Network pharmacology: curing causal mechanisms instead of treating symptoms
For complex diseases, most drugs are highly ineffective, and the success rate of drug
discovery is in constant decline. While low quality, reproducibility issues, and translational …
discovery is in constant decline. While low quality, reproducibility issues, and translational …
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 …
[HTML][HTML] Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination
Based on its clinical benefits, Trikafta—the combination of folding correctors VX-661
(tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor)—was FDA …
(tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor)—was FDA …
Effects of Elexacaftor/Tezacaftor/Ivacaftor Therapy on CFTR Function in Patients with Cystic Fibrosis and One or Two F508del Alleles
SY Graeber, C Vitzthum, ST Pallenberg… - American journal of …, 2022 - atsjournals.org
Rationale: The CFTR (cystic fibrosis transmembrane conductance regulator) modulator
combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical …
combination elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) was shown to improve clinical …
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
CE Wainwright, JS Elborn, BW Ramsey… - … England Journal of …, 2015 - Mass Medical Soc
Background Cystic fibrosis is a life-limiting disease that is caused by defective or deficient
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is …
cystic fibrosis transmembrane conductance regulator (CFTR) protein activity. Phe508del is …
Cystic fibrosis genetics: from molecular understanding to clinical application
GR Cutting - Nature Reviews Genetics, 2015 - nature.com
The availability of the human genome sequence and tools for interrogating individual
genomes provide an unprecedented opportunity to apply genetics to medicine. Mendelian …
genomes provide an unprecedented opportunity to apply genetics to medicine. Mendelian …
Lipid nanoparticle-delivered chemically modified mRNA restores chloride secretion in cystic fibrosis
The promise of gene therapy for the treatment of cystic fibrosis has yet to be fully clinically
realized despite years of effort toward correcting the underlying genetic defect in the cystic …
realized despite years of effort toward correcting the underlying genetic defect in the cystic …
Folding and misfolding of human membrane proteins in health and disease: from single molecules to cellular proteostasis
Advances over the past 25 years have revealed much about how the structural properties of
membranes and associated proteins are linked to the thermodynamics and kinetics of …
membranes and associated proteins are linked to the thermodynamics and kinetics of …
Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis
JF Dekkers, G Berkers, E Kruisselbrink… - Science translational …, 2016 - science.org
Identifying subjects with cystic fibrosis (CF) who may benefit from cystic fibrosis
transmembrane conductance regulator (CFTR)–modulating drugs is time-consuming, costly …
transmembrane conductance regulator (CFTR)–modulating drugs is time-consuming, costly …
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 …