A Single Injection of an Optimized Adeno-Associated Viral Vector into Cerebrospinal Fluid Corrects Neurological Disease in a Murine Model of GM1 Gangliosidosis

GM1 gangliosidosis is a rare neurodegenerative lysosomal storage disease caused by loss-of-function mutations in the gene encoding beta-galactosidase (β-gal). There are no approved treatments for GM1 gangliosidosis. Previous studies in animal models have demonstrated that adeno-associated viral (AAV...

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Detalles Bibliográficos
Autores principales: Hinderer, Christian, Nosratbakhsh, Brenden, Katz, Nathan, Wilson, James M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Mary Ann Liebert, Inc., publishers 2020
Materias:
Research Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7698982/
https://www.ncbi.nlm.nih.gov/pubmed/33045869
http://dx.doi.org/10.1089/hum.2018.206

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7698982/
https://www.ncbi.nlm.nih.gov/pubmed/33045869
http://dx.doi.org/10.1089/hum.2018.206

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