Article
Next generation triplex-forming PNAs for site-specific genome editing of the F508del CFTR mutation.
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2025
Gupta Anisha, Barone Christina, Quijano Elias, Piotrowski-Daspit Alexandra S, Perera J Dinithi, Riccardi Adele, Jamali Haya, Turchick Audrey, Zao Weixi, Saltzman W Mark, Glazer Peter M, Egan Marie E
Abstract excerpt
BACKGROUND: Cystic Fibrosis (CF) is an autosomal recessive genetic disease caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) protein for which there is no cure. One approach to cure CF is to correct the underlying mutations in the CFTR gene. We have used triplex-forming peptide nucleic acids (PNAs) loaded into biodegradable nanoparticles (NPs) in combination...
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