General Trends in the Calnexin-Dependent Expression and Pharmacological Rescue of Clinical CFTR Variants

Abstract excerpt

Cystic Fibrosis (CF) is a genetic disease caused by mutations in the cystic fibrosis transmembrane conductance regulator gene (CFTR). Though most people with CF have one or two copies of the delF508 mutation, there are hundreds of other distinct CF mutations that vary in their mechanistic effects and response to therapeutics. Endogenous chaperones are known to have divergent effects on the druggability of CF varia...

Topics

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• Benzodioxoles
• Calnexin
• Cell Membrane
• Cystic Fibrosis
• Cystic Fibrosis Transmembrane Conductance Regulator
• Gene Knockdown Techniques
• Mutation
• Pyrazoles

Identifiers and source

Literature Corpus work

0eac8a21-f426-5f93-900d-5e09a20bb636

DOI

10.1101/2025.04.03.647093