“How positively and calmly Max deals with his illness but has also pointed out that he is lucky, and that other people are much worse off - how much you have to pay attention to little things that you wouldn't have expected as a healthy person.” </p>
</>],
implementation:[<><p>This most important aspect of this meeting was less an insight, but the fact Max helped us to put a face to an abstract idea. Many of our ideas to treat CF were interesting and adventurous but meeting him put a lot into perspective. </p>
<p>Max played a significant role in shaping our project from the outset, particularly in guiding our focus on <OurLinkpath='judging'tabId='judging'scrollToId='best-integrated-human-practice'text='Integrated Human Practices'/>{/*Kaya*/}and <OurLinkpath='judging'tabId='judging'scrollToId='safety-security'text='Safety & Security'/>{/*Kaya*/}as special prizes. Through our discussions, we recognized the importance of these aspects in developing the best possible cure. This collaborative effort led us to pivot our target from the pancreas to the lung and move away from a diagnostic approach. His contributions not only provided valuable insights but also fostered a strong personal investment in our project.</p></>],
<p>Max played a significant role in shaping our project from the outset, particularly in guiding our focus on <OurLinkpath='judging'scrollToId='best-integrated-human-practice'text='Integrated Human Practices'/> and <OurLinkpath='judging'scrollToId='safety-security'text='Safety & Security'/> as special prizes. Through our discussions, we recognized the importance of these aspects in developing the best possible cure. This collaborative effort led us to pivot our target from the pancreas to the lung and move away from a diagnostic approach. His contributions not only provided valuable insights but also fostered a strong personal investment in our project.</p></>],
pictureurl_implementation:"",
interview:<><QaBoxq="How and when were you first diagnosed? "a="When I was about one year old. My mother did not do any screenings or prenatal testing. I was in pain but as an infant you cannot say that, so I screamed a lot. Many doctors shrug that off in small children but after some time a sweat test was done at the children's clinic."/>
<QaBoxq="What do you think about diagnosing via sweat tests?"a="I am a clear opponent of diagnosing via sweat tests, especially if it is used to rule out CF and people have atypical CF, because of which they do not get diagnosed because of that."/>
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 Cystic Fibrosis awareness month and learnt even more about the importance of physiotherapy.",
implementation:[<p>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 <HPLinktoOtherHPTabtab="mukomove"text="MukoMove"/>- we actively took part in Cystic Fibrosis awareness month and learnt even more about the importance of physiotherapy.</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>],
We were able to make contact with Julia through the self-help group of <ahref="https://www.muko.info/ ">Mukviszidose e.V. </a> of which Max is a member. She subsequently reached out to us following Max's request for potential candidates for an interview with a patient group.
She and her husband have a six-year-old daughter carrying the F508del mutation in the CFTR gene and a toddler without CF. </p>],
insights:[<p> The interview with Julia shifted our focus to a new group of stakeholders: The patient’s support systems. Most people do not get genetically tested before having children and due to that, many people could get in the position of having a loved one with CF.
We considered the societal impacts, such as the rising health care costs, which Nicole Friedlein emphasized during our interview. She explained how the long-term nature of treatment, frequent hospital visits, and the need for specialized medications place a significant
We considered the societal impacts, such as the rising health care costs, which <HPLinktoOtherHPTabtab="nicole"text="Nicole Friedlein "/>emphasized during our interview. She explained how the long-term nature of treatment, frequent hospital visits, and the need for specialized medications place a significant
financial burden on both patients and the health care system. This insight shaped our understanding of the broader economic challenges faced by families and institutions involved in managing chronic illnesses. Meanwhile, Julia brought attention to the psychological impact,
stressing the emotional strain that accompanies not only the illness itself but also the financial pressures. She also showed us more perspectives on parenting of children with CF, than we heard before, and told us about the way from the first diagnosis to growing accustomed
to and living with a child with CF. Julia also confirmed that most children will have no issue using an inhalative therapy like we envision our gene therapy to be and shone light onto the comparatively very good situation for CF patients in Germany. </p>],
implementation:[<p> This interview helped us confirm the delivery method we planned to use as we were previously concerned how and if children would be able to use the inhalative therapy. Besides that, Julia gave us further insights into the emotional side of
dealing with CF and we were able to discuss the situation for patients in Germany in comparison to other countries better in later <HPLinktoOtherHPTabtab="joshua"text="interviews"/>. </p>],
dealing with CF and the immense psychological burden. We have discussed this issue in a meeting with <HPLinktoOtherHPTabtab="psychol"text="psychologist"/> from a nearby clinic. We were also able to discuss the situation for patients in Germany in comparison to other countries better in later <HPLinktoOtherHPTabtab="joshua"text="interviews"/>. </p>],
interview:<>
<QaBoxq="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."/>
<QaBoxq="Does your son also have Cystic Fibrosis?"a="No, he doesn’t."/>
quote:"Children are the world's best “mucus hiders”.",
aimofcontact:[<a>During the last interview with <HPLinktoOtherHPTabtab="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>],
aimofcontact:[<p>During the last interview with <HPLinktoOtherHPTabtab="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. </p>],
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. ",
quote:"We’ve had to sit by and watch people die, knowing that better treatment exists but is inaccessible. ",
aimofcontact:[<p>We contacted the organization <ahref="https://www.cfvww.org/">CF vests worldwide</a> with the aim to hear more diverse perspectives beyond Germany.
