Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

F Van Goor, S Hadida… - Proceedings of the …, 2009 - National Acad Sciences
Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in the gene encoding the
CF transmembrane conductance regulator (CFTR), a protein kinase A (PKA)-activated …

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

G Veit, RG Avramescu, D Perdomo… - Science translational …, 2014 - science.org
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane regulator (CFTR) that
result in reduced anion conductance at the apical membrane of secretory epithelia …

Ion channel modulators in cystic fibrosis

M Gentzsch, MA Mall - Chest, 2018 - Elsevier
Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance
regulator (CFTR) gene and remains one of the most common life-shortening genetic …

Pharmacological modulation of ion channels for the treatment of cystic fibrosis

MC Pinto, IAL Silva, MF Figueira… - Journal of …, 2021 - Taylor & Francis
Cystic fibrosis (CF) is a life-shortening monogenic disease caused by mutations in the gene
encoding the CF transmembrane conductance regulator (CFTR) protein, an anion channel …

Targeting a genetic defect: cystic fibrosis transmembrane conductance regulator modulators in cystic fibrosis

N Derichs - European Respiratory Review, 2013 - Eur Respiratory Soc
Cystic fibrosis (CF) is caused by genetic mutations that affect the cystic fibrosis
transmembrane conductance regulator (CFTR) protein. These mutations can impact the …

Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Potentiator VX-770 (Ivacaftor) Opens the Defective Channel Gate of Mutant CFTR in a …

PDW Eckford, C Li, M Ramjeesingh, CE Bear - Journal of Biological …, 2012 - ASBMB
The cystic fibrosis transmembrane conductance regulator (CFTR) acts as a channel on the
apical membrane of epithelia. Disease-causing mutations in the cystic fibrosis gene can …

Challenges facing airway epithelial cell-based therapy for cystic fibrosis

A Berical, RE Lee, SH Randell… - Frontiers in pharmacology, 2019 - frontiersin.org
Mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene cause
the life-limiting hereditary disease, cystic fibrosis (CF). Decreased or absent functional CFTR …

High-affinity activators of cystic fibrosis transmembrane conductance regulator (CFTR) chloride conductance identified by high-throughput screening

T Ma, L Vetrivel, H Yang, N Pedemonte… - Journal of Biological …, 2002 - ASBMB
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator
(CFTR) protein that reduce cAMP-stimulated Cl− conductance in airway and other epithelia …

Cystic fibrosis: exploiting its genetic basis in the hunt for new therapies

JL Kreindler - Pharmacology & therapeutics, 2010 - Elsevier
Cystic fibrosis (CF) is caused by mutations in the gene encoding the cystic fibrosis
transmembrane conductance regulator (CFTR), an anion channel expressed in epithelial …

Rescue of ΔF508-CFTR trafficking and gating in human cystic fibrosis airway primary cultures by small molecules

F Van Goor, KS Straley, D Cao… - … of Physiology-Lung …, 2006 - journals.physiology.org
Cystic fibrosis (CF) is a fatal genetic disease caused by mutations in cftr, a gene encoding a
PKA-regulated Cl− channel. The most common mutation results in a deletion of …