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Article

Barriers to the Pharmacologic Rescue of W1282X CFTR.

Biochemistry - 6 Jan 2026

Manfredi Candela, Rab Andras, Joshi Disha, Winters Ashlyn G, Jackson JaNise J, Molina Sam, Koval Michael, Khazanov Netaly, Jacobson Madison, Oliver Kathryn, Senderowitz Hanoch, Sorscher Eric J, Hong Jeong S

Abstract excerpt

W1282X CFTR is the most prevalent CF-causing variant among cystic fibrosis patients of Ashkenazi descent and a mutational defect for which targeted drug therapy is not available. We show that administration of the potentiator VX-770 can augment levels of truncated W1282X CFTR in the plasma membrane, demonstrating that an established gating activator (i.e., "potentiator") also rescues W1282X protein expression and...

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Identifiers

PMID: 41386645
DOI: 10.1021/acs.biochem.5c00590
PMCID: PMC12781105

Genes

CFTR

Variants

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026.

Related research

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Research trail→

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Barriers to the Pharmacologic Rescue of W1282X CFTR.PMID 41386645Read ↗

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Breakthrough therapies: Cystic fibrosis (CF) potentiators and correctors.Semantic proximity Read ↗

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770.Semantic proximity Read ↗

Managing the underlying cause of cystic fibrosis: a future role for potentiators and correctors.Semantic proximity Read ↗

Mechanism-based corrector combination restores ΔF508-CFTR folding and function.Semantic proximity Read ↗

Synergistic Potentiation of Cystic Fibrosis Transmembrane Conductance Regulator Gating by Two Chemically Distinct Potentiators, Ivacaftor (VX-770) and 5-Nitro-2-(3-Phenylpropylamino) Benzoate.Semantic proximity Read ↗

PTI-801 (posenacaftor) shares a common mechanism with VX-445 (elexacaftor) to rescue p.Phe508del-CFTR.Semantic proximity Read ↗

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