Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770
نویسندگان
چکیده
منابع مشابه
Restoration of R117H CFTR folding and function in human airway cells through combination treatment with VX-809 and VX-770.
Cystic fibrosis (CF) is a lethal recessive genetic disease caused primarily by the F508del mutation in the CF transmembrane conductance regulator (CFTR). The potentiator VX-770 was the first CFTR modulator approved by the FDA for treatment of CF patients with the gating mutation G551D. Orkambi is a drug containing VX-770 and corrector VX809 and is approved for treatment of CF patients homozygou...
متن کاملDirect Binding of Vx-809 to Human Cftr Nbd1
Molecular Structure and Function, Peter Gilgan Center for Research and Learning, Hospital for Sick Children (R.P.H, J.E.D., P.A.C., J.F.-K.) and Department of Biochemistry, University of Toronto (J.F.-K.), Toronto, ON, Canada; Center for Structural Biology (Z.Y., C.G.B.) and Department of Chemistry (C.G.B.), University of Alabama at Birmingham, Birmingham, AL, USA; Department of Physiology at U...
متن کاملPseudomonas aeruginosa Reduces VX-809 Stimulated F508del-CFTR Chloride Secretion by Airway Epithelial Cells
BACKGROUND P. aeruginosa is an opportunistic pathogen that chronically infects the lungs of 85% of adult patients with Cystic Fibrosis (CF). Previously, we demonstrated that P. aeruginosa reduced wt-CFTR Cl secretion by airway epithelial cells. Recently, a new investigational drug VX-809 has been shown to increase F508del-CFTR Cl secretion in human bronchial epithelial (HBE) cells, and, in comb...
متن کاملCorrector VX-809 stabilizes the first transmembrane domain of CFTR.
Processing mutations that inhibit folding and trafficking of CFTR are the main cause of cystic fibrosis (CF). A potential CF therapy would be to repair CFTR processing mutants. It has been demonstrated that processing mutants of P-glycoprotein (P-gp), CFTR's sister protein, can be efficiently repaired by a drug-rescue mechanism. Many arginine suppressors that mimic drug-rescue have been identif...
متن کاملEffect of VX-770 (ivacaftor) and OAG on Ca2+ influx and CFTR activity in G551D and F508del-CFTR expressing cells.
BACKGROUND TRPC6 has been proposed to be responsible for the abnormal OAG-dependent Ca(2+) influx in cystic fibrosis (CF) cells and we hypothesized that it interacts with CFTR. Here, we investigated how this functional complex operates in CF and non-CF epithelial cells. METHODS Chinese hamster ovary (CHO) cells stably transfected with pNut vector containing wild type CFTR (CHO-WT), F508del-CF...
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ژورنال
عنوان ژورنال: American Journal of Physiology-Lung Cellular and Molecular Physiology
سال: 2016
ISSN: 1040-0605,1522-1504
DOI: 10.1152/ajplung.00186.2016