like people at CF, don't exist in enough numbers in those countries. And partly because then it creates problems in terms of monetary, like factors, right. In terms of how much they charge for the modulators. Right. So those don't exist. I don't know, like they have access to pancreatic enzymes and things like that, but, you know, regular access to regular care is still a challenge. The base of physicians that are family with treating CF is only growing in some of those places at least from my personal experience but it’s not by where we are. The children there are still mostly passing away when they are in their teenage years or early twenties. The sort of life span and life expectancy is no where close to what you would see in high-income countries. So, I think that is the biggest challenge, access to resources both, publicly and also from a private point."/>
+ <QaBox q="What do you believe, what steps should be done in the scientific community to ensure better representation of Asian populations, or in general, not only Western populations, in the genetic studies for Cystic Fibrosis?" a="Ah, it's a very good question; it's also a question that doesn't have a very straightforward answer, right? In the sense that one, you know, you have to raise awareness and ensure that people are getting diagnosed, that's step number one, right. A lot of the numbers they showed you are based on prediction; they're not based on a registry in any country with some of these populations, right. India doesn't have a registry, China doesn't have a registry. There are efforts to make a registry, but you know it's a little hard to get started when you know if you go and argue that there are 6,000 children with CF that are being born in a year in India, and then the response that I've received before is well, there are about 60,000 kids dying of diarrhea, and you know so the priority from public health is often different. It's starting to change. So one focus would be diagnosis and raising awareness, and if you come across groups that are trying to improve their ability to diagnose then you try to help them, right. What's your other question in terms of treatment and research? I think being aware of it if you do end up finding samples that have some of these unique variants. Right then, trying to form a strategy around it is not bad; I've seen people identify variants of CF that are unique to different populations. I think even Marianne Carlon's group picked up variants that are unique to Belgium and then tried prime editing on that right, so those sort of strategies help quite a bit, right? And this is partially the reason you know I continue to focus on mutation-agnostic strategies; I've not really branched out into the Prime Editing world or the Base Editing world primarily because this seems to be a pretty challenging issue, but that said, you want as many shots at goal as possible. So, you know, if you're able to use prime editing to fix Delta 508, that still helps the most number of people with CF. And then maybe, you know, you could use that platform that's really well validated at that point, then go start targeting individual variants. The other idea with prime editing is that there's hope that you can probably at least try to replace one exon at a time. And that sort of makes things a little bit more manageable, right? So if you, you know, it's a little bit more, like if you have to target every single variant, then you're thinking about hundreds of different medical products. But if you're thinking of replacing every exon, that's 27, that's a countable number. And I would argue that you don't even have to do all 27. You start with the hotspots, right? You start with exon 11, you start with exon 12. But then as part of the conversation, I think we should just be thinking about exons that might be hotspots for variants in other populations and at least, you know, keeping them somewhere in the packing order so that as you go through the most frequent variants, right? And you're starting to develop strategies for the most frequent variants. Those are on the list so that we eventually get to them."/>
+ <QaBox q="Yeah. Okay. And what do you think? Are there any healthcare challenges managing Cystic Fibrosis in Asian countries?" a="Oh, absolutely. Right. I think, you know, if you're in a high-income country like Japan, I think, you know, the biggest challenge is awareness and getting diagnosed. If you're in low- and middle-income countries, right, then the challenge becomes access to drugs. So modulators are not available in most of Asia, or at least in the lower and middle-income countries in Asia, that's not available, like, it's not available for sale. Primarily, partly because they think that those people, like people at CF, don't exist in enough numbers in those countries. And partly because then it creates problems in terms of monetary, like factors, right. In terms of how much they charge for the modulators. Right. So those don't exist. I don't know, like they have access to pancreatic enzymes and things like that, but, you know, regular access to regular care is still a challenge. The base of physicians that are family with treating CF is only growing in some of those places at least from my personal experience but it’s not by where we are. The children there are still mostly passing away when they are in their teenage years or early twenties. The sort of life span and life expectancy is no where close to what you would see in high-income countries. So, I think that is the biggest challenge, access to resources both, publicly and also from a private point."/>
<QaBox q="Okay. And do you think that the environmental or like pollution is doing, like, I don't know how to say it correctly. So that there are like respiratory challenges because, yeah, right." a="Absolutely! Right. It's, you know, your exposure to pathogens is higher. Your exposure to pollution is significantly higher in a lot of these countries, right? Even China, which is, you know, much better off than a country like India still has a lot of pollution, right? And so, or I've never been to China, but at least that's what I read, right? But India, I grew up there and I can firsthand tell you that there's a lot of pollution there, right? And that I'm sure it affects it because it's been documented to affect people, just people without CF, right? On an average, it's supposed to like, the reports that I've seen are like, it takes a few years off of life expectancy for even people that have not been diagnosed with anything else, right? So I'm sure if there's a person with CF, then that's going to affect them even more negatively in places like India. And then I think that the exposure to pathogens might be slight, it might be higher and the lower down in the economic scale you go, the more, the exposure to pathogens are going to have, right? I think the third factor would also might be nutrition, right? Like, so if you have CF, you, you know, here, one way they were managing it was through high fat calorie rich diet and access to those sort of high fat calorie rich diets may not be that prevalent in some of these other places. And so that might become a factor."/>
<QaBox q="Okay. And then one last question. So it's more a bit about you and your research. So what drew your attention to the issue of CF in Asia? So why did you start researching this topic?" a="Oh, so that was a very okay. So the way it happened was I was at a conference and I was a postdoc at Stanford previously, as you know. So I was at a conference and I was, we were just, I think had a sort of a, you know, a dinner for everybody from our institution. And I was chatting with the nurse in the table and sitting in the table and so the San Francisco area has a lot of people from Asia. And so the nurse particularly said that they had a lot of children with CF who happened to originate in India, which really surprised me because that's not a disease that I'd heard growing up as being some, a common genetic disease there. Right. And so that kind of drew my attention. And then in subsequent conversations, when I spoke with physicians who are practicing in India, nobody said that they hadn't seen people with CF, right? Whenever I had the conversation, people would be like, oh yeah, I've seen a patient with CF or I know of somebody that has a child with CF, right? Which was surprising. And then when I went to India, I went to a hospital in my hometown and I was talking to the pulmonologist there. And he said he had 30 or 40 patients with CF in that, just that hospital. And that's, you know, it's not even, you know, it's probably one of the bigger cities in India, but I wouldn't even know if it's in the top 10 biggest cities in India. And this is not even the biggest center in that city necessarily. Right. So I was just really surprised. And he said he didn't have to really put in any effort to go seek out any people with CF. This was just in the people that walked into that hospital that got diagnosed with CF, right? So that kind of solidified the perception that perhaps it was underdiagnosed. And then the reason it happened was because the pandemic sort of forced most of us out of the lab. And so this ended up being something you can actually work on without going to lab."/>
<QaBox q="Yeah, sounds really interesting the way you got there. at the beginning, you said you could tell us something, what came to you in mind when you heard about Project First, and you already prepared some slides. So I just want to ask if you maybe have something more to tell us that you haven't thought about yet, because the interview until now is highly interesting and I think we got a lot of new information until now. So maybe you have something. Maybe you have more interesting information for us." a="I think you guys covered a lot of what I had in mind, right? In terms of, I think if I were to start with prime editing and Delta 508, this is exactly the argument I would have made. I would have talked to all of the exact people that you have already spoken with, right? So I think that I don't have too much to add there. The only thing you could do is if you are looking at variants, right? Maybe I would try to come up with a plan for what variants you might go after, after Delta 508, right? And that could become part of your future research or something like that. Or if you can, I guess the one question I had for you is, so the correction of Delta 508 is sort of published, right? People have reported using lipid nanoparticles, at least to deliver base editing reagents, right? So how are you thinking about, like when you pitch the innovation in your project, how are you thinking about pitching your innovation? "/>
@@ -1824,16 +1824,16 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quoteNachname: "Ingatova, CF Research Experts ",
quoteVorname: "Prof. Dr. Zoya",
quote: "Precision is key to minimizing side effects and ensuring the safety of your therapy.",
- aimofcontact: "We conducted the interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti from the Institute of Biochemistry and Molecular Biology at the University of Hamburg, seeking to deepen our understanding of their research on fibrosis (CF) and explore additional CF mutations, as well as to learn more about cell culture techniques specific to CF research, since they send us the CFBE41o- cell line. Our aim was also to gather more information about their approaches in CF research, particularly their focus on treating genetic mutations like nonsense mutations, which are highly prevalent in CF.",
- insights: [<p>We were struck by Ignatova’s story about founding the iGEM team in Hamburg. Her passion for fostering creativity and innovation in science was inspiring. On a technical level, their advice on cell culture was incredibly practical and immediately useful. Dr. Nikhil Bharti explained how they handle CFBE41o- cells and ALI (air-liquid interface) cell culture. This advice directly addressed the challenges we’ve faced in our own lab, giving us a method to improve our cell culture success rates. During our interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti, their innovative approach to fibrosis (CF) therapy, particularly "read-through" and tRNA-based therapies, stood out. "Read-through" therapies aim to bypass premature stop codons that prevent full protein production, offering a way to restore the function of critical proteins like CFTR in CF. This approach has the potential to treat a broad range of genetic diseases caused by similar mutations. The tRNA-based therapy is even more precise, targeting mRNA to correct faulty codons without altering the DNA, making it safer for long-term use. This flexibility, along with the ability to apply these therapies beyond CF, broadened our understanding of how such strategies can revolutionize treatments for genetic disorders.Ignatova highlighted, that using cell models closely resembling properties of the cell types targeted by our therapy is important for ensuring applicability of our approach to patient cells and its safety. A key focus throughout the discussion was safety. Prof. Ignatova emphasized the importance of ensuring that the therapies are highly specific, targeting only the defective codons while avoiding natural stop codons to prevent uncontrolled protein production. Moreover, their careful consideration of delivery systems further demonstrated their commitment to minimizing risks like toxicity in unintended organs. Their meticulous approach to safety has influenced how we think about developing and applying these therapies, making it clear that ensuring patient safety is as critical as achieving therapeutic success.</p>],
+ aimofcontact: "We conducted the interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti from the Institute of Biochemistry and Molecular Biology at the University of Hamburg, seeking to deepen our understanding of their research on Cystic Fibrosis (CF) and explore additional CF mutations, as well as to learn more about cell culture techniques specific to CF research, since they send us the CFBE41o- cell line. Our aim was also to gather more information about their approaches in CF research, particularly their focus on treating genetic mutations like nonsense mutations, which are highly prevalent in CF.",
