Elexacaftor is a CFTR potentiator and acts synergistically with ivacaftor during acute and chronic treatment

Abstract Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR), which lead to early death due to progressive lung disease. The development of small-molecule modulators that directly interact with CFTR to aid in protein folding (“correctors”) an...

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Autores principales: Ciaran A. Shaughnessy, Pamela L. Zeitlin, Preston E. Bratcher
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2021
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Acceso en línea:https://doaj.org/article/ada0900b04f2474491bb981f326adf27
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