Article
General trends in the effects of VX-661 and VX-445 on the plasma membrane expression of clinical CFTR variants.
Cell chemical biology - 15 Jun 2023
McKee Andrew G, McDonald Eli F, Penn Wesley D, Kuntz Charles P, Noguera Karen, Chamness Laura M, Roushar Francis J, Meiler Jens, Oliver Kathryn E, Plate Lars, Schlebach Jonathan P
Abstract excerpt
Cystic fibrosis (CF) is caused by mutations that compromise the expression and/or function of the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel. Most people with CF harbor a common misfolded variant (ΔF508) that can be partially rescued by therapeutic "correctors" that restore its expression. Nevertheless, many other CF variants are insensitive to correctors. Using deep mutational...
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