Two Small Molecules Restore Stability to a Subpopulation of the Cystic Fibrosis Transmembrane Conductance Regulator with the Predominant Disease-causing Mutation

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...

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Detalhes bibliográficos
Publicado no:J Biol Chem
Main Authors: Meng, Xin, Wang, Yiting, Wang, Xiaomeng, Wrennall, Joe A., Rimington, Tracy L., Li, Hongyu, Cai, Zhiwei, Ford, Robert C., Sheppard, David N.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Biochemistry and Molecular Biology 2017
Assuntos:
Molecular Bases of Disease
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC5339754/
https://ncbi.nlm.nih.gov/pubmed/28087700
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1074/jbc.M116.751537
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