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Article

Mechanism-based corrector combination restores ΔF508-CFTR folding and function.

Nature chemical biology - 1 Jul 2013

Okiyoneda Tsukasa, Veit Guido, Dekkers Johanna F, Bagdany Miklos, Soya Naoto, Xu Haijin, Roldan Ariel, Verkman Alan S, Kurth Mark, Simon Agnes, Hegedus Tamas, Beekman Jeffrey M, Lukacs Gergely L

Abstract excerpt

The most common cystic fibrosis mutation, ΔF508 in nucleotide binding domain 1 (NBD1), impairs cystic fibrosis transmembrane conductance regulator (CFTR)-coupled domain folding, plasma membrane expression, function and stability. VX-809, a promising investigational corrector of ΔF508-CFTR misprocessing, has limited clinical benefit and an incompletely understood mechanism, hampering drug development. Given the...

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Identifiers

PMID: 23666117
DOI: 10.1038/nchembio.1253
PMCID: PMC3840170

Genes

CFTR

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-08.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

Related research

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Research trail→

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Mechanism-based corrector combination restores ΔF508-CFTR folding and function.PMID 23666117Read ↗

Pharmacological chaperones improve intra-domain stability and inter-domain assembly via distinct binding sites to rescue misfolded CFTR.Semantic proximity Read ↗

Synergy-based small-molecule screen using a human lung epithelial cell line yields ΔF508-CFTR correctors that augment VX-809 maximal efficacy.Semantic proximity Read ↗

Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination.Semantic proximity Read ↗

Correctors of the Major Cystic Fibrosis Mutant Interact through Membrane-Spanning Domains.Semantic proximity Read ↗

Elucidation of Global Trends in the Effects of VX-661 and VX-445 on the Expression of Clinical CFTR VariantsSemantic proximity Read ↗

Folding correctors can restore CFTR posttranslational folding landscape by allosteric domain-domain coupling.Semantic proximity Read ↗

Proteostasis Landscapes of Cystic Fibrosis Variants Reveals Drug Response VulnerabilitySemantic proximity Read ↗

General trends in the effects of VX-661 and VX-445 on the plasma membrane expression of clinical CFTR variants.Semantic proximity Read ↗

Correction of both NBD1 energetics and domain interface is required to restore ΔF508 CFTR folding and function.Semantic proximity Read ↗

A new corrector rescues F508del-CFTR folding through stabilization of the TMD1-NBD1 linkerSemantic proximity Read ↗

Computational Exploration of Potential CFTR Binding Sites for Type I Corrector Drugs.Semantic proximity Read ↗

Structure-guided combination of novel CFTR correctors to improve the function of F508del-CFTR in airway epithelial cells.Semantic proximity Read ↗

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