Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine

Emerging Therapeutic Approaches for Cystic Fibrosis. From Gene Editing to Personalized Medicine

An improved understanding of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein structure and the consequences of CFTR gene mutations have allowed the development of novel therapies targeting specific defects underlying CF. Some strategies are mutation specific and have alre...

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Bibliographic Details
Main Authors: Iwona Pranke, Anita Golec, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus
Format: Article
Language:English
Published: Frontiers Media S.A. 2019-02-01
Series:Frontiers in Pharmacology
Subjects:
cystic fibrosis
CFTR
ivacaftor
CFTR modulator
gene therapy
Online Access:https://www.frontiersin.org/article/10.3389/fphar.2019.00121/full

Internet

https://www.frontiersin.org/article/10.3389/fphar.2019.00121/full

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