+ insights: [<p>We were struck by Ignatova’s story about founding the iGEM team in Hamburg. Her passion for fostering creativity and innovation in science was inspiring. On a technical level, their advice on cell culture was incredibly practical and immediately useful. Dr. Nikhil Bharti explained how they handle CFBE41o- cells and ALI (air-liquid interface) cell culture. This advice directly addressed the challenges we’ve faced in our own lab, giving us a method to improve our cell culture success rates. During our interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti, their innovative approach to Cystic Fibrosis (CF) therapy, particularly "read-through" and tRNA-based therapies, stood out. "Read-through" therapies aim to bypass premature stop codons that prevent full protein production, offering a way to restore the function of critical proteins like CFTR in CF. This approach has the potential to treat a broad range of genetic diseases caused by similar mutations. The tRNA-based therapy is even more precise, targeting mRNA to correct faulty codons without altering the DNA, making it safer for long-term use. This flexibility, along with the ability to apply these therapies beyond CF, broadened our understanding of how such strategies can revolutionize treatments for genetic disorders.Ignatova highlighted, that using cell models closely resembling properties of the cell types targeted by our therapy is important for ensuring applicability of our approach to patient cells and its safety. A key focus throughout the discussion was safety. Prof. Ignatova emphasized the importance of ensuring that the therapies are highly specific, targeting only the defective codons while avoiding natural stop codons to prevent uncontrolled protein production. Moreover, their careful consideration of delivery systems further demonstrated their commitment to minimizing risks like toxicity in unintended organs. Their meticulous approach to safety has influenced how we think about developing and applying these therapies, making it clear that ensuring patient safety is as critical as achieving therapeutic success.</p>],
implementation: "Prof. Ignatova's practical advice on cell culture had a transformative impact on our project. By adopting her method for CFBE41o- cells and improving our lab's sterilization protocols, we successfully established the cell line and significantly reduced the risk of contamination. In addition, her emphasis on safety in gene therapy guided us to review our Prime Editing construct and lipid nanoparticle (LNP) design. We focused on minimizing toxicity and off-target effects while ensuring precise targeting of lung tissues and the F508del mutation of the CFTR gene, making our approach safer and more efficient",
- summary: "In our interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti from the Institute of Biochemistry and Molecular Biology at the University of Hamburg, we aimed to deepen our understanding of their research on fibrosis (CF) and explore additional mutations. Their innovative approaches, particularly tRNA-based therapies, aim to restore the function of proteins like CFTR by bypassing premature stop codons, presenting potential treatments for various genetic diseases. They highlighted the importance of precision in therapy to minimize side effects and enhance safety, emphasizing careful delivery systems to avoid toxicity. Prof. Ignatova's practical insights into cell culture techniques significantly improved our lab's success with CFBE41o- cells, guiding us to enhance sterilization protocols and refine our Prime Editing constructs. This transformative exchange underscored the critical balance between achieving therapeutic success and ensuring patient safety.",
+ summary: "In our interview with Prof. Dr. Zoya Ignatova and Dr. Nikhil Bharti from the Institute of Biochemistry and Molecular Biology at the University of Hamburg, we aimed to deepen our understanding of their research on Cystic Fibrosis (CF) and explore additional mutations. Their innovative approaches, particularly tRNA-based therapies, aim to restore the function of proteins like CFTR by bypassing premature stop codons, presenting potential treatments for various genetic diseases. They highlighted the importance of precision in therapy to minimize side effects and enhance safety, emphasizing careful delivery systems to avoid toxicity. Prof. Ignatova's practical insights into cell culture techniques significantly improved our lab's success with CFBE41o- cells, guiding us to enhance sterilization protocols and refine our Prime Editing constructs. This transformative exchange underscored the critical balance between achieving therapeutic success and ensuring patient safety.",
language: "en",
interview: <>
<QaBox q="We have heard you are passionate about iGEM. What inspired you to get involved, and what has your experience been like with the competition?" a="My journey with iGEM began when I moved to Hamburg in 2014. Back then, Hamburg did not have its own iGEM team. Despite Hamburg lacking an iGEM presence, there were motivated students who were eager to establish a team. We started quite late with me as a principal instructor, around April, with the competition scheduled for October, so we had limited time. However, we managed to form a team and participate. Fortunately, we were successful in convincing the university administration to establish a steady support for the initiative, which ensured stable funding, including covering registration fees early on. This financial and logistical support gave the team the security to focus on their projects. Over the years, the Hamburg iGEM team has become a well-known and respected group at the university. It is a creative environment where students can push the boundaries of science through interesting and impactful projects. I moved on to other duties after several years of supervision, but I am proud to have played a role in its foundation. The university has recognized iGEM within the curriculum of Molecular Life Science, allowing students to earn credit points and have their work reflected on their transcripts. This acknowledgment further incentivizes students and ensures that their efforts are formally recognized." />
<QaBox q="We have been having trouble with CFBE41o- cells not adhering well. Any advice?" a="CFBE41o- cells can be tricky when it comes to adhesion, but you do not necessarily need to coat your cell culture vessels with fibronectin unless you are doing very specific studies, such as primary culture comparisons. For seeding, we simplify the process by skipping the PBS washing step. Instead, we seed the cells directly into DMEM supplemented with 10% fetal calf serum (FCS) and streptomycin. These cells may take a few days to recover and begin adhering properly, that usually works without requiring extra coatings." />
<QaBox q="How do you manage fungal contamination in ALI cultures?" a="Fungal contamination is one of the more frustrating challenges in cell culture because it is difficult to eliminate once it takes place. In cases of contamination, the best course of action is to shut down all ongoing cell culture work and clean everything thoroughly. You should start by running a sterilization cycle in your incubators, which ideally should reach around 180°C. This should kill any fungal spores. If your incubators do not have that capability, you will need to autoclave everything and clean all surfaces and equipment multiple times with ethanol. It is crucial to remove all traces of contamination, as fungal spores can spread rapidly. The key is prevention through rigorous cleaning and maintenance protocols, and unfortunately, sometimes the only solution is to start fresh with new cultures after a full decontamination round." />
- <QaBox q="What are you currently researching?" a="Our primary research focus is on genetic diseases caused by nonsense mutations, also known as premature termination codons (PTCs). While fibrosis (CF) is a major area of interest due to its high prevalence and the impact of specific mutations like the F508del, our research extends far beyond CF. We are targeting a broader category of genetic diseases that share a common feature—early stop codons that lead to production of truncated proteins, which are non-functional. In CF, for instance, our main goal is to restore full-length CFTR protein production in primary patient-derived cells bearing various PTCs. One approach we are exploring is known as a 'read-through' therapy, which involves bypassing the premature stop codon so that the cell can continue producing the full protein. This strategy is applicable not only to CF but can be used in many other genetic disorders caused by nonsense mutations. Briefly, the read-through therapies we develop are tRNA-based therapeutic approaches, in which we design suppressor transfer RNAs (sup-tRNAs) to selectively target and read through PTCs, restoring the production of full-length disease protein without altering the natural termination codons. It is a highly specific and safe method, and because we are targeting mRNA rather than DNA, it allows for terminating the therapies by any unforeseen side effects." />
+ <QaBox q="What are you currently researching?" a="Our primary research focus is on genetic diseases caused by nonsense mutations, also known as premature termination codons (PTCs). While Cystic Fibrosis (CF) is a major area of interest due to its high prevalence and the impact of specific mutations like the F508del, our research extends far beyond CF. We are targeting a broader category of genetic diseases that share a common feature—early stop codons that lead to production of truncated proteins, which are non-functional. In CF, for instance, our main goal is to restore full-length CFTR protein production in primary patient-derived cells bearing various PTCs. One approach we are exploring is known as a 'read-through' therapy, which involves bypassing the premature stop codon so that the cell can continue producing the full protein. This strategy is applicable not only to CF but can be used in many other genetic disorders caused by nonsense mutations. Briefly, the read-through therapies we develop are tRNA-based therapeutic approaches, in which we design suppressor transfer RNAs (sup-tRNAs) to selectively target and read through PTCs, restoring the production of full-length disease protein without altering the natural termination codons. It is a highly specific and safe method, and because we are targeting mRNA rather than DNA, it allows for terminating the therapies by any unforeseen side effects." />
<QaBox q="What are your downstream validation methods?" a="After we have developed a therapeutic approach, the first step is to validate whether it works at the protein level. First, we check whether the full-length protein is being produced. For CFTR, for example, we look at whether the protein is being correctly synthesized. We also conduct functional tests to ensure its functionality. For CFTR specifically, we test the activity of the ion channel by measuring ion flow through the cell membrane. Another test involves monitoring the height of the air-liquid interface (ALI) cultures, which reports on the ionic balance across the membrane. These functional tests are crucial for confirming that the therapy is not only leading to a production of the protein but is also restoring its function." />
<QaBox q="How often would patients need to undergo this therapy?" a="Since our approach is designed to correct nonsense mutations during translation the therapy would need to be administered periodically. Based on our current understanding, we anticipate that patients might need treatment every three to four weeks, but this has to be determined in clinical settings." />
<QaBox q="How does your tRNA-based approach address safety issues?" a="Safety is the top priority of our tRNA-based therapeutic approach. At molecular level, we ensure that the suppressor tRNAs we use are highly specific—they are engineered to target only PTCs without affecting natural stop codons, which are essential for terminating the synthesis of every protein. In addition to the specificity, we address the immune response that can be triggered by any nucleic acids, including tRNA. Generally, tRNA has a lower immunogenicity than other molecules, such as mRNA, because of its partially double-stranded structure, which reduces the activation of the innate immune reaction. Another critical safety aspect is the safety of the delivery system. We need to ensure that the tRNA reaches the right type of cells without causing toxicity or accumulating in untargeted tissues like the liver, which is a common issue with many gene therapies. We are also working on optimizing our delivery methods. This precision is key to minimizing side effects and ensuring the safety of our therapy." />
@@ -1856,8 +1856,8 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
heading: "Shaping CF Therapies: Lessons Learned from Patients' Real-World Experiences",
interviewtabid: "dino",
cardtext: "",
- quote: "Living with fibrosis has been a journey of perseverance and adaptation. Despite the challenges, I've learned that taking personal responsibility for my health, staying informed, and embracing the advancements in research can lead to a much-improved quality of life.",
- aimofcontact: [<p>Our goal in reaching out to fibrosis (CF) patients was to gain a firsthand understanding of their day-to-day experiences living with the condition. We aimed to explore how recent advancements in treatments have impacted their lives and transformed their approach to managing the disease. This initiative is vital for our project, as it helps us comprehend the significance of these treatments and their broader implications. A key aspect of our outreach was partnering with the <a href="https://www.instagram.com/accounts/login/?next=https%3A%2F%2Fwww.instagram.com%2Fmukodino%2F&is_from_rle" >MukoDino</a>
+ quote: "Living with Cystic Fibrosis has been a journey of perseverance and adaptation. Despite the challenges, I've learned that taking personal responsibility for my health, staying informed, and embracing the advancements in research can lead to a much-improved quality of life.",
+ aimofcontact: [<p>Our goal in reaching out to Cystic Fibrosis (CF) patients was to gain a firsthand understanding of their day-to-day experiences living with the condition. We aimed to explore how recent advancements in treatments have impacted their lives and transformed their approach to managing the disease. This initiative is vital for our project, as it helps us comprehend the significance of these treatments and their broader implications. A key aspect of our outreach was partnering with the <a href="https://www.instagram.com/accounts/login/?next=https%3A%2F%2Fwww.instagram.com%2Fmukodino%2F&is_from_rle" >MukoDino</a>
who has extensive reach within the CF community. This collaboration enabled us to distribute our survey widely among those affected, as we recognized that we could not conduct as many interviews as we could reach through the survey. We also sought to gather insights on research priorities and the future direction of CF therapies. </p>],
insights: [<p>From talking with the CF patient, we gained some valuable insights that have shaped how we move forward with our project:
Firstly, hearing about the day-to-day challenges with current CF treatments was eye-opening. It highlighted just how crucial it is for us to develop therapies that are not only effective but also make life easier for patients. We’ve taken this to heart and are working to make our treatment process as efficient and user-friendly as possible.
@@ -1872,27 +1872,27 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
The feedback on global disparities in CF care highlighted the importance of creating a treatment that is not only effective but also affordable and accessible. We’re considering how to scale our therapy to make it available in regions with limited access to advanced treatments.
Additionally, the emphasis on the need for continued advancements in enzyme development and gene therapy has guided us to balance immediate therapeutic benefits with long-term research goals. This ensures that our project addresses both current needs and future possibilities in CF treatment.
