From a2cfbe9d9714a247501436dd5fb6384c81b8002c Mon Sep 17 00:00:00 2001 From: Liliana Sanfilippo <lsanfilippo@techfak.uni-bielefeld.de> Date: Thu, 21 Nov 2024 17:43:29 +0100 Subject: [PATCH] "AirBuddy" nicht kursiv schreiben --- src/contents/description.tsx | 4 ++-- 1 file changed, 2 insertions(+), 2 deletions(-) diff --git a/src/contents/description.tsx b/src/contents/description.tsx index 5d8eb830..d597eeaa 100644 --- a/src/contents/description.tsx +++ b/src/contents/description.tsx @@ -342,8 +342,8 @@ export function Description() { </Section> <Section title="Our Achievement" id="Our Achievement"> - <p>We have successfully demonstrated a <b>proof of concept</b> for our gene therapy approach targeting Cystic Fibrosis. In initial experiments, HEK cells carrying a 3-base deletion analogous to the <i>F508del</i> mutation were transfected with our prime editing complex. The results met our expectations, confirming the viability of our approach for precise gene correction. Based on these findings, we optimized the prime editing complex, leading to the creation of <i>PrimeGuide</i>, a more compact and efficient editing tool. </p> - <p>Central to our <b>delivery system</b> is <b>AirBuddy</b>, a lung-specific lipid nanoparticle designed to stabilize and protect the prime editing complex during transport to lung epithelial cells. <i>AirBuddy</i> ensures that the protein complex is delivered specifically to lung cells, enhancing the efficiency of the gene-editing process. By modifying the lipid nanoparticle with protective features, we achieved increased stability, ensuring effective delivery to the target cells. </p> + <p>We have successfully demonstrated a <b>proof of concept</b> for our gene therapy approach targeting Cystic Fibrosis. In initial experiments, HEK cells carrying a 3-base deletion analogous to the <i>F508del</i> mutation were transfected with our prime editing complex. The results met our expectations, confirming the viability of our approach for precise gene correction. Based on these findings, we optimized the prime editing complex, leading to the creation of <b>PrimeGuide</b>, a more compact and efficient editing tool. </p> + <p>Central to our <b>delivery system</b> is <b>AirBuddy</b>, a lung-specific lipid nanoparticle designed to stabilize and protect the prime editing complex during transport to lung epithelial cells. <b>AirBuddy</b> ensures that the protein complex is delivered specifically to lung cells, enhancing the efficiency of the gene-editing process. By modifying the lipid nanoparticle with protective features, we achieved increased stability, ensuring effective delivery to the target cells. </p> <p>We further optimized the prime editing fusion protein, <b>PrimeGuide</b>, to streamline its components, resulting in a smaller and more efficient prime editing complex. This improvement significantly enhances the precision of the gene editing process, reducing off-target effects and increasing the overall success of mutation correction. </p> <p>In subsequent experiments, <b>HEK and lung (CFBE41o-)cells</b> carrying the CFTR <i>F508del</i> mutation were successfully <b>transfected</b> with the optimized prime editing complex. Our results indicated successful correction of the mutation, confirming the potential of our approach for treating Cystic Fibrosis. </p> <p>Additionally, we explored <b>downstream applications</b>. Primary cell cultures were treated with lipid nanoparticles to introduce a reporter RNA. We also established 2D cultures transfected with YFP, a sodium-sensitive reporter protein, to assess ion channel functionality. Finally, in CFTR-deficient organoids, our system facilitated repair of the CFTR channel, evidenced by an increase in organoid volume upon treatment. This suggests successful functional restoration of CFTR activity. </p> -- GitLab