Article
Generation and characterization of induced pluripotent stem cell (iPSC) lines from patients affected with Tay-Sachs and Sandhoff disease.
Stem cell research - 1 Apr 2026
Jovanovic Vukasin M, Chen Catherine Z, Toro Camilo, Hurd Kerstin A, Zou Jizhong, Tifft Cynthia J, Zheng Wei, Henderson Mark J, Marugan Juan, Tristan Carlos A
Abstract excerpt
Tay-Sachs and Sandhoff diseases, are sphingolipidoses caused by rare genetic mutations in the HEXA and HEXB genes, that encode the alpha and beta subunits of lysosomal hexosaminidase, respectively. Here, we report the generation and characterization of three Tay-Sachs and one Sandhoff iPSC lines derived from patients with late-onset disease carrying mutations at the HEXA or HEXB gene. The Tay-Sachs patients...
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