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Neural stem cells for disease modeling and evaluation of therapeutics for Tay-Sachs disease Journal article
ORPHANET JOURNAL OF RARE DISEASES, 2018,Volume: 13
Authors:  Vu, Mylinh;  Li, Rong;  Baskfield, Amanda;  Lu, Billy;  Farkhondeh, Atena;  Gorshkov, Kirill;  Motabar, Omid;  Beers, Jeanette;  Chen, Guokai;  Zou, Jizhong;  Espejo-Mojica, Angela J.;  Rodriguez-Lopez, Alexander;  Almeciga-Diaz, Carlos J.;  Barrera, Luis A.;  Jiang, Xuntian;  Ory, Daniel S.;  Marugan, Juan J.;  Zheng, Wei
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Tay-Sachs disease  Induced pluripotent stem cells  Neural stem cells  Cyclodextrin  HP beta CD  delta-tocopherol  Enzyme replacement therapy  Hexosaminidase A  GM2 gangliosidosis  High throughput screening  Drug discovery