Lumacaftor inhibits channel activity of rescued F508del cystic fibrosis transmembrane conductance regulator

2025/05/07 by Adam Ambrosetti, Adam D. Ambrosetti, Zachary J. Hagedorn +15
Medicine · #Cystic Fibrosis Research Advances

paper · doi:10.1152/ajplung.00287.2024

openalex publication_date 2025/05/07 · openalex created_date 2025/10/10 · openalex updated_date 2026/08/01

Abstract

Small-molecule correctors bind to F508del cystic fibrosis transmembrane conductance regulator (CFTR) and restore its trafficking to the plasma membrane to function as an anion channel. Despite its high efficacy as a corrector, lumacaftor inhibits the channel opening of rescued F508del CFTR, making it a weak CFTR modulator. The current work highlights the impact of CFTR correctors on the channel activity of rescued F508del CFTR as an important variable in the efficacy of modulator therapy.

Citations

• Characteristics of a Human Cell Line Transformed by DNA from Human Adenovirus Type 5

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