Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR

Class Ia/b cystic fibrosis transmembrane regulator (CFTR) variants cause severe lung disease in 10% of cystic fibrosis (CF) patients and are untreatable with small-molecule pharmaceuticals. Genetic replacement of CFTR offers a cure, but its effectiveness is limited in vivo. We hypothesized that enha...

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Detalles Bibliográficos
Autores principales: Woodall, Maximillian, Tarran, Robert, Lee, Rhianna, Anfishi, Hafssa, Prins, Stella, Counsell, John, Vergani, Paola, Hart, Stephen, Baines, Deborah
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10494266/
https://www.ncbi.nlm.nih.gov/pubmed/37701179
http://dx.doi.org/10.1016/j.omtm.2023.08.006

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