Increased efficacy of VX-809 in different cellular systems results from an early stabilization effect of F508del-CFTR.

ABSTRACT: Cystic fibrosis (CF), the most common recessive autosomal disease among Caucasians, is caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. The most common mutation, F508del, leads to CFTR impaired plasma membrane trafficking. Therapies modulating CFTR basic defect are emerging, such as VX-809, a corrector of F508del-CFTR traffic which just succeeded in a Phase III clinical trial. We recently showed that VX-809 is additive to two other correctors (VRT-325 and compound 4a). Here, we aimed to determine whether the differential rescuing by these compounds results from cell-specific factors or rather from distinct effects at the early biogenesis and/or processing. The rescuing efficiencies of the above three correctors were first compared in diffe

SUBMITTER: Farinha CM 

PROVIDER: S-EPMC4492728 | biostudies-literature | 2015 Aug

REPOSITORIES: biostudies-literature