Novel Drug Candidates Improve Ganglioside Accumulation and Neural Dysfunction in GM1 Gangliosidosis Models with Autophagy Activation

GM1 gangliosidosis is a lysosomal storage disease caused by loss of lysosomal β-galactosidase activity and characterized by progressive neurodegeneration due to massive accumulation of GM1 ganglioside in the brain. Here, we generated induced pluripotent stem cells (iPSCs) derived from patients with...

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Detalles Bibliográficos
Autores principales: Kajihara, Ryutaro, Numakawa, Tadahiro, Odaka, Haruki, Yaginuma, Yuji, Fusaki, Noemi, Okumiya, Toshika, Furuya, Hirokazu, Inui, Seiji, Era, Takumi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2020
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7220856/
https://www.ncbi.nlm.nih.gov/pubmed/32302553
http://dx.doi.org/10.1016/j.stemcr.2020.03.012

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