Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

Cerebral organoids derived from Sandhoff disease-induced pluripotent stem cells exhibit impaired neurodifferentiation[S]

Sandhoff disease, one of the GM2 gangliosidoses, is a lysosomal storage disorder characterized by the absence of β-hexosaminidase A and B activity and the concomitant lysosomal accumulation of its substrate, GM2 ganglioside. It features catastrophic neurodegeneration and death in early childhood. Ho...

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Bibliographic Details
Main Authors: Maria L. Allende, Emily K. Cook, Bridget C. Larman, Adrienne Nugent, Jacqueline M. Brady, Diane Golebiowski, Miguel Sena-Esteves, Cynthia J. Tifft, Richard L. Proia
Format: Article
Language:English
Published: Elsevier 2018-03-01
Series:Journal of Lipid Research
Subjects:
storage diseases
gangliosides
Tay-Sachs disease
sphingolipids
brain lipids
Clustered Regularly Interspaced Short Palindromic Repeats/Cas9
Online Access:http://www.sciencedirect.com/science/article/pii/S0022227520331424

Internet

http://www.sciencedirect.com/science/article/pii/S0022227520331424

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