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

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...

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Detalhes bibliográficos
Publicado no:JCI Insight
Main Authors: Veit, Guido, Roldan, Ariel, Hancock, Mark A., Da Fonte, Dillon F., Xu, Haijin, Hussein, Maytham, Frenkiel, Saul, Matouk, Elias, Velkov, Tony, Lukacs, Gergely L.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society for Clinical Investigation 2020
Assuntos:
Research Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7526550/
https://ncbi.nlm.nih.gov/pubmed/32853178
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/jci.insight.139983
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