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Article

A uniquely efficacious type of CFTR corrector with complementary mode of action.

Science advances - 1 Mar 2024

Marchesin Valentina, Monnier Lucile, Blattmann Peter, Chevillard Florent, Kuntz Christine, Forny Camille, Kamper Judith, Studer Rolf, Bossu Alexandre, Ertel Eric A, Nayler Oliver, Brotschi Christine, Williams Jodi T, Gatfield John

Abstract excerpt

Three distinct pharmacological corrector types (I, II, III) with different binding sites and additive behavior only partially rescue the F508del-cystic fibrosis transmembrane conductance regulator (CFTR) folding and trafficking defect observed in cystic fibrosis. We describe uniquely effective, macrocyclic CFTR correctors that were additive to the known corrector types, exerting a complementary "type IV"...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Identifiers

PMID: 38427726
DOI: 10.1126/sciadv.adk1814

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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