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<Sectiontitle="Abstract"id="Abstract">
<pid="obenindescription">We are proud to introduce our next-generation prime editing technology <PreCyse/> . We aim to develop an innovative gene therapy against cystic fibrosis, tackling the most common mutation ΔF508 of the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene. We optimize lipid nanoparticles (LNPs) for the efficient and cell-specific delivery of our therapeutic mRNA. Current treatment strategies are limited in terms of speed, precision and effectiveness, often failing to achieve long-lasting improvements. In addition, high costs and limited accessibility of pharmaceuticals contribute to adverse prognosis of many patients. We want to develop a monthly applied which represents a cure that is more advanced and user-friendly compared to other medications due to its longer lasting time, lowering the frequency of use. </p>
<p>We chose to focus on CF and specifically the ΔF508 mutation due to its prevalence and the severe impact it has on patients' lives. Additionally, our team includes members who have close friends affected by this condition, giving us a personal connection and a strong motivation to find a solution. By targeting the ΔF508 mutation, we aim to develop a therapy that could potentially, not only benefit many CF patients and make a significant improvement in their lives, but also can serve as a template, which research groups can use to target other genetic diseases. </p>
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<pdata-aos="zoom-y-out">Cystic fibrosis (CF) is the most common life-limiting genetic disorder in the Caucasian population. In Europe, CF affecting about 1 in 3,000 newborns
<SupScrollLinklabel="1"/>.</p>
<p> It is caused by mutations in the CFTR gene, which controls ions and water movement in cells. This leads to thick mucus, clogging airways, and frequent infections. The defective CFTR protein impacts the respiratory and digestive systems, causing chronic lung infections, breathing difficulties, and malnutrition. CF's severity varies, but it reduces life quality and expectancy. There are over 1,700 CFTR mutations; the ΔF508 mutation is most common, present in 70% of cases. It prevents proper protein folding, affecting its function. </p>
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<p>The mutations can be divided into six classes [9]:</p>
<p>Class I mutations prevent the synthesis of CFTR proteins altogether, meaning no channels are produced.</p>
<p>Class II mutations, which include the common F508del mutation (responsible for about 85% of cases [10]), disrupt the maturation process of the protein. As a result, the defective channels are quickly degraded by the cell.</p>
<p>Class III mutations, known as “gating” mutations, reduce the likelihood that the CFTR channel will open correctly, impairing its function.</p>
<p>Class IV, V, and VI mutations are rare. These mutations result in the production of unstable or inefficient CFTR proteins, which do not function adequately and are produced in insufficient numbers.</p>
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@@ -76,8 +94,7 @@ export function Description() {
<p>It is a deletion of the three nucleotides "CTT" at position 508, which removes the phenylalanine residue
without causing a frameshift. This deletion leads to defects in the kinetic and thermodynamic folding
of the NBD1 domain <SupScrollLinklabel="16"/>. However, this not only leads to misfolding of CFTR but also to defects in
trafficking and premature degradation, resulting in reduced surface expression of CFTR <SupScrollLinklabel="17"/>. </p>
trafficking and premature degradation, resulting in reduced surface expression of CFTR <SupScrollLinklabel="17"/>. </p>
<p>We chose to focus on CF and specifically the ΔF508 mutation due to its prevalence and the severe impact it has on patients' lives. Additionally, our team includes members who have close friends affected by this condition, giving us a personal connection and a strong motivation to find a solution. By targeting the ΔF508 mutation, we aim to develop a therapy that could potentially, not only benefit many CF patients and make a significant improvement in their lives, but also can serve as a template, which research groups can use to target other genetic diseases. </p>
<p>To correct the mutation, we are utilizing Prime Editing technologies. Prime Editing is a genome editing technique that allows precise DNA modifications without causing double-strand breaks<SupScrollLinklabel="2"/>. Structurally, the Prime Editing complex consists of a Cas9 endonuclease fused to a reverse transcriptase (RT) and guided by a pegRNA, which directs the complex to the target site in the genome. </p>
