A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

A Precision Medicine Approach to Optimize Modulator Therapy for Rare CFTR Folding Mutants

Trikafta, a triple-combination drug, consisting of folding correctors VX-661 (tezacaftor), VX-445 (elexacaftor) and the gating potentiator VX-770 (ivacaftor) provided unprecedented clinical benefits for patients with the most common cystic fibrosis (CF) mutation, F508del. Trikafta indications were r...

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Bibliographic Details
Main Authors: Guido Veit, Tony Velkov, Haijin Xu, Nathalie Vadeboncoeur, Lara Bilodeau, Elias Matouk, Gergely L. Lukacs
Format: Article
Language:English
Published: MDPI AG 2021-07-01
Series:Journal of Personalized Medicine
Subjects:
cystic fibrosis
cystic fibrosis transmembrane conducatance regulator (CFTR)
CFTR modulator combination
primary human nasal epithelia
CFTR missense mutations
precision medicine
Online Access:https://www.mdpi.com/2075-4426/11/7/643

Internet

https://www.mdpi.com/2075-4426/11/7/643

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