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Micro-laminin gene therapy can function as an inhibitor of muscle disease in the dy(W) mouse model of MDC1A

Gene replacement for laminin-α2-deficient congenital muscular dystrophy 1A (MDC1A) is currently not possible using a single adeno-associated virus (AAV) vector due to the large size of the LAMA2 gene. LAMA2 encodes laminin-α2, a subunit of the trimeric laminin-211 extracellular matrix (ECM) protein...

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Detalles Bibliográficos
Autores principales:	Packer, Davin, Martin, Paul T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2021
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8026908/ https://www.ncbi.nlm.nih.gov/pubmed/33869655 http://dx.doi.org/10.1016/j.omtm.2021.02.004

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