After the founder Rod connected us with Joshua, Joshua was so kind to conduct an interview with us not only about the perspectives and
After the founder Rod connected us with Joshua, he was so kind to conduct an interview with us not only about the perspectives and
stories he heard but also about his personal experiences with his daughter and living in a country where CF care is very hard to get. Joshua
(from the USA) and his family live in Thailand where he and his wife run a children’s home. Their daughter is the only child with CF.</p>,
<p>It is possible to learn more about Joshua and his family though the <ahref="https://thebonnellfoundation.org/cf-vests-worldwide/">
podcast of the Bonnel foundation</a>.</p>],
insights:[<p> Joshua showed us just how dire the situation is for CF patients is in some regions. It was shocking to hear there is only one doctor
knowledgeable about CF in Thailand and that many doctors dismiss the possibility of CF due to racial bias and misinformation. Additionally, we confirmed how much the accessibility
of care depends on the healthcare system, as we already touched on during the interview with <HPLinktoOtherHPTabtab="nicole"text="Nicole Friedlein"/>,. On the parenting level, Joshua brought in many perspectives contrary to what we previously heard. In the interview with <HPLinktoOtherHPTabtab="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?
of care depends on the healthcare system, as we already touched on during the interview with <HPLinktoOtherHPTabtab="nicole"text="Nicole Friedlein"/>. On the parenting level, Joshua brought in many perspectives contrary to what we previously heard. In the interview with <HPLinktoOtherHPTabtab="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 Joshua 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 Cystic Fibrosis, especially in the context of prime editing. Mattijs Bulceans's paper on
targeting the mutations L227R and N1303K <TabScrollLinktab="joshua"scrollId="desc-1"num="1"/> was one of few papers. After explicitly searching for Cystic Fibrosis records for specific countries and
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 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
with CF expert <HPLinktoOtherHPTabtab="sriram"text="Sriram Vaidyanathan"/>. 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
figuring out if we indeed made a mistake and we want to encourage other teams to do the same. iGem stands for innovation – but also for growth.
Especially in the context of Integrated Human Practices, it is important to examine both the positive and the negative to create a project with a
quote:"It’s important to keep my hygiene practices practical and effective, focusing on regular cleaning rather than excessive measures that may create more risks for invection.",
summary:[<p>In the feedback on the hygiene concept, the author states that the number of toilets required can be reduced as only a marginal number of people will be present at the university. He praised the first four points of the concept as very good and well thought out.
With regard to points five and six, he clarifies that no special hygiene items are required for the stay as long as the toilets are cleaned regularly and there is an additional service for the washbasins. He explains that the sources of danger can be significantly reduced by regular cleaning and the presence of toilet lids, which eliminates the need for special hygiene measures.