By integrating these insights, we aim to ensure our project not only advances scientific understanding but also meets the practical needs of CF patients, ultimately leading to more effective and accessible treatments. </p>],
- summary: "The aim of our outreach to fibrosis (CF) patients was to gain insights into their daily experiences and how recent treatment advancements have affected their lives. Partnering with MukoDino allowed us to distribute a survey widely, as interviews were limited. Through discussions with patients, we learned about the challenges posed by current treatments, emphasizing the need for therapies that simplify management and reduce physical strain. This feedback has driven our focus on developing more efficient inhalation therapies using spray-dried lipid nanoparticles (LNPs) to deliver gene-editing tools directly to the lungs. We also recognized the global disparities in CF care, motivating us to consider scalability and accessibility for those in resource-limited regions. Patient insights underscored the importance of personalized treatments tailored to individual genetic mutations, prompting us to prioritize enzyme development and gene therapy in our research. Overall, these insights have shaped our project to align closely with the real-world needs of CF patients, aiming for impactful and accessible solutions.",
+ summary: "The aim of our outreach to Cystic Fibrosis (CF) patients was to gain insights into their daily experiences and how recent treatment advancements have affected their lives. Partnering with MukoDino allowed us to distribute a survey widely, as interviews were limited. Through discussions with patients, we learned about the challenges posed by current treatments, emphasizing the need for therapies that simplify management and reduce physical strain. This feedback has driven our focus on developing more efficient inhalation therapies using spray-dried lipid nanoparticles (LNPs) to deliver gene-editing tools directly to the lungs. We also recognized the global disparities in CF care, motivating us to consider scalability and accessibility for those in resource-limited regions. Patient insights underscored the importance of personalized treatments tailored to individual genetic mutations, prompting us to prioritize enzyme development and gene therapy in our research. Overall, these insights have shaped our project to align closely with the real-world needs of CF patients, aiming for impactful and accessible solutions.",
months: "September",
interview: <>
- <QaBox q="How did you come up with the name ‘Muko-Dino’?" a="The name ‘Muko-Dino’ has a personal history. At a fibrosis meeting, another patient joked that I, as a 58-year-old fibrosis patient, was a ‘Muko-Dino’. The name alludes to my age, because in the past, fibrosis patients had a much shorter life expectancy. So it was unusual for someone with the disease to still be so active at my age. The name stuck - initially as a joke, but I have since adopted it as a kind of nickname." />
- <QaBox q="When were you diagnosed with fibrosis?" a="I was diagnosed at the age of six after a sweat test. I had lost a lot of weight in the first year of my life and fibrosis was already suspected, but it took a while before the diagnosis was actually confirmed." />
- <QaBox q="What were your thoughts and those of your family after the diagnosis?" a="My mum was naturally shocked. When you have a child, you are happy, and then to receive a diagnosis like this is a huge shock. For parents of fibrosis patients, there is always a basic fear because the disease is life-threatening. Today, many parents hope that their child will live to be at least two years old so that they can receive modulators that can greatly improve the symptoms." />
- <QaBox q="What mutation do you have?" a="I have the Delta 508 mutation in duplicate, i.e. homozygous. This is the most common form of fibrosis mutation and causes the typical symptoms associated with the disease, such as digestive and lung problems." />
- <QaBox q="What were the first steps after the diagnosis?" a="Back then, in the 1960s, there were hardly any specialised fibrosis outpatient clinics. My paediatrician prescribed me Pankreon, an early enzyme preparation that was supposed to help me with digestion. Nowadays, the process is very different: A child is referred to a specialised outpatient clinic immediately after diagnosis, where they receive comprehensive care. This includes medical care, nutritional counselling, physiotherapy and support with socio-legal issues." />
- <QaBox q="How has treatment changed over the years?" a="The treatment of fibrosis has improved dramatically over the last few decades. In the past, low-fat diets had to be followed, even with enzymes, which were not as effective as today's preparations. Back then, if you ate something fatty like chocolate or chips, you got diarrhoea. Nowadays, enzymes are much more effective and patients have hardly any dietary restrictions. As far as antibiotics are concerned, resistance is a big issue. That's why doctors change antibiotics regularly to prevent resistance from developing. Modulators have revolutionised the lives of many patients, even if they can trigger psychological side effects in some. But the alternative, frequent pneumonia, is much worse." />
- <QaBox q="How do you assess the progress made in fibrosis research?" a="The progress is absolutely remarkable. When I was born, the average life expectancy of a fibrosis patient was one to five years. Today we are talking about over 60 years. This development is not only due to medical innovations, but also to better adherence on the part of patients, who follow their therapies more regularly and efficiently." />
- <QaBox q="Is there anything you would like to see in the future of therapy?" a="Personally, I am almost perfectly happy, as the current modulators have given me an enormously improved quality of life. My wish for the entire fibrosis community is that gene therapy will be further developed. A treatment that addresses the genetic causes of the disease would be a huge breakthrough and it would be ideal if this could be achieved without serious side effects." />
+ <QaBox q="How did you come up with the name ‘Muko-Dino’?" a="The name ‘Muko-Dino’ has a personal history. At a Cystic Fibrosis meeting, another patient joked that I, as a 58-year-old Cystic Fibrosis patient, was a ‘Muko-Dino’. The name alludes to my age, because in the past, Cystic Fibrosis patients had a much shorter life expectancy. So it was unusual for someone with the disease to still be so active at my age. The name stuck - initially as a joke, but I have since adopted it as a kind of nickname." />
+ <QaBox q="When were you diagnosed with Cystic Fibrosis?" a="I was diagnosed at the age of six after a sweat test. I had lost a lot of weight in the first year of my life and Cystic Fibrosis was already suspected, but it took a while before the diagnosis was actually confirmed." />
+ <QaBox q="What were your thoughts and those of your family after the diagnosis?" a="My mum was naturally shocked. When you have a child, you are happy, and then to receive a diagnosis like this is a huge shock. For parents of Cystic Fibrosis patients, there is always a basic fear because the disease is life-threatening. Today, many parents hope that their child will live to be at least two years old so that they can receive modulators that can greatly improve the symptoms." />
+ <QaBox q="What mutation do you have?" a="I have the Delta 508 mutation in duplicate, i.e. homozygous. This is the most common form of Cystic Fibrosis mutation and causes the typical symptoms associated with the disease, such as digestive and lung problems." />
+ <QaBox q="What were the first steps after the diagnosis?" a="Back then, in the 1960s, there were hardly any specialised Cystic Fibrosis outpatient clinics. My paediatrician prescribed me Pankreon, an early enzyme preparation that was supposed to help me with digestion. Nowadays, the process is very different: A child is referred to a specialised outpatient clinic immediately after diagnosis, where they receive comprehensive care. This includes medical care, nutritional counselling, physiotherapy and support with socio-legal issues." />
+ <QaBox q="How has treatment changed over the years?" a="The treatment of Cystic Fibrosis has improved dramatically over the last few decades. In the past, low-fat diets had to be followed, even with enzymes, which were not as effective as today's preparations. Back then, if you ate something fatty like chocolate or chips, you got diarrhoea. Nowadays, enzymes are much more effective and patients have hardly any dietary restrictions. As far as antibiotics are concerned, resistance is a big issue. That's why doctors change antibiotics regularly to prevent resistance from developing. Modulators have revolutionised the lives of many patients, even if they can trigger psychological side effects in some. But the alternative, frequent pneumonia, is much worse." />
+ <QaBox q="How do you assess the progress made in Cystic Fibrosis research?" a="The progress is absolutely remarkable. When I was born, the average life expectancy of a Cystic Fibrosis patient was one to five years. Today we are talking about over 60 years. This development is not only due to medical innovations, but also to better adherence on the part of patients, who follow their therapies more regularly and efficiently." />
+ <QaBox q="Is there anything you would like to see in the future of therapy?" a="Personally, I am almost perfectly happy, as the current modulators have given me an enormously improved quality of life. My wish for the entire Cystic Fibrosis community is that gene therapy will be further developed. A treatment that addresses the genetic causes of the disease would be a huge breakthrough and it would be ideal if this could be achieved without serious side effects." />
<QaBox q="Which areas of research do you think should be strengthened?" a="There are a few areas that I consider to be particularly important. On the one hand, enzymes should be further developed in order to work even better and further minimise side effects. Another major topic is bacteriophage research, which could help patients with multi-resistant germs. Modulators should also be further optimised and, of course, gene therapy must be driven forward in order to find a sustainable solution." />
- <QaBox q="What role does personal responsibility play in fibrosis?" a="Personal responsibility is extremely important. Anyone living with fibrosis has to deal intensively with their own disease and take responsibility for their health. This means regularly informing yourself about new treatment options and being disciplined about your own treatment. If you don't take care of yourself, you run the risk of not fully utilising the advances in medicine. The healthcare system gives you many options, but you have to actively utilise them. Luck definitely plays a role, but it is not enough on its own. I have been lucky in my medical history because I live in a country with good access to medical care and have been able to benefit from the modulators. But even the best luck doesn't help without initiative and commitment. You have to play an active role in making the most of the opportunities that life offers you." />
+ <QaBox q="What role does personal responsibility play in Cystic Fibrosis?" a="Personal responsibility is extremely important. Anyone living with Cystic Fibrosis has to deal intensively with their own disease and take responsibility for their health. This means regularly informing yourself about new treatment options and being disciplined about your own treatment. If you don't take care of yourself, you run the risk of not fully utilising the advances in medicine. The healthcare system gives you many options, but you have to actively utilise them. Luck definitely plays a role, but it is not enough on its own. I have been lucky in my medical history because I live in a country with good access to medical care and have been able to benefit from the modulators. But even the best luck doesn't help without initiative and commitment. You have to play an active role in making the most of the opportunities that life offers you." />
<QaBox q="What does your daily routine look like?" a="My daily therapy takes about two to three hours. This includes taking enzymes and antibiotics as well as inhalations with saline solution and antibiotics. I also do regular autogenic drainage and stretching exercises to support my lungs. It is important to stick to this routine consistently, as even a few days without therapy can lead to inflammation." />
<QaBox q="What is the most important part of your daily routine?" a="It's difficult to single out one part because all aspects work together. If I left out the enzymes, I would only be able to eat salad because my body wouldn't be able to absorb any nutrients. Without the modulators and antibiotics, I would quickly get pneumonia, which means I would have to take weeks off. So regularity is the key to staying healthy in the long term." />
<QaBox q="Do you do any sporting activities?" a="Yes, I cycle to work every day and take the stairs to the eighth floor where I have my office. I don't do intensive sports like going to the gym, but I do exercise regularly in my everyday life, which is very important for my health." />
- <QaBox q="Do you use physiotherapy?" a="I do my own physiotherapy at home as I have learnt the techniques well over the years. I used to visit a physiotherapist regularly, but I no longer need to. Also, there are hardly any specialised physiotherapists for fibrosis patients in my area." />
+ <QaBox q="Do you use physiotherapy?" a="I do my own physiotherapy at home as I have learnt the techniques well over the years. I used to visit a physiotherapist regularly, but I no longer need to. Also, there are hardly any specialised physiotherapists for Cystic Fibrosis patients in my area." />
<QaBox q="Which organ affects you the most in your everyday life?" a="Thanks to the modulators, none actually. But if I don't take the enzymes, I can't eat anything, which leads to rapid weight loss. Without the antibiotics and modulators, I would be more susceptible to infections and would quickly develop pneumonia, which would require weeks of recovery." />
<QaBox q="Have you or anyone close to you taken part in clinical trials?" a="I personally have not participated in clinical trials, but I encourage other patients to do so, as trials are often the only way to test and develop new treatments." />
- <QaBox q="What impact do advances in fibrosis research have on patients' lives?" a="Advances in fibrosis research have had an enormous positive impact on patients' lives. In the past, life expectancy at diagnosis was often only a few years. Today, many patients can live into old age. The introduction of modulators that specifically stabilise the disease-causing proteins and improve their function has significantly improved patients' quality of life. Research has also contributed to patients being better informed about their disease and having effective therapies available, leading to better overall disease management." />
- <QaBox q="How do you assess the differences in the treatment and support of fibrosis patients in Germany compared to other countries?" a="In Europe, especially in Germany, we live at a very high medical level, even if there are challenges such as the shortage of specialists. The difference in prosperity in the USA is greater, but at the same time it is also a driver of innovation. Compared to countries as some third-world countries, where fibrosis patients have to wait in hospitals for appointments between infectious patients, we are complaining at a high level here. There is always room for improvement, but overall we have a brilliant healthcare system." />
+ <QaBox q="What impact do advances in Cystic Fibrosis research have on patients' lives?" a="Advances in Cystic Fibrosis research have had an enormous positive impact on patients' lives. In the past, life expectancy at diagnosis was often only a few years. Today, many patients can live into old age. The introduction of modulators that specifically stabilise the disease-causing proteins and improve their function has significantly improved patients' quality of life. Research has also contributed to patients being better informed about their disease and having effective therapies available, leading to better overall disease management." />
+ <QaBox q="How do you assess the differences in the treatment and support of Cystic Fibrosis patients in Germany compared to other countries?" a="In Europe, especially in Germany, we live at a very high medical level, even if there are challenges such as the shortage of specialists. The difference in prosperity in the USA is greater, but at the same time it is also a driver of innovation. Compared to countries as some third-world countries, where Cystic Fibrosis patients have to wait in hospitals for appointments between infectious patients, we are complaining at a high level here. There is always room for improvement, but overall we have a brilliant healthcare system." />
<QaBox q="Do you feel sufficiently informed by the available sources of information?" a="Yes, the information situation is good. You can find sufficient information from organisations such as the CF Foundation, the CF Trust and Mukoviszidose e.V.. However, you have to get involved yourself and actively seek out the information." />
<QaBox q="Is there anything else you would like to tell us?" a="It is important to understand that CF patients are very different, including in terms of their cultural and family background. In addition, surveys and questions should be reviewed in advance by patients or parents to ensure that they are understandable and do not contain unfortunate wording. If you need any help or anything, I would love to help you. Send me the link as soon as the website is ready and I'll give you feedback. I can also circulate the survey in the community. I'm always available if you have any further questions or need support." />
</>,
@@ -1913,7 +1913,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
quote: "Many families are confronted with extremely high therapy and treatment costs. These are often expensive and unavailable everywhere.",
quoteVorname: "Elena",
quoteNachname: "Wiesler, Phsychotherapist at hospital Bielefeld ",
- aimofcontact: [<p>In previous interviews, <HPLinktoOtherHPTab tab="maxfirst" text="Max" /> and <HPLinktoOtherHPTab tab="dino" text="Thomas" /> shared how the psychological burden of living with fibrosis weighs heavily on patients. Parents <HPLinktoOtherHPTab tab="joshua" text="Joshua" /> and <HPLinktoOtherHPTab tab="julia" text="Julia" /> also emphasized that mental health challenges are a major issue for both patients and their families. This prompted us to delve deeper into the psychological, social, and medical difficulties faced by fibrosis (CF) patients and their support systems. A key goal was to understand how gene therapies are perceived and how they may affect the quality of life for CF patients. We aimed to gather insights from various perspectives—patients, caregivers, and healthcare professionals—to ensure our project aligns with their needs and addresses the most pressing challenges.
