Impaired Neural Differentiation of Induced Pluripotent Stem Cells Generated from a Mouse Model of Sandhoff Disease

@inproceedings{Ogawa2013ImpairedND,
  title={Impaired Neural Differentiation of Induced Pluripotent Stem Cells Generated from a Mouse Model of Sandhoff Disease},
  author={Yasuhiro Ogawa and Makoto Tanaka and Miho Tanabe and Toshihiro Suzuki and Tadayasu Togawa and Tomoko Fukushige and Takuro Kanekura and Hitoshi Sakuraba and Kazuhiko Oishi},
  booktitle={PloS one},
  year={2013}
}
Sandhoff disease (SD) is a glycosphingolipid storage disease that arises from mutations in the Hexb gene and the resultant deficiency in β-hexosaminidase activity. This deficiency results in aberrant lysosomal accumulation of the ganglioside GM2 and related glycolipids, and progressive deterioration of the central nervous system. Dysfunctional glycolipid storage causes severe neurodegeneration through a poorly understood pathogenic mechanism. Induced pluripotent stem cell (iPSC) technology… CONTINUE READING

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