Rescue of Mutant CFTR Channel Activity by Investigational Co-Potentiator Therapy

Rescue of Mutant CFTR Channel Activity by Investigational Co-Potentiator Therapy

<b>Background:</b> The potentiator VX-770 (ivacaftor) has been approved as a monotherapy for over 95 cystic fibrosis (CF)-causing variants associated with gating/conductance defects of the CF transmembrane conductance regulator (CFTR) channel. However, despite its therapeutic success, VX...

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Bibliographic Details
Main Authors: Mafalda Bacalhau, Filipa C. Ferreira, Marcelo Folhadella M. F. Azevedo, Talita P. Rosa, Camilla D. Buarque, Miquéias Lopes-Pacheco
Format: Article
Language:English
Published: MDPI AG 2025-01-01
Series:Biomedicines
Subjects:
CFTR modulator
cystic fibrosis
drug development
potentiator
rare mutation
R334W
Online Access:https://www.mdpi.com/2227-9059/13/1/82
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