Hematopoietic stem cell gene therapy ameliorates CNS involvement in murine model of GM1-gangliosidosis

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GM1-gangliosidosis is a progressive neurodegenerative glycosphingolipidosis resulting from a GLB1 gene mutation causing a deficiency of the lysosomal enzyme β-galactosidase, which leads to the abnormal accumulation of GM1 ganglioside in the central nervous system. In the most severe early infantile...

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Detalles Bibliográficos
Autores principales: Tsunogai, Toshiki, Ohashi, Toya, Shimada, Yohta, Higuchi, Takashi, Kimura, Ayaka, Watabe, Ayako M., Kato, Fusao, Ida, Hiroyuki, Kobayashi, Hiroshi
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/PMC9118356/
https://www.ncbi.nlm.nih.gov/pubmed/35615711
http://dx.doi.org/10.1016/j.omtm.2022.04.012

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