+ aimofcontact: [<p>In previous interviews, <HPLinktoOtherHPTab tab="maxfirst" text="Max" /> and <HPLinktoOtherHPTab tab="dino" text="Thomas" /> shared how the psychological burden of living with Cystic Fibrosis weighs heavily on patients. Parents <HPLinktoOtherHPTab tab="joshua" text="Joshua" /> and <HPLinktoOtherHPTab tab="julia" text="Julia" /> also emphasized that mental health challenges are a major issue for both patients and their families. This prompted us to delve deeper into the psychological, social, and medical difficulties faced by Cystic Fibrosis (CF) patients and their support systems. A key goal was to understand how gene therapies are perceived and how they may affect the quality of life for CF patients. We aimed to gather insights from various perspectives—patients, caregivers, and healthcare professionals—to ensure our project aligns with their needs and addresses the most pressing challenges.
Given the complexity of these psychological aspects, it was crucial for us to engage with psychologists to gain a professional, expert opinion. We visited the medical professionals at the klinikum bethel. Their input helped us better understand the mental health impacts of CF and the potential emotional adjustments required when integrating gene therapies into treatment plans. This guidance was invaluable in shaping our approach to developing a holistic solution that addresses not only the medical needs but also the emotional well-being of patients and their families. It informed our Integrated Health Program (IHP) strategy, emphasizing the importance of interpersonal relationships, effective communication, and community engagement, extending beyond purely scientific considerations. </p>],
insights: [<p>Through our discussions, several valuable insights emerged that have significantly deepened our understanding of the challenges faced by CF patients and their families:
CF patients and their families often endure immense psychological strain. Anxiety, depression, and frustration are common, exacerbated by the constant uncertainty about the disease’s progression and the effectiveness of new treatments. The emotional toll is profound—not just due to the physical burden of the illness, but also because of the hope and fear that come with emerging therapies. While new treatments bring promise, they also raise concerns about their potential success and the unknowns that accompany them.
@@ -1923,12 +1923,12 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
These insights have shaped our approach, reinforcing the importance of considering both the psychological and practical aspects of new gene therapies, to better address the needs of CF patients and their families. </p>],
implementation: [<p>These findings directly influenced several key areas of the project. We adapted the project to emphasise ease of use and minimal disruption to patients' daily lives. For example, we focused on developing a therapy delivery system that was as non-invasive as possible. Recognising the mental health challenges, we integrated our project with a simple therapeutic method to reduce the mental burden on patients. We have emphasised transparency in communicating the benefits, risks and expectations of gene therapy to ensure that patients have a realistic understanding of the potential outcomes. This includes working closely with patient organisations to disseminate clear and accurate information. We are actively engaging with CF patient communities and healthcare professionals to gather ongoing feedback and ensure that the project evolves based on real patient experiences and challenges. Therefore we used our survey to gather feedback from patients and their families.
By integrating these insights, we aim to create a gene therapy project that addresses not only the medical needs, but also the emotional and practical concerns of CF patients and their families. </p>],
- summary: "Our project aims to address the psychological and medical challenges faced by fibrosis (CF) patients and their families, particularly regarding gene therapies. We engaged with psychologists and gathered insights from patients and caregivers, revealing significant emotional strain and a mix of optimism and concern about new treatments. Key findings highlighted the importance of psychological support and the practicality of therapies in ensuring patient engagement. In response, we are developing a user-friendly therapy delivery system that minimizes disruption to daily life while emphasizing transparent communication about treatment risks and benefits. Our goal is to create a comprehensive gene therapy solution that meets the medical and emotional needs of CF patients.",
+ summary: "Our project aims to address the psychological and medical challenges faced by Cystic Fibrosis (CF) patients and their families, particularly regarding gene therapies. We engaged with psychologists and gathered insights from patients and caregivers, revealing significant emotional strain and a mix of optimism and concern about new treatments. Key findings highlighted the importance of psychological support and the practicality of therapies in ensuring patient engagement. In response, we are developing a user-friendly therapy delivery system that minimizes disruption to daily life while emphasizing transparent communication about treatment risks and benefits. Our goal is to create a comprehensive gene therapy solution that meets the medical and emotional needs of CF patients.",
months: "September",
interview:<>
- <QaBox q="Which psychological challenges are particularly relevant for fibrosis patients?" a="Psychological problems are often a major issue for fibrosis patients. Many patients experience anxiety and depression, and their parents are also often affected. This is exacerbated by the constant strain and stress associated with the disease. Special attention is therefore paid to psychological support during diagnosis and ongoing treatment. Regular screenings for anxiety and depression as well as the early involvement of parents in the treatment process are central components of care."/>
- <QaBox q="What significance do the new therapies have for fibrosis patients?" a="New therapies are ‘game changers’ for fibrosis patients, as they significantly improve life expectancy and quality of life. In the past, fibrosis was mainly a paediatric disease with a short life expectancy. Today, new therapies make it possible to significantly extend life expectancy and improve quality of life. Nevertheless, the disease persists, and patients still require comprehensive treatment. Improving quality of life through early and continuous therapy therefore remains of great importance."/>
- <QaBox q="How is psychological support integrated into regular treatment?" a="Psychological support is an integral part of the treatment of fibrosis. Care is taken to ensure that both patients and their families are supported at an early stage. This includes regular screenings for anxiety and depression, psycho-educational measures and, if necessary, further psychotherapeutic support. The team works on an interdisciplinary basis to ensure that all aspects of patient care are taken into account. If necessary, external help is also arranged."/>
+ <QaBox q="Which psychological challenges are particularly relevant for Cystic Fibrosis patients?" a="Psychological problems are often a major issue for Cystic Fibrosis patients. Many patients experience anxiety and depression, and their parents are also often affected. This is exacerbated by the constant strain and stress associated with the disease. Special attention is therefore paid to psychological support during diagnosis and ongoing treatment. Regular screenings for anxiety and depression as well as the early involvement of parents in the treatment process are central components of care."/>
+ <QaBox q="What significance do the new therapies have for Cystic Fibrosis patients?" a="New therapies are ‘game changers’ for Cystic Fibrosis patients, as they significantly improve life expectancy and quality of life. In the past, Cystic Fibrosis was mainly a paediatric disease with a short life expectancy. Today, new therapies make it possible to significantly extend life expectancy and improve quality of life. Nevertheless, the disease persists, and patients still require comprehensive treatment. Improving quality of life through early and continuous therapy therefore remains of great importance."/>
+ <QaBox q="How is psychological support integrated into regular treatment?" a="Psychological support is an integral part of the treatment of Cystic Fibrosis. Care is taken to ensure that both patients and their families are supported at an early stage. This includes regular screenings for anxiety and depression, psycho-educational measures and, if necessary, further psychotherapeutic support. The team works on an interdisciplinary basis to ensure that all aspects of patient care are taken into account. If necessary, external help is also arranged."/>
<QaBox q="How is co-operation between medical specialists and psychologists improved?" a="The collaboration between medical specialists and psychologists is characterised by short communication channels and close cooperation. Specialists can exchange information quickly and make decisions together. This enables comprehensive and coordinated care for patients. Effective communication channels are already in place and this close co-operation is seen as very positive. Improvements could be achieved through additional time slots for dialogue or expanded resources."/>
<QaBox q="How do families react to the news of a serious diagnosis and how important is it that they receive support at an early stage?" a="Families are often shocked at first when they receive the diagnosis. They first have to come to terms with it and process it. Initially, many don't ask for psychological support straight away, although that would be helpful. It would be good if they were informed about all available resources at an early stage, even if they don't want to make use of them straight away."/>
<QaBox q="How does access to gene therapy affect the psychological distress of patients and families?" a="Access to gene therapy can have a significant impact on psychological distress. When therapy is effective, families often see great progress and feel relieved. But if there is no suitable therapy, many are stuck with older, less effective treatments, which can lead to frustration and a sense of disadvantage. The difference in quality of life and outlook is huge."/>
@@ -1938,16 +1938,16 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<QaBox q="How does the role of support groups and patient organisations influence confidence in new therapies?" a="Support groups and patient organisations are crucial for confidence in new therapies. If they are actively involved in research and provide transparent information, this strengthens patient confidence. The use of donations and the establishment of registries by such organisations creates trust and shows that there are serious efforts to improve the situation."/>
<QaBox q="What are the challenges in adapting therapies to different genetic mutations?" a="Adapting therapies to different genetic mutations is a major challenge. While there has been progress in the treatment of certain mutations such as Delta-F508, we are still at the beginning with others. In the long term, a modular gene therapy that is customised to the specific mutations would be ideal. It will take a lot of work to develop these therapies for all relevant mutations."/>
<QaBox q="How do families and patients deal with the rapid feedback on experimental therapies?" a="If you realise that a therapy is not working as expected, this is communicated very quickly. The feedback system is quite effective: either there is cause for euphoria because everything is going well, or there is bad news. This quick feedback is also reassuring because it means you don't have to be in the dark for long. You are simply grateful when you know how the therapy is going, even if it is not having the desired effect."/>
