Article

Mechanism of dual pharmacological correction and potentiation of human CFTR

2022-10-11

Abstract excerpt

<h4>ABSTRACT</h4> Cystic fibrosis (CF) is caused by mutations in a chloride channel called the human Cystic Fibrosis Transmembrane Conductance Regulator (hCFTR). We used cryo-EM global conformational ensemble reconstruction to characterize the mechanism by which the breakthrough drug VX445 (Elexacaftor) simultaneously corrects both protein-folding and channel-gating defects caused by CF mutations. VX445 drives hC...

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Literature Corpus work: 24ebe7b4-1a59-5789-acf3-9b8622f4d981
DOI: 10.1101/2022.10.10.510913

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