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 …
transmembrane conductance regulator (CFTR) gene, which result in impairment of CFTR …
Chemistry and biology of multicomponent reactions
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 …
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 …
CF transmembrane conductance regulator (CFTR), a protein kinase A (PKA)-activated …
Structure, gating, and regulation of the CFTR anion channel
The cystic fibrosis transmembrane conductance regulator (CFTR) belongs to the ATP
binding cassette (ABC) transporter superfamily but functions as an anion channel crucial for …
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 …
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 …
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 …
(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 …
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 …
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
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 …
the CF transmembrane conductance regulator (CFTR) chloride channel. The ΔF508 …