- <QaBox q="How important is the community for fibrosis sufferers?" a="The fibrosis community is incredibly strong and well connected. That's really impressive. Those affected often have no other point of contact than this community to exchange information. It's a reliable source of valid information, and that's worth its weight in gold. The community is honest and realistic - there is no sugarcoating, the information is direct and well documented."/>
- <QaBox q="What is the relationship between different specialist disciplines in the treatment of fibrosis?" a="In fibrosis treatment, the specialist disciplines work together as equals. At congresses, all disciplines such as physios, doctors, psychologists and nutritionists are equally represented. Everyone takes each other seriously and there is a strong interest in developing each other further. This is really exciting because it shows that everyone is working together to provide the best care."/>
+ <QaBox q="How important is the community for Cystic Fibrosis sufferers?" a="The Cystic Fibrosis community is incredibly strong and well connected. That's really impressive. Those affected often have no other point of contact than this community to exchange information. It's a reliable source of valid information, and that's worth its weight in gold. The community is honest and realistic - there is no sugarcoating, the information is direct and well documented."/>
+ <QaBox q="What is the relationship between different specialist disciplines in the treatment of Cystic Fibrosis?" a="In Cystic Fibrosis treatment, the specialist disciplines work together as equals. At congresses, all disciplines such as physios, doctors, psychologists and nutritionists are equally represented. Everyone takes each other seriously and there is a strong interest in developing each other further. This is really exciting because it shows that everyone is working together to provide the best care."/>
<QaBox q="How do families deal with the challenges of therapy and the financial aspects?" a="Many families are confronted with extremely high therapy and treatment costs. There has been progress and many treatment options in Germany, but these are often expensive and not available everywhere. As a result, some families are forced to leave their home country in order to receive better medical care. This is an enormous burden and shows how unfair the distribution of resources is worldwide."/>
<QaBox q="How is medical care for refugees organised?" a="For refugees from countries such as Ukraine or other crisis areas, care is often a challenge. During emergency care, the children are treated as if they were German patients. But when the refugees have to return to their home countries, the therapy often ends, which is an enormous burden for the families. It is difficult for them to prepare for the future when their status is unclear, and they constantly live with the fear of being deported."/>
<QaBox q="How much psychological stress is caused by therapies and their implementation?" a="Therapies can be a major psychological burden, even if they have fundamentally positive effects. Regular inhalations, tablets and other treatments are often tedious and require a lot of discipline. Some patients find it extremely challenging to stick to a regular therapy schedule, especially if the therapy does not bring any immediately visible progress in the long term. It is important to be realistic about the burden of therapy, as it can have a major impact on daily life and well-being."/>
<QaBox q="How do patients react to new therapies and the associated challenges?" a="Many patients are open to new therapies but implementing them can be a major challenge. If a new therapy doesn't work immediately at first or even has side effects, this can be demotivating. This is particularly difficult if you have been undergoing treatment for a long time and are hoping to make great progress. The path to a better condition is often arduous and not every therapy brings the desired improvement. Nevertheless, it is important to keep going and persevere with the therapy, even if there are hard times."/>
<QaBox q="How much of the overall illness is psychological distress, in addition to the physical symptoms and distress from therapies?" a="The psychological part of the burden is difficult to quantify, as it varies greatly from individual to individual and is influenced by many factors. The interaction between psychological stability and physical health is considerable, as psychological stress can impair self-care and thus physical health. At different stages of life, the psychological component can vary. For example, it can increase during puberty and young adulthood. The psychological component is therefore not small and varies depending on the individual situation and phase of life."/>
- <QaBox q="How is the visibility of the disease assessed through projects such as MukoMove or projects for children?" a="The visibility of the disease through such projects can be helpful in raising awareness. With rare diseases such as fibrosis, the disease often remains abstract if there are no people directly affected nearby. Educational projects such as MukoMove can help children develop a better understanding of the disease, even if the impact is limited if there are no direct points of reference. However, it can be helpful if patients themselves explain their disease in schools or classes, as this provides direct and personal insights."/>
- <QaBox q="What are important aspects of designing a gene therapy project so that it is viewed positively by fibrosis patients?" a="When designing a gene therapy project, care should be taken to minimise the practical hurdles. The therapy"/>
- <QaBox q="What tips can be given to improve the accessibility and acceptance of projects or therapies in fibrosis patients? " a="It is important to ensure the accessibility of projects and that they are practical to implement. The burden on patients should be minimised. This includes ensuring that the therapy is not only effective but also as pleasant as possible. In addition, communication about the progress of the therapy should be transparent and understandable to build trust and make it clear to patients how they can benefit from the new developments. "/>
+ <QaBox q="How is the visibility of the disease assessed through projects such as MukoMove or projects for children?" a="The visibility of the disease through such projects can be helpful in raising awareness. With rare diseases such as Cystic Fibrosis, the disease often remains abstract if there are no people directly affected nearby. Educational projects such as MukoMove can help children develop a better understanding of the disease, even if the impact is limited if there are no direct points of reference. However, it can be helpful if patients themselves explain their disease in schools or classes, as this provides direct and personal insights."/>
+ <QaBox q="What are important aspects of designing a gene therapy project so that it is viewed positively by Cystic Fibrosis patients?" a="When designing a gene therapy project, care should be taken to minimise the practical hurdles. The therapy"/>
+ <QaBox q="What tips can be given to improve the accessibility and acceptance of projects or therapies in Cystic Fibrosis patients? " a="It is important to ensure the accessibility of projects and that they are practical to implement. The burden on patients should be minimised. This includes ensuring that the therapy is not only effective but also as pleasant as possible. In addition, communication about the progress of the therapy should be transparent and understandable to build trust and make it clear to patients how they can benefit from the new developments. "/>
</>,
pictureurl_aim:"https://static.igem.wiki/teams/5247/integrated-human-practices/on-our-way-to-interview-psychologists.webp",
},
@@ -1975,7 +1975,7 @@ export const timelinedata: Array<TimelineDatenpunkt> = [
<QaBox q="Are you familiar with iGEM, by the way?" a="Of course, I know it."/>
<QaBox q="Did you participate yourself at some point?" a="Unfortunately, I didn't. I belong to an earlier generation. iGEM actually started relatively recently."/>
<QaBox q="We thought for the structure of the interview, we would start by giving you a brief overview of our project so far to familiarize you with it. Then, we’ll move on to the questions. Is that okay?" a="Yes, of course, please go ahead."/>
- <QaBox q="We've been working on this project for more than half a year now. It began because one of our team members has a friend with fibrosis. That got us interested in the topic. We started by investigating how gene editing technologies like CRISPR-Cas9 could be applied to fibrosis. Then, we explored prime editing and considered if it could be used for this disease or adapted for other applications. Initially, we wondered if we could make prime editing more compact, especially since delivery is challenging due to its large complex size. We looked into various delivery methods, including AAVs (Adeno-Associated Viruses). Our first approach was to explore alternative nickases and possibly engineer new ones. That's how we came across your research – Fanzor. We also considered other candidates like CasX. Are you familiar with CasX?" a="Yes, I am."/>
+ <QaBox q="We've been working on this project for more than half a year now. It began because one of our team members has a friend with Cystic Fibrosis. That got us interested in the topic. We started by investigating how gene editing technologies like CRISPR-Cas9 could be applied to Cystic Fibrosis. Then, we explored prime editing and considered if it could be used for this disease or adapted for other applications. Initially, we wondered if we could make prime editing more compact, especially since delivery is challenging due to its large complex size. We looked into various delivery methods, including AAVs (Adeno-Associated Viruses). Our first approach was to explore alternative nickases and possibly engineer new ones. That's how we came across your research – Fanzor. We also considered other candidates like CasX. Are you familiar with CasX?" a="Yes, I am."/>
<QaBox q="We're also experimenting with changes to the editing complex itself. In addition, we aim to deliver the editing complex using nanoparticles. We chose to focus on the lungs, hoping that targeting this area would reduce the need for AAV viruses, making the delivery less immunogenic and not as limited by size." a="So, in this iGEM project, you're working on both reducing the size of the prime editor and developing nanoparticles for delivery?"/>
<QaBox q="Yes, that’s the plan. Before we start with the main questions, how much time do you have? Is half an hour okay?" a="No problem, half an hour is fine."/>
<QaBox q="Great! Then, let’s start with the first question. Our approach to modifying the endonuclease FANZOR started with understanding its mechanism. Could we go over this mechanism with you to ensure we understood it correctly?" a="Of course, please go ahead."/>
diff --git a/src/sources/description-sources.tsx b/src/sources/description-sources.tsx
index 291bcec5cc93a80d6057c829e006bb1a239a97a2..ff3aaedf13f535c75d2657d98017bd489b3de714 100644
--- a/src/sources/description-sources.tsx
+++ b/src/sources/description-sources.tsx
@@ -14,7 +14,7 @@ const bibtexSources = [
author={Rodrigues, Roberta and Gabetta, Carmen S. and Pedro, Karla P. and Valdetaro, Fabioand Fernandes, Maria I. M. and Magalhães, PatrÃcia K. R. and Januário, José N., Maciel, Léa M. Z.},
year={2008},
pages={475-484},
- title={Cystic fibrosis and neonatal screening},
+ title={Cystic Cystic Fibrosis and neonatal screening},
volume={24},
journal={Cadernos de Saúde Pública},
doi={10.1590/S0102-311X2008001600002}
@@ -52,7 +52,7 @@ const bibtexSources = [
