Additive Potentiation of R334W-CFTR Function by Novel Small Molecules

Additive Potentiation of R334W-CFTR Function by Novel Small Molecules

The R334W (c.1000C>T, p.Arg334Trp) is a rare cystic fibrosis (CF)-causing mutation for which no causal therapy is currently approved. This mutation leads to a significant reduction of CF transmembrane conductance regulator (CFTR) channel conductance that still allows for residual function. Potent...

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Bibliographic Details
Main Authors: Mafalda Bacalhau, Filipa C. Ferreira, Iris A. L. Silva, Camilla D. Buarque, Margarida D. Amaral, Miquéias Lopes-Pacheco
Format: Article
Language:English
Published: MDPI AG 2023-01-01
Series:Journal of Personalized Medicine
Subjects:
CFTR modulator
co-potentiation
cystic fibrosis
drug discovery
intestinal organoids
potentiator
Online Access:https://www.mdpi.com/2075-4426/13/1/102

Internet

https://www.mdpi.com/2075-4426/13/1/102

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