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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發表在:	Frontiers in Pharmacology
Main Authors:	Iwona Pranke, Anita Golec, Alexandre Hinzpeter, Aleksander Edelman, Isabelle Sermet-Gaudelus
格式:	Article
語言:	英语
出版:	Frontiers Media S.A. 2019-02-01
主題:	cystic fibrosis CFTR ivacaftor CFTR modulator gene therapy
在線閱讀:	https://www.frontiersin.org/article/10.3389/fphar.2019.00121/full