He also argues that contactless facilities are not necessary as people wash or sanitise their hands after using the toilet anyway. Finally, he asks whether sources are needed for the hygiene concept and offers his help to create a revised version, which he would then like to review again before it is forwarded to Mr Johannfunke.</p>],
summary:[<p>Inspired by <HPLinktoOtherHPTabtab="maxfirst"text="Max's"/> feedback on the unique challenges faced by individuals with immunocompromised systems, we initiated the development of a specialized <OurLinkpath='contribution'scrollToId='our-contributions'text="hygiene concept"/>. Max’s insights provided invaluable guidance in addressing the specific needs of cystic fibrosis (CF) patients and others with weakened immune systems. His input emphasized practical measures to ensure a safer and more hygienic environment in public spaces, particularly in university restrooms.
Through detailed discussions with Max, we developed guidelines that balance rigorous hygiene with feasibility. His feedback highlighted that while regular cleaning and essential facilities like toilet lids are crucial, overly complex measures, such as contactless systems, may not be necessary if proper hand hygiene is maintained. He praised the initial points of the concept as thoughtful and effective, further refining our approach to focus on high-impact solutions.
To ensure a robust and comprehensive framework, we sought additional review and advice from <HPLinktoOtherHPTabtab="johannfunke"text="Johann Funke"/> . By incorporating Max’s feedback and Johann's expertise, we aim to create a practical hygiene protocol that addresses the needs of immunocompromised individuals, making university spaces safer and more inclusive. </p>],
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 <ahref="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>],
aimofcontact:[<p>Our goal in reaching out to 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 <ahref="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.
The patient also talked about the heavy toll that existing treatments can take, both in terms of time and physical strain. This feedback has pushed us to focus on streamlining our approach, especially with our prime editing and inhalation therapy. We want to cut down on the complexity and frequency of treatments, making things simpler and less burdensome for patients.
We also learned about the ongoing need for more advanced therapies. The patient stressed the importance of continuing to push the boundaries with modulator therapies and gene editing. This has really driven us to prioritize our spray-dried lipid nanoparticles (LNPs) approach, which aims to deliver gene-editing tools straight to the lungs, tackling the root cause of CF.
We also learned about the ongoing need for more advanced therapies. The patient stressed the importance of continuing to push the boundaries with modulator therapies and gene editing. This has really driven us to prioritize our spray-dried LNPs approach, which aims to deliver gene-editing tools straight to the lungs, tackling the root cause of CF.
The discussion also gave us a clearer picture of the global disparities in CF care. It became evident that access to advanced treatments varies a lot around the world. This has made us think about how we can scale our therapy to be more accessible, even in places with fewer resources.
The patient’s personal experiences underscored the need for treatments that are tailored to individual needs. We’re using this insight to make sure our therapy can be adapted to different genetic mutations and patient responses.
Finally, their emphasis on enzyme development and gene therapy has influenced our research focus. We’re now prioritizing these areas to ensure our work addresses both current needs and future possibilities in CF treatment.
Overall, these insights have really helped us align our project with the real-world needs of CF patients, making sure that what we’re developing is both practical and impactful. </p>],
implementation:[<p>We incorporated the CF patient’s insights into our project by making several key adjustments to better align with the real-world needs of CF patients. Understanding the challenges they face and the impact of current treatments helped us refine our approach significantly.
Firstly, we focused on developing prime editing via inhalation therapy with spray-dried lipid nanoparticles (LNPs). This method directly targets genetic mutations in the lungs, addressing the patient’s need for more effective and less invasive treatments. We’re also fine-tuning the formulation and delivery of our LNPs to ensure they reach lung tissue efficiently while minimizing any discomfort.
Firstly, we focused on developing prime editing via inhalation therapy with spray-dried LNPs. This method directly targets genetic mutations in the lungs, addressing the patient’s need for more effective and less invasive treatments. We’re also fine-tuning the formulation and delivery of our LNPs to ensure they reach lung tissue efficiently while minimizing any discomfort.
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>],
Psychological support, family counselling, and the involvement of patient communities are essential in helping patients and their families cope with the emotional and mental challenges of both the disease and its treatments. The strength of these support systems can make a profound difference in how well patients navigate the challenges of living with CF, particularly when adapting to new therapies.
We also learned that while new therapies are exciting, their success often depends on their practicality. Treatments that are complex, invasive, or burdensome are less likely to be adopted, even if they promise significant benefits. Simplicity, ease of use, and reducing the treatment burden are critical factors in ensuring patients fully engage with and benefit from these therapies.
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.
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 <OurLinkpath='human-practices'scrollToId='implementation-feedback'text="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 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.",
