Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases

Current Genetic Survey and Potential Gene-Targeting Therapeutics for Neuromuscular Diseases

Neuromuscular diseases (NMDs) belong to a class of functional impairments that cause dysfunctions of the motor neuron-muscle functional axis components. Inherited monogenic neuromuscular disorders encompass both muscular dystrophies and motor neuron diseases. Understanding of their causative genetic...

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Bibliographic Details
Main Authors: Wei Chiu, Ya-Hsin Hsun, Kao-Jung Chang, Aliaksandr A. Yarmishyn, Yu-Jer Hsiao, Yueh Chien, Chian-Shiu Chien, Chun Ma, Yi-Ping Yang, Ping-Hsing Tsai, Shih-Hwa Chiou, Ting-Yi Lin, Hao-Min Cheng
Format: Article
Language:English
Published: MDPI AG 2020-12-01
Series:International Journal of Molecular Sciences
Subjects:
gene therapy
CRISPR
neuromuscular disease
duchenne muscular dystrophy
spinal muscular atrophy
Online Access:https://www.mdpi.com/1422-0067/21/24/9589

Internet

https://www.mdpi.com/1422-0067/21/24/9589

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