Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease

Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease

Abstract Background Tay-Sachs disease (TSD) is a rare neurodegenerative disorder caused by autosomal recessive mutations in the HEXA gene on chromosome 15 that encodes β-hexosaminidase. Deficiency in HEXA results in accumulation of GM2 ganglioside, a glycosphingolipid, in lysosomes. Currently, there...

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Main Authors: Mylinh Vu, Rong Li, Amanda Baskfield, Billy Lu, Atena Farkhondeh, Kirill Gorshkov, Omid Motabar, Jeanette Beers, Guokai Chen, Jizhong Zou, Angela J. Espejo-Mojica, Alexander Rodríguez-López, Carlos J. Alméciga-Díaz, Luis A. Barrera, Xuntian Jiang, Daniel S. Ory, Juan J. Marugan, Wei Zheng
Format: Article
Language:English
Published: BMC 2018-09-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Tay-Sachs disease
Induced pluripotent stem cells
Neural stem cells
Cyclodextrin
HPβCD
δ-tocopherol
Online Access:http://link.springer.com/article/10.1186/s13023-018-0886-3

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