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Genome Editing of Monogenic Neuromuscular Diseases: A Systematic Review

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 identi...

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Bibliographic Details
Published in:JAMA Neurol
Main Authors: Long, Chengzu, Amoasii, Leonela, Bassel-Duby, Rhonda, Olson, Eric N.
Format: Artigo
Language:Inglês
Published: 2016
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC5695221/
https://ncbi.nlm.nih.gov/pubmed/27668807
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1001/jamaneurol.2016.3388
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