Article

Distinct proteostasis states drive pharmacologic chaperone susceptibility for Cystic Fibrosis Transmembrane Conductance Regulator misfolding mutants

2021-09-09

Abstract excerpt

<h4>ABSTRACT</h4> Pharmacological chaperones represent a class of therapeutic compounds for treating protein misfolding diseases. One of the most prominent examples is the FDA-approved pharmacological chaperone lumacaftor (VX-809), which has transformed cystic fibrosis (CF) therapy. CF is a fatal disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR). VX-809 corrects folding...

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Literature Corpus work: 7ee0d3af-b34f-5977-9d99-58d3cebd3c01
DOI: 10.1101/2021.09.09.459524

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