Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

Genetic and Chemical Correction of Cholesterol Accumulation and Impaired Autophagy in Hepatic and Neural Cells Derived from Niemann-Pick Type C Patient-Specific iPS Cells

Niemann-Pick type C (NPC) disease is a fatal inherited lipid storage disorder causing severe neurodegeneration and liver dysfunction with only limited treatment options for patients. Loss of NPC1 function causes defects in cholesterol metabolism and has recently been implicated in deregulation of au...

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Main Authors: Maetzel, Dorothea (Contributor), Sarkar, Sovan (Contributor), Wang, Haoyi (Contributor), Abi-Mosleh, Lina (Author), Xu, Ping (Contributor), Gao, Qing (Contributor), Mitalipova, Maisam (Contributor), Jaenisch, Rudolf (Contributor), Cheng, Albert Wu (Author)
Other Authors: Massachusetts Institute of Technology. Department of Biology (Contributor), Whitehead Institute for Biomedical Research (Contributor), Cheng, Albert W. (Contributor)
Format: Article
Language:English
Published: Elsevier B.V., 2015-04-03T13:48:59Z.
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