Article
Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation.
The Journal of biological chemistry - 3 Mar 2017
Meng Xin, Wang Yiting, Wang Xiaomeng, Wrennall Joe A, Rimington Tracy L, Li Hongyu, Cai Zhiwei, Ford Robert C, Sheppard David N
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations that disrupt the plasma membrane expression, stability, and function of the cystic fibrosis transmembrane conductance regulator (CFTR) Cl- channel. Two small molecules, the CFTR corrector lumacaftor and the potentiator ivacaftor, are now used clinically to treat CF, although some studies suggest that they have counteracting effects on CFTR stability. Here, we...
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