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Article

Emerging preclinical modulators developed for F508del-CFTR have the potential to be effective for ORKAMBI resistant processing mutants.

Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society - 1 Jan 2021

Laselva Onofrio, Bartlett Claire, Popa Alec, Ouyang Hong, Gunawardena Tarini N A, Gonska Tanja, Moraes Theo J, Bear Christine E

Abstract excerpt

BACKGROUND: F508del is prototypical of Class 2 CFTR mutations associated with protein misprocessing and reduced function. Corrector compounds like lumacaftor partially rescue the processing defect of F508del-CFTR whereas potentiators like ivacaftor, enhance its channel activity once trafficked to the cell surface. We asked if emerging modulators developed for F508del-CFTR can rescue Class 2 mutations previously...

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Identifiers

PMID: 32741662
DOI: 10.1016/j.jcf.2020.07.015

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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.

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