Pharmacology & ToxicologyScience AdvancesPeer-reviewed
A Novel, Uniquely Efficacious Type of CFTR Corrector with Complementary Mode of Action
Valentina Marchesin, Lucile Monnier, Peter Blattmann…·John 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.
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