CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives

CRISPR-Based Therapeutic Gene Editing for Duchenne Muscular Dystrophy: Advances, Challenges and Perspectives

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease arising from loss-of-function mutations in the <i>dystrophin</i> gene and characterized by progressive muscle degeneration, respiratory insufficiency, cardiac failure, and premature death by the age of thirty. Albeit DMD...

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Bibliographic Details
Main Authors: Guofang Chen, Tingyi Wei, Hui Yang, Guoling Li, Haisen Li
Format: Article
Language:English
Published: MDPI AG 2022-09-01
Series:Cells
Subjects:
DMD
CRISPR
dystrophin
gene therapy
double cut
single cut
Online Access:https://www.mdpi.com/2073-4409/11/19/2964

Internet

https://www.mdpi.com/2073-4409/11/19/2964

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