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

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 ...

Source: Orphanet Journal of Rare Diseases - September 17, 2018 Category: Internal Medicine 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 Rodr íguez-López, Carlos J. Alméciga-Díaz, Luis A. Barrera, Xuntian Jiang, Da Tags: Research Source Type: research