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Article

Distinct proteostasis states drive pharmacologic chaperone susceptibility for cystic fibrosis transmembrane conductance regulator misfolding mutants.

Molecular biology of the cell - 1 Jun 2022

McDonald Eli Fritz, Sabusap Carleen Mae P, Kim Minsoo, Plate Lars

Abstract excerpt

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 CF transmembrane conductance regulator (CFTR). VX-809 corrects folding of F508del CFTR, the most...

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Identifiers

PMID: 35389766
DOI: 10.1091/mbc.E21-11-0578
PMCID: PMC9561855

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