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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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Detalhes bibliográficos
Publicado no:JAMA Neurol
Main Authors: Long, Chengzu, Amoasii, Leonela, Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Artigo
Idioma:Inglês
Publicado em: 2016
Assuntos:
Acesso em linha: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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