Article

A Novel, Uniquely Efficacious Type of CFTR Corrector with Complementary Mode of Action

2023-08-16

Abstract excerpt

<h4>ABSTRACT</h4> Three distinct pharmacological corrector types (I, II, III) with different binding sites and additive behaviour only partially rescue the F508del-CFTR folding and trafficking defect observed in cystic fibrosis. Here, we describe novel, uniquely effective, macrocyclic CFTR correctors that were additive to the known corrector types, thus exerting a new, complementary “type-IV” corrector mechanism....

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Literature Corpus work: bdf2bc36-e1a5-572e-ba63-171f4c531c8a
DOI: 10.1101/2023.08.15.552928

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