AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates

GM1 gangliosidosis is a rare, inherited neurodegenerative disorder caused by mutations in the GLB1 gene, which encodes the lysosomal hydrolase acid β-galactosidase (β-gal). β-gal deficiency leads to toxic accumulation of GM1 ganglioside, predominantly in the central nervous system (CNS), resulting i...

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Detalles Bibliográficos
Autores principales: Hocquemiller, Michaël, Giersch, Laura, Mei, Xin, Gross, Amanda L., Randle, Ashley N., Gray-Edwards, Heather L., Hudson, Judith A., Todeasa, Sophia, Stoica, Lorelei, Martin, Douglas R., Sena-Esteves, Miguel, Aiach, Karen, Laufer, Ralph
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9594110/
https://www.ncbi.nlm.nih.gov/pubmed/36320411
http://dx.doi.org/10.1016/j.omtm.2022.10.004

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