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Article

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

JCI insight - 17 Sept 2020

Veit Guido, Roldan Ariel, Hancock Mark A, Da Fonte Dillon F, Xu Haijin, Hussein Maytham, Frenkiel Saul, Matouk Elias, Velkov Tony, Lukacs Gergely L

Abstract excerpt

Based on its clinical benefits, Trikafta - the combination of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor), and the gating potentiator VX-770 (ivacaftor) - was FDA approved for treatment of patients with cystic fibrosis (CF) carrying deletion of phenylalanine at position 508 (F508del) of the CF transmembrane conductance regulator (CFTR) on at least 1 allele. Neither the mechanism of action of...

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Identifiers

PMID: 32853178
DOI: 10.1172/jci.insight.139983
PMCID: PMC7526550

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Allosteric folding correction of F508del and rare CFTR mutants by elexacaftor-tezacaftor-ivacaftor (Trikafta) combination.PMID 32853178Read ↗

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