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AAV-based gene therapies for the muscular dystrophies

Muscular dystrophy (MD) is a group of progressive genetic diseases affecting the musculature that are characterized by inflammatory infiltrates, necrosis and connective tissue and fat replacement of the affected muscles. Unfortunately, treatments do not exist for the vast majority of MD patients. Ad...

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Detalhes bibliográficos
Publicado no:Hum Mol Genet
Main Authors: Crudele, Julie M, Chamberlain, Jeffrey S
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2019
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6796995/
https://ncbi.nlm.nih.gov/pubmed/31238336
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddz128
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