Treatment of adult metachromatic leukodystrophy model mice using intrathecal administration of type 9 AAV vector encoding arylsulfatase A

Metachromatic leukodystrophy (MLD) is a lysosomal storage disease caused by an arylsulfatase A (ARSA) deficiency and characterized by severe neurological symptoms resulting from demyelination within the central and peripheral nervous systems. We investigated the feasibility and efficacy of intrathec...

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Detalles Bibliográficos
Autores principales: Miyake, Noriko, Miyake, Koichi, Sakai, Atsushi, Yamamoto, Motoko, Suzuki, Hidenori, Shimada, Takashi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2021
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8521568/
https://www.ncbi.nlm.nih.gov/pubmed/34654893
http://dx.doi.org/10.1038/s41598-021-99979-2

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8521568/
https://www.ncbi.nlm.nih.gov/pubmed/34654893
http://dx.doi.org/10.1038/s41598-021-99979-2

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