Article
Elexacaftor/VX-445-mediated CFTR interactome remodeling reveals differential correction driven by mutation-specific translational dynamics.
The Journal of biological chemistry - 1 Oct 2023
Kim Minsoo, McDonald Eli Fritz, Sabusap Carleen Mae P, Timalsina Bibek, Joshi Disha, Hong Jeong S, Rab Andras, Sorscher Eric J, Plate Lars
Abstract excerpt
Cystic fibrosis (CF) is one of the most prevalent lethal genetic diseases with over 2000 identified mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Pharmacological chaperones such as lumacaftor (VX-809), tezacaftor (VX-661), and elexacaftor (VX-445) treat mutation-induced defects by stabilizing CFTR and are called correctors. These correctors improve proper folding and thus...
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