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Safety and Efficacy of Intravenous and Intrathecal Delivery of AAV9-Mediated ARSA in Minipigs

Metachromatic leukodystrophy (MLD) is a hereditary neurodegenerative disease characterized by demyelination and motor and cognitive impairments due to deficiencies of the lysosomal enzyme arylsulfatase A (ARSA) or the saposin B activator protein (SapB). Current treatments are limited; however, gene...

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Detalles Bibliográficos
Autores principales:	Mullagulova, Aysilu, Shaimardanova, Alisa, Solovyeva, Valeriya, Mukhamedshina, Yana, Chulpanova, Daria, Kostennikov, Alexander, Issa, Shaza, Rizvanov, Albert
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	MDPI 2023
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10253118/ https://www.ncbi.nlm.nih.gov/pubmed/37298156 http://dx.doi.org/10.3390/ijms24119204

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