Gene Editing

Publication Title: 
JAMA neurology

Importance: Muscle weakness, the most common symptom of neuromuscular disease, may result from muscle dysfunction or may be caused indirectly by neuronal and neuromuscular junction abnormalities. To date, more than 780 monogenic neuromuscular diseases, linked to 417 different genes, have been identified in humans.

Long, Chengzu
Amoasii, Leonela
Bassel-Duby, Rhonda
Olson, Eric N.
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