Expression of gain-of-function CFTR in cystic fibrosis airway cells restores epithelial function better than wild-type or codon-optimized CFTR
Author:
Funder
National Institutes of Health
Cystic Fibrosis Foundation
Cystic Fibrosis Trust
Publisher
Elsevier BV
Subject
Genetics,Molecular Biology,Molecular Medicine
Reference60 articles.
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2. Extensive posttranscriptional deletion of the coding sequences for part of nucleotide-binding fold 1 in respiratory epithelial mRNA transcripts of the cystic fibrosis transmembrane conductance regulator gene is not associated with the clinical manifestations of cystic fibrosis.
3. A little CFTR goes a long way: CFTR-dependent sweat secretion from G551D and R117H-5T cystic fibrosis subjects taking ivacaftor;Char;PLoS One,2014
4. Lentiviral-mediated phenotypic correction of cystic fibrosis pigs;Cooney;JCI Insight,2016
5. Gene Therapy for Cystic Fibrosis: Progress and Challenges of Genome Editing;Maule;Int. J. Mol. Sci.,2020
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