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Article

Rescue of multiple class II CFTR mutations by elexacaftor+tezacaftor+ivacaftor mediated in part by the dual activities of elexacaftor as both corrector and potentiator.

The European respiratory journal - 1 Jun 2021

Laselva Onofrio, Bartlett Claire, Gunawardena Tarini N A, Ouyang Hong, Eckford Paul D W, Moraes Theo J, Bear Christine E, Gonska Tanja

Abstract excerpt

Positive results in pre-clinical studies of the triple combination of elexacaftor, tezacaftor and ivacaftor, performed in airway epithelial cell cultures obtained from patients harbouring the class II cystic fibrosis transmembrane conductance regulator (CFTR) mutation F508del-CFTR, translated to impressive clinical outcomes for subjects carrying this mutation in clinical trials and approval of Trikafta.Encouraged...

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Identifiers

PMID: 33303536
DOI: 10.1183/13993003.02774-2020
PMCID: PMC8209484

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