From CFTR biology toward combinatorial pharmacotherapy: expanded classification of cystic fibrosis mutations
G Veit, RG Avramescu, AN Chiang… - Molecular biology of …, 2016 - Am Soc Cell Biol
More than 2000 mutations in the cystic fibrosis transmembrane conductance regulator
(CFTR) have been described that confer a range of molecular cell biological and functional …
(CFTR) have been described that confer a range of molecular cell biological and functional …
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] CFTR function, pathology and pharmacology at single-molecule resolution
The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel that
regulates salt and fluid homeostasis across epithelial membranes. Alterations in CFTR …
regulates salt and fluid homeostasis across epithelial membranes. Alterations in CFTR …
[HTML][HTML] Atomic structure of the cystic fibrosis transmembrane conductance regulator
The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel
evolved from the ATP-binding cassette (ABC) transporter family. In this study, we determined …
evolved from the ATP-binding cassette (ABC) transporter family. In this study, we determined …
Potentiator ivacaftor abrogates pharmacological correction of ΔF508 CFTR in cystic fibrosis
DM Cholon, NL Quinney, ML Fulcher… - Science translational …, 2014 - science.org
Cystic fibrosis (CF) is caused by mutations in the CF transmembrane conductance regulator
(CFTR). Newly developed “correctors” such as lumacaftor (VX-809) that improve CFTR …
(CFTR). Newly developed “correctors” such as lumacaftor (VX-809) that improve CFTR …
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 …
apical membrane of epithelia. Disease-causing mutations in the cystic fibrosis gene can …
Conformational changes of CFTR upon phosphorylation and ATP binding
The cystic fibrosis transmembrane conductance regulator (CFTR) is an anion channel
evolved from an ATP-binding cassette transporter. CFTR channel gating is strictly coupled to …
evolved from an ATP-binding cassette transporter. CFTR channel gating is strictly coupled to …
Vx-770 potentiates CFTR function by promoting decoupling between the gating cycle and ATP hydrolysis cycle
Vx-770 (Ivacaftor), a Food and Drug Administration (FDA)-approved drug for clinical
application to patients with cystic fibrosis (CF), shifts the paradigm from conventional …
application to patients with cystic fibrosis (CF), shifts the paradigm from conventional …
Structural mechanisms of CFTR function and dysfunction
Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) chloride channel plays a
critical role in regulating transepithelial movement of water and electrolyte in exocrine …
critical role in regulating transepithelial movement of water and electrolyte in exocrine …
[HTML][HTML] Residual function of cystic fibrosis mutants predicts response to small molecule CFTR modulators
Abstract Treatment of individuals with cystic fibrosis (CF) has been transformed by small
molecule therapies that target select pathogenic variants in the CF transmembrane …
molecule therapies that target select pathogenic variants in the CF transmembrane …