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 …
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 …
Orkambi® and amplifier co‐therapy improves function from a rare CFTR mutation in gene‐edited cells and patient tissue
SV Molinski, S Ahmadi, W Ip, H Ouyang… - EMBO molecular …, 2017 - embopress.org
The combination therapy of lumacaftor and ivacaftor (Orkambi®) is approved for patients
bearing the major cystic fibrosis (CF) mutation: ΔF508. It has been predicted that Orkambi® …
bearing the major cystic fibrosis (CF) mutation: ΔF508. It has been predicted that Orkambi® …
Identifying the molecular target sites for CFTR potentiators GLPG1837 and VX-770
The past two decades have witnessed major breakthroughs in developing compounds that
target the chloride channel CFTR for the treatment of patients with cystic fibrosis. However …
target the chloride channel CFTR for the treatment of patients with cystic fibrosis. However …
[HTML][HTML] One size does not fit all: the past, present and future of cystic fibrosis causal therapies
MM Ensinck, MS Carlon - Cells, 2022 - mdpi.com
Cystic fibrosis (CF) is the most common monogenic disorder, caused by mutations in the CF
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …
transmembrane conductance regulator (CFTR) gene. Over the last 30 years, tremendous …
Identification of binding sites for ivacaftor on the cystic fibrosis transmembrane conductance regulator
O Laselva, Z Qureshi, ZW Zeng, EV Petrotchenko… - Iscience, 2021 - cell.com
Summary Ivacaftor (VX-770) was the first cystic fibrosis transmembrane conductance
regulator (CFTR) modulatory drug approved for the treatment of patients with cystic fibrosis …
regulator (CFTR) modulatory drug approved for the treatment of patients with cystic fibrosis …
A common mechanism for CFTR potentiators
Cystic fibrosis (CF) is a channelopathy caused by loss-of-function mutations of the cystic
fibrosis transmembrane conductance regulator (CFTR) gene, which encodes a …
fibrosis transmembrane conductance regulator (CFTR) gene, which encodes a …
Timing of CFTR pore opening and structure of its transition state
In CFTR, the chloride ion channel mutated in cystic fibrosis (CF) patients, pore opening is
coupled to ATP-binding-induced dimerization of two cytosolic nucleotide binding domains …
coupled to ATP-binding-induced dimerization of two cytosolic nucleotide binding domains …
The molecular evolution of function in the CFTR chloride channel
DT Infield, KM Strickland, A Gaggar… - Journal of General …, 2021 - rupress.org
The ATP-binding cassette (ABC) transporter superfamily includes many proteins of clinical
relevance, with genes expressed in all domains of life. Although most members use the …
relevance, with genes expressed in all domains of life. Although most members use the …
Structure basis of CFTR folding, function and pharmacology
TC Hwang, I Braakman, P van der Sluijs… - Journal of Cystic …, 2023 - Elsevier
The root cause of cystic fibrosis (CF), the most common life-shortening genetic disease in
the Caucasian population, is the loss of function of the CFTR protein, which serves as a …
the Caucasian population, is the loss of function of the CFTR protein, which serves as a …