author={Bobadilla, J. L. and Macke Jr, M. and Fine, J. P. and Farrell, P. M.},
year={2002},
pages={575-606},
- title={Cystic fibrosis: A worldwide analysis of CFTR mutations - correlation with incidence data and application to screening},
+ title={Cystic Cystic Fibrosis: A worldwide analysis of CFTR mutations - correlation with incidence data and application to screening},
volume={19(6)},
journal={Human Mutation},
doi={10.1002/humu.10041}
@@ -70,9 +70,9 @@ const bibtexSources = [
`@misc{arena2024internet,
author={Clinical Trials Arena},
- title={Cystic fibrosis: global clinical trials landscape and treatments},
+ title={Cystic Cystic Fibrosis: global clinical trials landscape and treatments},
year={2024},
- url={https://www.clinicaltrialsarena.com/sponsored/-fibrosis-global-clinical-trials-landscape-and-treatments/},
+ url={https://www.clinicaltrialsarena.com/sponsored/-Cystic Fibrosis-global-clinical-trials-landscape-and-treatments/},
note={Zugriff am 23. September 2024}
}`,
@@ -101,7 +101,7 @@ const bibtexSources = [
}`,
`@article{Elborn2016,
- title={Cystic fibrosis},
+ title={Cystic Cystic Fibrosis},
volume={388},
ISSN={0140-6736},
url={http://dx.doi.org/10.1016/s0140-6736(16)00576-6},
@@ -140,7 +140,7 @@ const bibtexSources = [
}`,
/* 14 */
`@article{PUCHELLE2002115,
- title={Airway mucus in fibrosis},
+ title={Airway mucus in Cystic Fibrosis},
journal={Paediatric Respiratory Reviews},
volume={3},
number={2},
@@ -150,7 +150,7 @@ const bibtexSources = [
doi={https://doi.org/10.1016/S1526-0550(02)00005-7},
url={https://www.sciencedirect.com/science/article/pii/S1526055002000057},
author={Puchelle, Edith and Bajolet, Odile and Abély, Michel},
- keywords={ fibrosis, airway mucus, airway surface liquid, rheology, mucociliary clearance, mucus cough transport}
+ keywords={ Cystic Fibrosis, airway mucus, airway surface liquid, rheology, mucociliary clearance, mucus cough transport}
}`, /* 15 */
`@article{Anzalone2019,
author = {Anzalone, Andrew and Randolph, Peyton and Davis, Jessie and Sousa, Ayalur and Koblan, Luke and Levy, Jonathan and Chen, Patrick and Wilson, Charlotte and Newby, Greg and Raguram, Aditya and Liu, David},
@@ -175,7 +175,7 @@ const bibtexSources = [
,
`@article{Gadsby2006,
- title={The ABC protein turned chloride channel whose failure causes fibrosis},
+ title={The ABC protein turned chloride channel whose failure causes Cystic Fibrosis},
author={Gadsby, David C. and Vergani, Paola and Csanády, L{\'a}szl{\'o}},
@@ -328,7 +328,7 @@ month={ },
pages={475-484},
-title={Cystic fibrosis and neonatal screening},
+title={Cystic Cystic Fibrosis and neonatal screening},
volume={24},
@@ -420,7 +420,7 @@ day = {20},
@article{Pozo2020,
- title = {Cystic fibrosis-related diabetes: The unmet need},
+ title = {Cystic Cystic Fibrosis-related diabetes: The unmet need},
volume = {11},
@@ -497,9 +497,9 @@ day = {20},
`@misc{CFT22024internet,
author = {Cystic Fibrosis Trust},
- title = {How does fibrosis affect the body?},
+ title = {How does Cystic Fibrosis affect the body?},
year = {2024},
- url = {https://www.fibrosis.org.uk/what-is--fibrosis/how-does--fibrosis-affect-the-body​},
+ url = {https://www.Cystic Fibrosis.org.uk/what-is--Cystic Fibrosis/how-does--Cystic Fibrosis-affect-the-body​},
note = {Zugriff am 25. September 2024}
} `,
@@ -538,7 +538,7 @@ day = {20},
} `,
`@article{Dodge_1995,
-title={Male fertility in fibrosis},
+title={Male fertility in Cystic Fibrosis},
volume={346},
@@ -566,9 +566,9 @@ pages={587–588}
`@article{Davis_2006, title={Cystic Fibrosis Since 1938}, volume={173}, ISSN={1535-4970}, url={http://dx.doi.org/10.1164/rccm.200505-840oe}, DOI={10.1164/rccm.200505-840oe}, number={5}, journal={American Journal of Respiratory and Critical Care Medicine}, publisher={American Thoracic Society}, author={Davis, Pamela B.}, year={2006}, month=mar, pages={475–482} } `,
`@misc{CFT2024internet,
author = {Cystic Fibrosis Trust},
- title = {Fertility and fibrosis},
+ title = {Fertility and Cystic Fibrosis},
year = {2024},
- url = {https://www.fibrosis.org.uk/what-is--fibrosis/how-does--fibrosis-affect-the-body/symptoms-of--fibrosis/fertility ​},
+ url = {https://www.Cystic Fibrosis.org.uk/what-is--Cystic Fibrosis/how-does--Cystic Fibrosis-affect-the-body/symptoms-of--Cystic Fibrosis/fertility ​},
note = {Zugriff am 25. September 2024}
} `,
`@misc{SGW2024internet,
@@ -589,7 +589,7 @@ pages={587–588}
author = {Cystic Fibrosis Foundation},
title = {About Cystic Fibrosis},
year = {2024},
- url = {https://www.cff.org/intro-cf/about--fibrosis​},
+ url = {https://www.cff.org/intro-cf/about--Cystic Fibrosis​},
note = {Zugriff am 25. September 2024}
} `,
`@article{Rowe2005,
@@ -619,7 +619,7 @@ pages={587–588}
pages = {1992–2001}
} `,
-`@article{O_Sullivan_2009, title={Cystic fibrosis}, volume={373}, ISSN={0140-6736}, url={http://dx.doi.org/10.1016/s0140-6736(09)60327-5}, DOI={10.1016/s0140-6736(09)60327-5}, number={9678}, journal={The Lancet}, publisher={Elsevier BV}, author={O’Sullivan, Brian P and Freedman, Steven D}, year={2009}, month=may, pages={1891–1904} } `,
+`@article{O_Sullivan_2009, title={Cystic Cystic Fibrosis}, volume={373}, ISSN={0140-6736}, url={http://dx.doi.org/10.1016/s0140-6736(09)60327-5}, DOI={10.1016/s0140-6736(09)60327-5}, number={9678}, journal={The Lancet}, publisher={Elsevier BV}, author={O’Sullivan, Brian P and Freedman, Steven D}, year={2009}, month=may, pages={1891–1904} } `,
`@misc{Muko62024internet,
author = {Lungen Informationsdienst},
title = {Mukoviszidose: Medikamente und Behandlung},
@@ -654,7 +654,7 @@ doi = {10.3238/arztebl.2017.0564},
year = {2017},
-abstract = {Background: Universal screening of newborn babies for fibrosis was launched in Germany on 1 September 2016. Here we present up-to-date information on the diagnosis, treatment, and prognosis of this disease.Methods: This article is based on relevant publications retrieved by a selective search in PubMed, along with guidelines from Germany and abroad and systematic reviews.Results: Cystic fibrosis is caused by a gene mutation leading to dysfunction of the fibrosis transmembrane conductance regulator (CFTR) protein. It affects multiple organ systems—the lungs, pancreas, upper airways, liver, intestine, and reproductive organs—to varying degrees. Its incidence among newborn babies in Germany is between 1 in 3300 and 1 in 4800. Its diagnosis requires both clinical evidence (positive newborn screening, sibling[s] with fibrosis, clinical signs) and the demonstration of CFTR dysfunction by an elevated chloride concentration in sweat, and/or two disease-causing mutations, and/or abnormal electrophysiological findings (nasal potential difference measurement, intestinal short-circuit current measurement). Patients should be cared for by specialized fibrosis centers in close cooperation with their primary care physicians. The median life span of patients with this disease has risen steadily to the current value of 40 years. Aside from symptomatic treatment, the first mutation-specific treatments have recently become available.Conclusion: Early diagnosis and optimized treatment prolong the lives of persons with fibrosis and improve their quality of life. Causally directed treatment for all patients and their effects on the course of disease are now central issues for further research.},
+abstract = {Background: Universal screening of newborn babies for Cystic Fibrosis was launched in Germany on 1 September 2016. Here we present up-to-date information on the diagnosis, treatment, and prognosis of this disease.Methods: This article is based on relevant publications retrieved by a selective search in PubMed, along with guidelines from Germany and abroad and systematic reviews.Results: Cystic Cystic Fibrosis is caused by a gene mutation leading to dysfunction of the Cystic Fibrosis transmembrane conductance regulator (CFTR) protein. It affects multiple organ systems—the lungs, pancreas, upper airways, liver, intestine, and reproductive organs—to varying degrees. Its incidence among newborn babies in Germany is between 1 in 3300 and 1 in 4800. Its diagnosis requires both clinical evidence (positive newborn screening, sibling[s] with Cystic Fibrosis, clinical signs) and the demonstration of CFTR dysfunction by an elevated chloride concentration in sweat, and/or two disease-causing mutations, and/or abnormal electrophysiological findings (nasal potential difference measurement, intestinal short-circuit current measurement). Patients should be cared for by specialized Cystic Fibrosis centers in close cooperation with their primary care physicians. The median life span of patients with this disease has risen steadily to the current value of 40 years. Aside from symptomatic treatment, the first mutation-specific treatments have recently become available.Conclusion: Early diagnosis and optimized treatment prolong the lives of persons with Cystic Fibrosis and improve their quality of life. Causally directed treatment for all patients and their effects on the course of disease are now central issues for further research.},
URL = {https://www.aerzteblatt.de/int/article.asp?id=193172},
@@ -713,7 +713,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,
`@article{Castellani2008,
- title={Consensus on the use and interpretation of fibrosis mutation analysis in clinical practice},
+ title={Consensus on the use and interpretation of Cystic Fibrosis mutation analysis in clinical practice},
author={Castellani, Carlo and Cuppens, H. and Macek Jr, Milan and Cassiman, Jean-Marie and Kerem, Eitan and Durie, Peter and Tullis, Elizabeth and Assael, B. M. and Bombieri, Cristina and Brown, Amanda and others},
journal={Journal of Cystic Fibrosis},
volume={7},
@@ -737,7 +737,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,
`@article{Southern2009,
- title={Newborn screening for fibrosis},
+ title={Newborn screening for Cystic Fibrosis},
author={Southern, Kevin W. and Mérelle, Marieke M. E. and Dankert-Roelse, Jeannette E. and Nagelkerke, Nico},
journal={Cochrane Database of Systematic Reviews},
volume={1},
@@ -748,7 +748,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,
`@article{Cutting2015,
- title={Cystic fibrosis genetics: from molecular understanding to clinical application},
+ title={Cystic Cystic Fibrosis genetics: from molecular understanding to clinical application},
author={Cutting, Garry R.},
journal={Nature Reviews Genetics},
volume={16},
@@ -759,7 +759,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,
/* 61 */
`@article{Crossley1979,
- title={Dried-blood spot screening for fibrosis in the newborn},
+ title={Dried-blood spot screening for Cystic Fibrosis in the newborn},
author={Crossley, John R. and Elliott, Robert B. and Smith, Patricia A.},
journal={The Lancet},
volume={1},
@@ -771,7 +771,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
}`,
`@book{Paracchini2011id,
- title = "Cystic fibrosis newborn screening: Distribution of blood
+ title = "Cystic Cystic Fibrosis newborn screening: Distribution of blood
immunoreactive trypsinogen concentrations in hypertrypsinemic
@@ -800,7 +800,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
} `,
`@article{southern2007survey,
- title={A survey of newborn screening for fibrosis in Europe},
+ title={A survey of newborn screening for Cystic Fibrosis in Europe},
author={Southern, Kevin W and Munck, Anne and Pollitt, Rodney and Travert, Georges and Zanolla, Luisa and Dankert-Roelse, Jeannette and Castellani, Carlo and ECFS CF Neonatal Screening Working Group and others},
@@ -820,7 +820,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
/* 64)*/
`@ARTICLE{Mishra2008-ia,
- title = "Diagnosis of fibrosis by sweat testing: age-specific
+ title = "Diagnosis of Cystic Fibrosis by sweat testing: age-specific
reference intervals",
@@ -838,7 +838,7 @@ eprint = {https://www.aerzteblatt.de/pdf.asp?id=193172}
>50 years and subjects who were pancreatic insufficient with
- fibrosis (CF) were recruited. Sweat collection was
+ Cystic Fibrosis (CF) were recruited. Sweat collection was
performed on all subjects with the Wescor Macroduct system.
