“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 were interesting and adventurous but meeting him put a lot into perspective. </p>
<p>Our focus shifted to the safety of our creation. When coming up with ideas, we asked ourselves,
<p>Our focus shifted to the safety[Link Biosafety] of our creation. When coming up with ideas, we asked ourselves,
Is this idea a promising or an interesting one?
Would it be thrilling to create or benefit patients? </p>
<p>Due to this, Max had a profound influence on our project from the beginning and is the main reason why we chose Integrated Human Practices and Safety & Security as our special prizes. Only after this discussion did we decide on targeting the lung instead of the pancreas and discarded the idea of a diagnostic approach. He did not only give us important information but most importantly personal investment into our project. </p></>],
<p>Due to this, Max had a profound influence on our project from the beginning and is the main reason why we chose Integrated Human Practices[Link Best HP] and Safety & Security[Link Best Biosafety] as our special prizes. Only after this discussion did we decide on targeting the lung instead of the pancreas and discarded the idea of a diagnostic approach. He did not only give us important information but most importantly personal investment into 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."/>
affiliation:"Technical Faculty of Bielefeld University",
pictureurl:pics['kristian'],
tag:"Academia",
heading:"Discussion about the delivery method- AVV vs. LNPs",
heading:"Discussion about the delivery method- AVV vs. LNPs and prime editing and gene therapy innovations",
interviewtabid:"kristian",
cardtext:"",
language:"de",
quote:"X",
aimofcontact:"X",
insights:"X",
implementation:"X",
summary:"",
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 cystic fibrosis. 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 cystic fibrosis.
Prof. Dr. Kristian Müller emphasized the critical role of delivery systems in the success of gene therapies, particularly in the context of cystic fibrosis 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 cystic fibrosis. 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 cystic fibrosis, 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 cystic fibrosis as a model disease, given its prevalence and the potential for impactful treatments through targeted genetic corrections.",
interview:<><iframetitle="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></>,
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 interviews<HPLinktoOtherHPTabtab="joshua"text="Joshua"/>. </p>],
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>],
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."/>
