Human GLB1 knockout cerebral organoids: A model system for testing AAV9-mediated GLB1 gene therapy for reducing GM1 ganglioside storage in GM1 gangliosidosis

GM1 gangliosidosis is an autosomal recessive neurodegenerative disorder caused by the deficiency of lysosomal β-galactosidase (β-gal) and resulting in accumulation of GM1 ganglioside. The disease spectrum ranges from infantile to late onset and is uniformly fatal, with no effective therapy currently...

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Detalhes bibliográficos
Publicado no:Mol Genet Metab Rep
Main Authors: Latour, Yvonne L., Yoon, Robin, Thomas, Sarah E., Grant, Christina, Li, Cuiling, Sena-Esteves, Miguel, Allende, Maria L., Proia, Richard L., Tifft, Cynthia J.
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2019
Assuntos:
Research Paper
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6744524/
https://ncbi.nlm.nih.gov/pubmed/31534909
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymgmr.2019.100513
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