Efficacy of Adeno-Associated Virus Serotype 9-Mediated Gene Therapy for AB-Variant GM2 Gangliosidosis

GM2 gangliosidoses are a group of neurodegenerative lysosomal storage disorders that are characterized by the accumulation of GM2 gangliosides (GM2), leading to rapid neurological decline and death. The hydrolysis of GM2 requires the specific synthesis, processing, and combination of products of thr...

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Detalles Bibliográficos
Autores principales: Vyas, Meera, Deschenes, Natalie M., Osmon, Karlaina J. L., Chen, Zhilin, Ahmad, Imtiaz, Kot, Shalini, Thompson, Patrick, Richmond, Chris, Gray, Steven J., Walia, Jagdeep S.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10572999/
https://www.ncbi.nlm.nih.gov/pubmed/37834060
http://dx.doi.org/10.3390/ijms241914611

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10572999/
https://www.ncbi.nlm.nih.gov/pubmed/37834060
http://dx.doi.org/10.3390/ijms241914611

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