@@ -919,7 +919,7 @@ note = {Accessed on 02. October 2024}
}`,
`@article{farrell2020impact,
- title={The impact of the CFTR gene discovery on fibrosis diagnosis, counseling, and preventive therapy},
+ title={The impact of the CFTR gene discovery on Cystic Fibrosis diagnosis, counseling, and preventive therapy},
author={Farrell, Philip M and Rock, Michael J and Baker, Mei W},
journal={Genes},
volume={11},
@@ -930,7 +930,7 @@ note = {Accessed on 02. October 2024}
}`,
`@article{Flume2009,
- title={Cystic fibrosis pulmonary guidelines: airway clearance therapies},
+ title={Cystic Cystic Fibrosis pulmonary guidelines: airway clearance therapies},
author={Flume, Patrick A. and Robinson, Karen A. and O'Sullivan, Brian P and Finder, Jon D and Vender, Robin L and Willey-Courand, Donna B and White, Thomas B and Marshall, Bruce C.},
journal={Respiratory Care},
volume={54},
@@ -942,7 +942,7 @@ note = {Accessed on 02. October 2024}
}`,
`@article{REISMAN1988632,
- title={Role of conventional physiotherapy in fibrosis},
+ title={Role of conventional physiotherapy in Cystic Fibrosis},
journal={The Journal of Pediatrics},
volume={113},
number={4},
@@ -955,7 +955,7 @@ note = {Accessed on 02. October 2024}
}`,
`@article{Wainwright2015,
- title={Lumacaftor-ivacaftor in patients with fibrosis homozygous for Phe508del CFTR},
+ title={Lumacaftor-ivacaftor in patients with Cystic Fibrosis homozygous for Phe508del CFTR},
author={Wainwright, Claire E and Elborn, J Stuart and Ramsey, Bonnie W and Marigowda, Girish and Huang, Xinxin and Cipolli, Marco and Colombo, Carlo and Davies, Jane C and De Boeck, Kris and Flume, Patrick A and others},
journal={New England Journal of Medicine},
volume={373},
@@ -980,7 +980,7 @@ note = {Accessed on 02. October 2024}
journal={Respiratory Care}
}`,
`@ARTICLE{Guo2022-ch,
- title={Worldwide rates of diagnosis and effective treatment for fibrosis},
+ title={Worldwide rates of diagnosis and effective treatment for Cystic Fibrosis},
author={Guo, Jonathan and Garratt, Anna and Hill, Andrew},
journal={Journal of Cystic Fibrosis},
volume={21},
@@ -989,12 +989,12 @@ note = {Accessed on 02. October 2024}
year={2022},
month={may},
doi={10.1016/j.jcf.2021.12.001},
- keywords={CFTR modulator, Cystic fibrosis, Epidemiology, Ivacaftor/tezacaftor/elexacaftor},
+ keywords={CFTR modulator, Cystic Cystic Fibrosis, Epidemiology, Ivacaftor/tezacaftor/elexacaftor},
publisher={Elsevier BV}
}`,
`@article{Griesenbach2013,
- title={Gene therapy progress and prospects: fibrosis},
+ title={Gene therapy progress and prospects: Cystic Fibrosis},
author={Griesenbach, Uta and Alton, Eric WFW},
journal={Gene Therapy},
volume={20},
@@ -1050,7 +1050,7 @@ note = {Accessed on 02. October 2024}
`@article{roda2022new,
author={Roda, Juliana and Pinto-Silva, Catarina and Silva, Iris A. I. and Maia, Carla and Almeida, Susana and Ferreira, Ricardo and Oliveira, Guiomar},
- title={New drugs in fibrosis: what has changed in the last decade?},
+ title={New drugs in Cystic Fibrosis: what has changed in the last decade?},
journal={Therapeutic Advances in Chronic Disease},
volume={13},
pages={20406223221098136},
@@ -1104,7 +1104,7 @@ note = {Accessed on 02. October 2024}
}`,
/* 84 */
`@article{Terlizzi2021,
- title={Hypertonic saline in people with fibrosis: review of comparative studies and clinical practice},
+ title={Hypertonic saline in people with Cystic Fibrosis: review of comparative studies and clinical practice},
author={Terlizzi, Valeria and Masi, Eleonora and Francalanci, Marco and Braggion, Chiara},
journal={Italian Journal of Pediatrics},
volume={47},
diff --git a/src/sources/eng-delivery-sources.tsx b/src/sources/eng-delivery-sources.tsx
index b4030c045c1485f11a110c3741f2c27c7cbeec6a..c70fa0b6a3aef5d3051fda54acebd208560f1f33 100644
--- a/src/sources/eng-delivery-sources.tsx
+++ b/src/sources/eng-delivery-sources.tsx
@@ -77,7 +77,7 @@ const bibtexSources = [
}
`,`
@article{wei_lung_2023,
- title = {Lung {SORT} {LNPs} enable precise homology-directed repair mediated {CRISPR}/{Cas} genome correction in fibrosis models},
+ title = {Lung {SORT} {LNPs} enable precise homology-directed repair mediated {CRISPR}/{Cas} genome correction in Cystic Fibrosis models},
author = {Wei, Tuo and Sun, Yehui and Cheng, Qiang and Chatterjee, Sumanta and Traylor, Zachary and Johnson, Lindsay T. and Coquelin, Melissa L. and Wang, Jialu and Torres, Michael J. and Lian, Xizhen and Wang, Xu and Xiao, Yufen and Hodges, Craig A. and Siegwart, Daniel J.},
year = 2023,
month = nov,
@@ -121,7 +121,7 @@ const bibtexSources = [
issn = {0168-3659},
url = {https://www.sciencedirect.com/science/article/pii/S0168365922006137},
keywords = {Lipid nanoparticles, LNP, Onpattro®, RNA therapeutics, siRNA delivery, Spray drying, Pulmonary delivery, Respiratory diseases, Human precision-cut lung slices, Formulation screening},
- abstract = {While all the siRNA drugs on the market target the liver, the lungs offer a variety of currently undruggable targets which could potentially be treated with RNA therapeutics. Hence, local, pulmonary delivery of RNA nanoparticles could finally enable delivery beyond the liver. The administration of RNA drugs via dry powder inhalers offers many advantages related to physical, chemical and microbial stability of RNA and nanosuspensions. The present study was therefore designed to test the feasibility of engineering spray dried lipid nanoparticle (LNP) powders. Spray drying was performed using 5% lactose solution (m/V), and the targets were set to obtain nanoparticle sizes after redispersion of spray-dried powders around 150 nm, a residual moisture level below 5%, and RNA loss below 15% at maintained RNA bioactivity. The LNPs consisted of an ionizable cationic lipid which is a sulfur-containing analog of DLin-MC3-DMA, a helper lipid, cholesterol, and PEG-DMG encapsulating siRNA. Prior to the spray drying, the latter process was simulated with a novel dual emission fluorescence spectroscopy method to preselect the highest possible drying temperature and excipient solution maintaining LNP integrity and stability. Through characterization of physicochemical and aerodynamic properties of the spray dried powders, administration criteria for delivery to the lower respiratory tract were fulfilled. Spray dried LNPs penetrated the lung mucus layer and maintained bioactivity for >90% protein downregulation with a confirmed safety profile in a lung adenocarcinoma cell line. Additionally, the spray dried LNPs successfully achieved up to 50% gene silencing of the house keeping gene GAPDH in ex vivo human precision-cut lung slices at without increasing cytokine levels. This study verifies the successful spray drying procedure of LNP-siRNA systems maintaining their integrity and mediating strong gene silencing efficiency on mRNA and protein levels both in vitro and ex vivo. The successful spray drying procedure of LNP-siRNA formulations in 5% lactose solution creates a novel siRNA-based therapy option to target respiratory diseases such as lung cancer, asthma, COPD, fibrosis and viral infections.}
+ abstract = {While all the siRNA drugs on the market target the liver, the lungs offer a variety of currently undruggable targets which could potentially be treated with RNA therapeutics. Hence, local, pulmonary delivery of RNA nanoparticles could finally enable delivery beyond the liver. The administration of RNA drugs via dry powder inhalers offers many advantages related to physical, chemical and microbial stability of RNA and nanosuspensions. The present study was therefore designed to test the feasibility of engineering spray dried lipid nanoparticle (LNP) powders. Spray drying was performed using 5% lactose solution (m/V), and the targets were set to obtain nanoparticle sizes after redispersion of spray-dried powders around 150 nm, a residual moisture level below 5%, and RNA loss below 15% at maintained RNA bioactivity. The LNPs consisted of an ionizable cationic lipid which is a sulfur-containing analog of DLin-MC3-DMA, a helper lipid, cholesterol, and PEG-DMG encapsulating siRNA. Prior to the spray drying, the latter process was simulated with a novel dual emission fluorescence spectroscopy method to preselect the highest possible drying temperature and excipient solution maintaining LNP integrity and stability. Through characterization of physicochemical and aerodynamic properties of the spray dried powders, administration criteria for delivery to the lower respiratory tract were fulfilled. Spray dried LNPs penetrated the lung mucus layer and maintained bioactivity for >90% protein downregulation with a confirmed safety profile in a lung adenocarcinoma cell line. Additionally, the spray dried LNPs successfully achieved up to 50% gene silencing of the house keeping gene GAPDH in ex vivo human precision-cut lung slices at without increasing cytokine levels. This study verifies the successful spray drying procedure of LNP-siRNA systems maintaining their integrity and mediating strong gene silencing efficiency on mRNA and protein levels both in vitro and ex vivo. The successful spray drying procedure of LNP-siRNA formulations in 5% lactose solution creates a novel siRNA-based therapy option to target respiratory diseases such as lung cancer, asthma, COPD, Cystic Fibrosis and viral infections.}
}
`,`
@article{jiang_combinatorial_2024,
@@ -154,7 +154,7 @@ const bibtexSources = [
url = {https://respiratory-research.biomedcentral.com/articles/10.1186/s12931-024-02800-7},
urldate = {2024-09-10},
abstract = {
- Background  Pulmonary ionocytes have been identified in the airway epithelium as a small population of ion transporting cells expressing high levels of CFTR ( fibrosis transmembrane conductance regulator), the gene mutated in fibrosis. By providing an infinite source of airway epithelial cells (AECs), the use of human induced pluripotent stem cells (hiPSCs) could overcome some challenges of studying ionocytes. However, the production of AEC epithelia containing ionocytes from hiPSCs has proven difficult. Here, we present a platform to produce hiPSCderived AECs (hiPSC-AECs) including ionocytes and investigate their role in the airway epithelium.
+ Background  Pulmonary ionocytes have been identified in the airway epithelium as a small population of ion transporting cells expressing high levels of CFTR ( Cystic Fibrosis transmembrane conductance regulator), the gene mutated in Cystic Fibrosis. By providing an infinite source of airway epithelial cells (AECs), the use of human induced pluripotent stem cells (hiPSCs) could overcome some challenges of studying ionocytes. However, the production of AEC epithelia containing ionocytes from hiPSCs has proven difficult. Here, we present a platform to produce hiPSCderived AECs (hiPSC-AECs) including ionocytes and investigate their role in the airway epithelium.
Methods  hiPSCs were differentiated into lung progenitors, which were expanded as 3D organoids and matured by air-liquid interface culture as polarised hiPSC-AEC epithelia. Using CRISPR/Cas9 technology, we generated a hiPSCs knockout (KO) for FOXI1, a transcription factor that is essential for ionocyte specification. Differences between FOXI1 KO hiPSC-AECs and their wild-type (WT) isogenic controls were investigated by assessing gene and protein expression, epithelial composition, cilia coverage and motility, pH and transepithelial barrier properties.
