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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Detalhes bibliográficos
Main Authors: Maetzel, Dorothea, Sarkar, Sovan, Wang, Haoyi, Abi-Mosleh, Lina, Xu, Ping, Cheng, Albert W., Gao, Qing, Mitalipova, Maisam, Jaenisch, Rudolf
Formato: Artigo
Idioma:Inglês
Publicado em: Elsevier 2014
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4050353/
https://ncbi.nlm.nih.gov/pubmed/24936472
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.stemcr.2014.03.014
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