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

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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Bibliographic Details
Main Authors: Toshiki Tsunogai, Toya Ohashi, Yohta Shimada, Takashi Higuchi, Ayaka Kimura, Ayako M. Watabe, Fusao Kato, Hiroyuki Ida, Hiroshi Kobayashi
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
GM1-gangliosidosis
ex vivo gene therapy
lentiviral vector
lysosomal storage disorder
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000602

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http://www.sciencedirect.com/science/article/pii/S2329050122000602

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