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...

Descripción completa

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

Internet

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

Ejemplares similares