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Article

Nanomolar-potency 'co-potentiator' therapy for cystic fibrosis caused by a defined subset of minimal function CFTR mutants.

Scientific reports - 27 Nov 2019

Phuan Puay-Wah, Tan Joseph-Anthony, Rivera Amber A, Zlock Lorna, Nielson Dennis W, Finkbeiner Walter E, Haggie Peter M, Verkman Alan S

Abstract excerpt

Available CFTR modulators provide no therapeutic benefit for cystic fibrosis (CF) caused by many loss-of-function mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) chloride channel, including N1303K. We previously introduced the concept of 'co-potentiators' (combination-potentiators) to rescue CFTR function in some minimal function CFTR mutants. Herein, a screen of ~120,000 drug-like...

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Identifiers

PMID: 31776420
DOI: 10.1038/s41598-019-54158-2
PMCID: PMC6881293

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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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