Pre-clinical Gene Therapy with AAV9/AGA in Aspartylglucosaminuria Mice Provides Evidence for Clinical Translation

Aspartylglucosaminuria (AGU) is an autosomal recessive lysosomal storage disease caused by loss of the enzyme aspartylglucosaminidase (AGA), resulting in AGA substrate accumulation. AGU patients have a slow but progressive neurodegenerative disease course, for which there is no approved disease-modi...

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Detalles Bibliográficos
Autores principales: Chen, Xin, Snanoudj-Verber, Sarah, Pollard, Laura, Hu, Yuhui, Cathey, Sara S., Tikkanen, Ritva, Gray, Steven J.
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/PMC7934581/
https://www.ncbi.nlm.nih.gov/pubmed/33186692
http://dx.doi.org/10.1016/j.ymthe.2020.11.012

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7934581/
https://www.ncbi.nlm.nih.gov/pubmed/33186692
http://dx.doi.org/10.1016/j.ymthe.2020.11.012

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