diff --git a/src/sources/eng-pe-sources.tsx b/src/sources/eng-pe-sources.tsx
index c72617183f6e05cf6a6b0712b688379828029514..2a19191d3e902a7d166418ad7ee2d7eb1d730910 100644
--- a/src/sources/eng-pe-sources.tsx
+++ b/src/sources/eng-pe-sources.tsx
@@ -137,7 +137,7 @@ const bibtexSources = [
doi = {10.1038/s41551-024-01233-3},
issn = {2157-846X},
rights = {2024 The Author(s)},
- abstractnote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
+ abstractnote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human Cystic Fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
language = {en}
}`,
`@article{Yan_Oyler-Castrillo_Ravisankar_Ward_Levesque_Jing_Simpson_Zhao_Li_Yan_2024,
diff --git a/src/sources/eng-peg-sources.tsx b/src/sources/eng-peg-sources.tsx
index 0ff82b49445f4eb542ad8fc49fcc4bb9c56dc2cb..f4ed5481f43b096937233a1f1c5e98eda0c9a324 100644
--- a/src/sources/eng-peg-sources.tsx
+++ b/src/sources/eng-peg-sources.tsx
@@ -65,12 +65,12 @@ const bibtexSources = [
doi = {10.1016/S0014-5793(01)02561-3},
issn = {1873-3468},
rights = {FEBS Letters 499 (2001) 1873-3468 © 2015 Federation of European Biochemical Societies},
- abstractnote = {The green fluorescent protein YFP-H148Q is sensitive to halides by a mechanism involving halide binding and a shift in pK a. However, a limitation of YFP-H148Q is its low halide sensitivity, with K d>100 mM for Cl−. Indicators with improved sensitivities are needed for cell transport studies, particularly in drug discovery by high-throughput screening, and for measurement of Cl− concentration in subcellular organelles. YFP-H148Q libraries were generated in which pairs of residues in the vicinity of the halide binding site were randomly mutated. An automated procedure was developed to screen bacterial colonies for improved halide sensitivity. Analysis of 1536 clones revealed improved anion sensitivities with K d down to 2 mM for I− (I152L), 40 mM for Cl− (V163S), and 10 mM for NO3 − (I152L). The anion-sensitive mechanism of these indicators was established and their utility in cells was demonstrated using transfected cells expressing the fibrosis transmembrane conductance regulator chloride channel.},
+ abstractnote = {The green fluorescent protein YFP-H148Q is sensitive to halides by a mechanism involving halide binding and a shift in pK a. However, a limitation of YFP-H148Q is its low halide sensitivity, with K d>100 mM for Cl−. Indicators with improved sensitivities are needed for cell transport studies, particularly in drug discovery by high-throughput screening, and for measurement of Cl− concentration in subcellular organelles. YFP-H148Q libraries were generated in which pairs of residues in the vicinity of the halide binding site were randomly mutated. An automated procedure was developed to screen bacterial colonies for improved halide sensitivity. Analysis of 1536 clones revealed improved anion sensitivities with K d down to 2 mM for I− (I152L), 40 mM for Cl− (V163S), and 10 mM for NO3 − (I152L). The anion-sensitive mechanism of these indicators was established and their utility in cells was demonstrated using transfected cells expressing the Cystic Fibrosis transmembrane conductance regulator chloride channel.},
language = {en}
}
`,`
@article{Bulcaen_Kortleven_Liu_Maule_Dreano_Kelly_Ensinck_Thierie_Smits_Ciciani_2024,
- title = {Prime editing functionally corrects fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
+ title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
author = {Bulcaen, Mattijs and Kortleven, Phéline and Liu, Ronald B. and Maule, Giulia and Dreano, Elise and Kelly, Mairead and Ensinck, Marjolein M. and Thierie, Sam and Smits, Maxime and Ciciani, Matteo and Hatton, Aurelie and Chevalier, Benoit and Ramalho, Anabela S. and Casadevall i Solvas, Xavier and Debyser, Zeger and Vermeulen, François and Gijsbers, Rik and Sermet-Gaudelus, Isabelle and Cereseto, Anna and Carlon, Marianne S.},
year = 2024,
month = may,
@@ -78,7 +78,7 @@ const bibtexSources = [
pages = 101544,
doi = {10.1016/j.xcrm.2024.101544},
issn = {2666-3791},
- abstractnote = {Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoidsâ€).}
+ abstractnote = {Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause Cystic Fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoidsâ€).}
}
`,`
@article{Renz_Valdivia-Francia_Sendoel_2020,
diff --git a/src/sources/joshua-inv-sources.tsx b/src/sources/joshua-inv-sources.tsx
index fd6a47e8c0c94e39039efc5632819ee7a11e85d9..56589c8862e7ecac2d671df2fe500050f89ad8ec 100644
--- a/src/sources/joshua-inv-sources.tsx
+++ b/src/sources/joshua-inv-sources.tsx
@@ -14,7 +14,7 @@ const bibtexSources = [
@article{eins,
-title = {Prime editing functionally corrects fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
+title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
journal = {Cell Reports Medicine},
@@ -45,7 +45,7 @@ month = {11},
pages = {1756-61},
-title = {Cystic fibrosis in three northeast Thai infants is CF really a rare disease in the Thai population?},
+title = {Cystic Cystic Fibrosis in three northeast Thai infants is CF really a rare disease in the Thai population?},
volume = {89},
@@ -63,7 +63,7 @@ month = {01},
pages = {8},
-title = {Cystic fibrosis in asia},
+title = {Cystic Cystic Fibrosis in asia},
volume = {4},
diff --git a/src/sources/liu-inv-sources.tsx b/src/sources/liu-inv-sources.tsx
index 414ffba18712982d476f4f4ca9c3be96d719c70a..cccf42e24cdaa35608abe3e6862b6687d6c2acac 100644
--- a/src/sources/liu-inv-sources.tsx
+++ b/src/sources/liu-inv-sources.tsx
@@ -64,7 +64,7 @@ title = {Systematic optimization of prime editing for the efficient functional c
rights = {2024 The Author(s)},
ISSN = {2157-846X},
DOI = {10.1038/s41551-024-01233-3},
-abstractNote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
+abstractNote = {Prime editing (PE) enables precise and versatile genome editing without requiring double-stranded DNA breaks. Here we describe the systematic optimization of PE systems to efficiently correct human Cystic Fibrosis (CF) transmembrane conductance regulator (CFTR) F508del, a three-nucleotide deletion that is the predominant cause of CF. By combining six efficiency optimizations for PE—engineered PE guide RNAs, the PEmax architecture, the transient expression of a dominant-negative mismatch repair protein, strategic silent edits, PE6 variants and proximal ‘dead’ single-guide RNAs—we increased correction efficiencies for CFTR F508del from less than 0.5% in HEK293T cells to 58% in immortalized bronchial epithelial cells (a 140-fold improvement) and to 25% in patient-derived airway epithelial cells. The optimizations also resulted in minimal off-target editing, in edit-to-indel ratios 3.5-fold greater than those achieved by nuclease-mediated homology-directed repair, and in the functional restoration of CFTR ion channels to over 50% of wild-type levels (similar to those achieved via combination treatment with elexacaftor, tezacaftor and ivacaftor) in primary airway cells. Our findings support the feasibility of a durable one-time treatment for CF.},
journal = {Nature Biomedical Engineering},
publisher = {Nature Publishing Group},
author = {Sousa, Alexander A. and Hemez, Colin and Lei, Lei and Traore, Soumba and Kulhankova, Katarina and Newby, Gregory A. and Doman, Jordan L. and Oye, Keyede and Pandey, Smriti and Karp, Philip H. and McCray, Paul B. and Liu, David R.},
diff --git a/src/sources/mattij-inv-sources.tsx b/src/sources/mattij-inv-sources.tsx
index 42c507b32c53fd7d50c171d726b46ebc59df511b..f8074fe843132fea8a35d8b5a441aaca33650b47 100644
--- a/src/sources/mattij-inv-sources.tsx
+++ b/src/sources/mattij-inv-sources.tsx
@@ -13,7 +13,7 @@ const bibtexSources = [
`
@article{Bulcaen_Kortleven_Liu_Maule_Dreano_Kelly_Ensinck_Thierie_Smits_Ciciani_et,
title = {
- Prime editing functionally corrects fibrosis-causing CFTR mutations in
+ Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in
human organoids and airway epithelial cells
},
author = {
@@ -34,7 +34,7 @@ const bibtexSources = [
Prime editing is a recent, CRISPR-derived genome editing technology capable
of introducing precise nucleotide substitutions, insertions, and deletions.
Here, we present prime editing approaches to correct L227R- and N1303K-CFTR,
- two mutations that cause fibrosis and are not eligible for current
+ two mutations that cause Cystic Fibrosis and are not eligible for current
market-approved modulator therapies. We show that, upon DNA correction of the
CFTR gene, the complex glycosylation, localization, and, most importantly,
function of the CFTR protein are restored in HEK293T and 16HBE cell lines.
diff --git a/src/sources/methods-sources.tsx b/src/sources/methods-sources.tsx
index 0226aaa7b8407e1bcbf8449a3f019711c92359c0..51a41f8b63a425e66cded1c0a166f5f5c8736f7e 100644
--- a/src/sources/methods-sources.tsx
+++ b/src/sources/methods-sources.tsx
@@ -171,7 +171,7 @@ const bibtexSources = [
`,
`
@article{BULCAEN2024101544,
-title = {Prime editing functionally corrects fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
+title = {Prime editing functionally corrects Cystic Fibrosis-causing CFTR mutations in human organoids and airway epithelial cells},
journal = {Cell Reports Medicine},
volume = {5},
number = {5},
@@ -181,9 +181,9 @@ issn = {2666-3791},
doi = {https://doi.org/10.1016/j.xcrm.2024.101544},
url = {https://www.sciencedirect.com/science/article/pii/S2666379124002349},
author = {Mattijs Bulcaen and Phéline Kortleven and Ronald B. Liu and Giulia Maule and Elise Dreano and Mairead Kelly and Marjolein M. Ensinck and Sam Thierie and Maxime Smits and Matteo Ciciani and Aurelie Hatton and Benoit Chevalier and Anabela S. Ramalho and Xavier {Casadevall i Solvas} and Zeger Debyser and François Vermeulen and Rik Gijsbers and Isabelle Sermet-Gaudelus and Anna Cereseto and Marianne S. Carlon},
-keywords = { fibrosis, prime editing, patient-derived organoids, human nasal epithelial cells, gene editing, machine learning, DETEOR, CRISPR},
+keywords = { Cystic Fibrosis, prime editing, patient-derived organoids, human nasal epithelial cells, gene editing, machine learning, DETEOR, CRISPR},
abstract = {Summary
-Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoidsâ€).}
+Prime editing is a recent, CRISPR-derived genome editing technology capable of introducing precise nucleotide substitutions, insertions, and deletions. Here, we present prime editing approaches to correct L227R- and N1303K-CFTR, two mutations that cause Cystic Fibrosis and are not eligible for current market-approved modulator therapies. We show that, upon DNA correction of the CFTR gene, the complex glycosylation, localization, and, most importantly, function of the CFTR protein are restored in HEK293T and 16HBE cell lines. These findings were subsequently validated in patient-derived rectal organoids and human nasal epithelial cells. Through analysis of predicted and experimentally identified candidate off-target sites in primary stem cells, we confirm previous reports on the high prime editor (PE) specificity and its potential for a curative CF gene editing therapy. To facilitate future screening of genetic strategies in a translational CF model, a machine learning algorithm was developed for dynamic quantification of CFTR function in organoids (DETECTOR: “detection of targeted editing of CFTR in organoidsâ€).}
}
new8.
@article{Ensinck_Deeersmaecker_Heylen_Ramalho_Gijsbers_Far,
@@ -238,8 +238,8 @@ new8.
`,
`@article{ehrhardt_towards_2006,
- title = {Towards an in vitro model of fibrosis small airway epithelium: characterisation of the human bronchial epithelial cell line {CFBE41o}-},
- shorttitle = {Towards an in vitro model of fibrosis small airway epithelium},
+ title = {Towards an in vitro model of Cystic Fibrosis small airway epithelium: characterisation of the human bronchial epithelial cell line {CFBE41o}-},
+ shorttitle = {Towards an in vitro model of Cystic Fibrosis small airway epithelium},
author = {Ehrhardt, Carsten and Collnot, Eva-Maria and Baldes, Christiane and Becker, Ulrich and Laue, Michael and Kim, Kwang-Jin and Lehr, Claus-Michael},
year = 2006,
month = mar,
@@ -253,7 +253,7 @@ new8.
urldate = {2024-09-09},
copyright = {http://www.springer.com/tdm},
language = {en},
- file = {Ehrhardt et al. - 2006 - Towards an in vitro model of fibrosis small.pdf:C\:\\Users\\Isabell\\Zotero\\storage\\RXLMCE3V\\Ehrhardt et al. - 2006 - Towards an in vitro model of fibrosis small.pdf:application/pdf}
+ file = {Ehrhardt et al. - 2006 - Towards an in vitro model of Cystic Fibrosis small.pdf:C\:\\Users\\Isabell\\Zotero\\storage\\RXLMCE3V\\Ehrhardt et al. - 2006 - Towards an in vitro model of Cystic Fibrosis small.pdf:application/pdf}
}
`
,