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Erica Akene Jessica Song Anastasija Petrovic

Abstract

Introduction: Cystic Fibrosis (CF) is a progressive genetic disease that causes the production of thick mucus in the lungs. A mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene leads to a malfunction of the CFTR protein. Current therapies for cystic fibrosis treat the symptoms rather but not the disease source. This study proposes using prime editing in combination with mesenchymal stem cell-derived exosomes (MSCEs) as an alternative treatment for CF. This treatment could potentially (1) correct the CFTR mutation in lung epithelial cells, and (2) regenerate tissue function after damage caused by cystic fibrosis in the lungs.


Methods: The MSCEs are obtained from adipose tissue through differential centrifugation and ultracentrifugation. They will then be surface engineered with a low molecular weight polyethylene glycol to help better penetrate the mucus layer and after incubated with the prime editor and liposomes to create the hybrid liposome MSCEs and encapsulate the prime editor.  Both in vivo and in vitro experiments with mice models and human lung organoids will be used to test the MSCEs and prime editor. After administration, efficiency of the treatment will be measured through the recombinant protein, FVII (rFVII) and microscopy.


Results: The MSCEs will be delivered through nebulization to reach the lung epithelial cells to correct the mutation, leading to the proper expression of the protein in the lungs. The MSCEs will further regenerate damage already caused by the disease.


Discussion: If the treatment is effective, we expect to see the production of thinner mucus in the lungs and an increased ability to breathe over time due to the MSCEs.


Conclusion: Currently, there is no cure for CF and the lifespan of CF patients is around 44 years. MSCEs and prime editing could be a safe and viable option for treating CF

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Section
Research Protocol