Using Prime Editing and Mesenchymal Stem Cell-Derived Exosomes to Treat Cystic Fibrosis: A Research Protocol

Introduction: Cystic Fibrosis (CF) is a progressive genetic disease that causes the production of thick mucus in lungs. A mutation cystic fibrosis transmembrane conductance regulator (CFTR) gene leads to malfunction CFTR protein. Current therapies for treat symptoms rather but not source. This study proposes using prime editing combination with mesenchymal stem cell-derived exosomes (MSCEs) as an alternative treatment CF. could potentially (1) correct lung epithelial cells, and (2) regenerate tissue function after damage caused by Methods: The MSCEs are obtained from adipose through differential centrifugation ultracentrifugation. They will then be surface engineered low molecular weight polyethylene glycol help better penetrate layer incubated editor liposomes create hybrid liposome encapsulate editor. Both vivo vitro experiments mice models human organoids used test After administration, efficiency measured recombinant protein, FVII (rFVII) microscopy. Results: delivered nebulization reach cells mutation, leading proper expression protein further already disease. Discussion: If effective, we expect see thinner lungs increased ability breathe over time due MSCEs. Conclusion: Currently, there no cure CF lifespan patients around 44 years. safe viable option treating