Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTRMutation
نویسندگان
چکیده
منابع مشابه
Effect of VX-770 in persons with cystic fibrosis and the G551D-CFTR mutation.
BACKGROUND A new approach in the treatment of cystic fibrosis involves improving the function of mutant cystic fibrosis transmembrane conductance regulator (CFTR). VX-770, a CFTR potentiator, has been shown to increase the activity of wild-type and defective cell-surface CFTR in vitro. METHODS We randomly assigned 39 adults with cystic fibrosis and at least one G551D-CFTR allele to receive or...
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...
متن کاملClinical phenotype of cystic fibrosis patients with the G551D mutation.
BACKGROUND Data on whether the phenotype of cystic fibrosis (CF) patients with compound heterozygocity for G551D (Gly551Asp) differs from patients with F508del (Phe508del) homozygous mutations is divergent. AIM We hypothesized that CF patients with the G551D mutation would have less severe disease than F508del homozygotes. DESIGN We compared the clinical phenotype of adult patients with a G...
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ژورنال
عنوان ژورنال: New England Journal of Medicine
سال: 2010
ISSN: 0028-4793,1533-4406
DOI: 10.1056/nejmoa0909825