CFTR modulators: the changing face of cystic fibrosis in the era of precision medicine

M Lopes-Pacheco - Frontiers in pharmacology, 2020 - frontiersin.org
Cystic fibrosis (CF) is a lethal inherited disease caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …

Chemistry and biology of multicomponent reactions

A Domling, W Wang, K Wang - Chemical reviews, 2012 - ACS Publications
Multicomponent reactions (MCRs) are one-pot reactions employing more than two starting
materials, for example, 3, 4,..., 7, where most of the atoms of the starting materials are …

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 …

Structure, gating, and regulation of the CFTR anion channel

L Csanády, P Vergani, DC Gadsby - Physiological reviews, 2019 - journals.physiology.org
The cystic fibrosis transmembrane conductance regulator (CFTR) belongs to the ATP
binding cassette (ABC) transporter superfamily but functions as an anion channel crucial for …

[HTML][HTML] Ivacaftor potentiation of multiple CFTR channels with gating mutations

H Yu, B Burton, CJ Huang, J Worley, D Cao… - Journal of Cystic …, 2012 - Elsevier
BACKGROUND: The investigational CFTR potentiator ivacaftor (VX-770) increased CFTR
channel activity and improved lung function in subjects with CF who have the G551D CFTR …

CFTR: folding, misfolding and correcting the ΔF508 conformational defect

GL Lukacs, AS Verkman - Trends in molecular medicine, 2012 - cell.com
Cystic fibrosis (CF), the most common lethal genetic disease in the Caucasian population, is
caused by loss-of-function mutations of the CF transmembrane conductance regulator …

CFTR function and prospects for therapy

JR Riordan - Annu. Rev. Biochem., 2008 - annualreviews.org
Mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator
(CFTR) epithelial anion channel cause cystic fibrosis (CF). The multidomain integral …

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 …

Chloride channels as drug targets

AS Verkman, LJV Galietta - Nature reviews Drug discovery, 2009 - nature.com
Chloride channels represent a relatively under-explored target class for drug discovery as
elucidation of their identity and physiological roles has lagged behind that of many other …

Small-molecule correctors of defective ΔF508-CFTR cellular processing identified by high-throughput screening

N Pedemonte, GL Lukacs, K Du, E Caci… - The Journal of …, 2005 - Am Soc Clin Investig
The most common cause of cystic fibrosis (CF) is deletion of phenylalanine 508 (ΔF508) in
the CF transmembrane conductance regulator (CFTR) chloride channel. The ΔF508 …