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

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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Bibliographic Details
Main Authors: Maximillian Woodall, Robert Tarran, Rhianna Lee, Hafssa Anfishi, Stella Prins, John Counsell, Paola Vergani, Stephen Hart, Deborah Baines
Format: Article
Language:English
Published: Elsevier 2023-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
cystic fibrosis
gene therapy
CFTR
airway epithelium
codon optimisation
anion secretion
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050123001201

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http://www.sciencedirect.com/science/article/pii/S2329050123001201

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