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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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Pubblicato in:Hum Mol Genet
Autori principali: Crudele, Julie M, Chamberlain, Jeffrey S
Natura: Artigo
Lingua:Inglês
Pubblicazione: Oxford University Press 2019
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Accesso online: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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