Gene therapy for cystic fibrosis: Challenges and prospects

Gene therapy for cystic fibrosis: Challenges and prospects

Cystic fibrosis (CF) is a life-threatening autosomal-recessive disease caused by mutations in a single gene encoding cystic fibrosis transmembrane conductance regulator (CFTR). CF effects multiple organs, and lung disease is the primary cause of mortality. The median age at death from CF is in the e...

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Bibliographic Details
Main Authors: Hongshu Sui, Xinghua Xu, Yanping Su, Zhaoqing Gong, Minhua Yao, Xiaocui Liu, Ting Zhang, Ziyao Jiang, Tianhao Bai, Junzuo Wang, Jingjun Zhang, Changlong Xu, Mingjiu Luo
Format: Article
Language:English
Published: Frontiers Media S.A. 2022-10-01
Series:Frontiers in Pharmacology
Subjects:
cystic fibrosis
gene therapy
viral vectors
animal models
airway delivery
Online Access:https://www.frontiersin.org/articles/10.3389/fphar.2022.1015926/full

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