A Novel, Uniquely Efficacious Type of CFTR Corrector with Complementary Mode of Action
V. Marchesin, L. Monnier, P. Blattmann, F. Chevillard, C. Kuntz, C. Forny, J. Kamper, R. Studer, A. Bossu, E. Ertel, O. Nayler, C. Brotschi, J. T. Williams, J. Gatfield
This paper describes a novel type-IV CFTR corrector that improves folding and trafficking of F508del-CFTR, including previously resistant mutations, through a unique binding site and mechanism.
bioRxiv · Aug 15, 2023Pharmacology & Toxicology