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CFTR

Transcriptomic analysis of CFTR-impaired endothelial cells reveals a pro-inflammatory phenotype

16 views | Feb 01 2021

Mathias Declercq et al. thought that normalisation of vascular inflammation might be a novel therapeutic strategy to ameliorate the disease severity of CF. [Read the Full Post]

Real-World Safety of CFTR Modulators in the Treatment of Cystic Fibrosis: A Systematic Review

18 views | Jan 05 2021

Renée V E Dagenais et al. thought that pharmacists played a key role in the safe initiation and monitoring of people with CF on CFTR modulator therapies. [Read the Full Post]

An integrated drug repurposing strategy for the rapid identification of potential SARS-CoV-2 viral inhibitors

43 views | Sep 01 2020

Alfonso Trezza et al. showed that Simeprevir and Lumacaftor bound the receptor-binding domain of the Spike protein with high affinity and prevented ACE2 interaction. [Read the Full Post]

The impact of ivacaftor on sinonasal pathology in S1251N-mediated cystic fibrosis patients

96 views | Jul 21 2020

Romee Gostelie et al. found that Ivacaftor appeared to improve sinonasal outcome parameters and thereby sinonasal health in patients with cystic fibrosis with an S1251N mutation. [Read the Full Post]

Corrector VX-809 promotes interactions between cytoplasmic loop one and the first nucleotide-binding domain of CFTR

165 views | Nov 12 2019

Loo TW et al. suggested that the mechanism by which VX-809 promotes maturation and stability of CFTR is by promoting CL1/NBD1 interactions. [Read the Full Post]

Some gating potentiators, including VX-770, diminish ΔF508-CFTR functional expression

343 views | Sep 29 2019

Veit G et al. indicated that VX-770 diminished the folding efficiency and the metabolic stability of ΔF508-CFTR at the endoplasmic reticulum (ER) and post-ER compartments, respectively, causing reduced cell surface ΔF508-CFTR density and function. VX-770-induced destabilization of ΔF508-CFTR was influenced by second-site suppressor mutations of the folding defect and was prevented by stabilization of the nucleotide-binding domain 1 (NBD1)-NBD2 interface. The reduced correction efficiency of ΔF508-CFTR, as well as of two other processing mutations in the presence of VX-770, suggests the need for further optimization of potentiators to maximize the clinical benefit of corrector-potentiator combination therapy in CF. [Read the Full Post]

Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation

322 views | Jan 18 2019

Accurso FJ et al. evaluated the safety and adverse-event profile of VX-770 showed that VX-770 was associated with within-subject improvements in CFTR and lung function. These findings provide support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis. [Read the Full Post]

Tezacaftor-Ivacaftor in Residual-Function Heterozygotes with Cystic Fibrosis

361 views | Jan 17 2019

Rowe SM et al. showed that CFTR modulator therapy with tezacaftor-ivacaftor or ivacaftor alone was efficacious in patients with cystic fibrosis who were heterozygous for the Phe508del deletion and a CFTR residual-function mutation. [Read the Full Post]

CFTR protects against vascular inflammation and atherogenesis in apolipoprotein E-deficient mice

301 views | Jan 08 2019

Li Z et al. suggested that CFTR may present a potential therapeutic target for the treatment of vascular inflammation and development of atherosclerotic disease. [Read the Full Post]

High-expressing cystic fibrosis transmembrane conductance regulator interacts with histone deacetylase 2 to promote the development of Ph+ leukemia through the HDAC2-mediated PTEN pathway

0 views | Oct 04 2018

Yan T et al. provides strong evidence that high-expression CFTR plays an important role in the development of Ph+ leukemia through the HDAC2-mediated PTEN pathway. [Read the Full Post]