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...

Полное описание

Библиографические подробности
Главные авторы: Maetzel, Dorothea, Sarkar, Sovan, Wang, Haoyi, Abi-Mosleh, Lina, Xu, Ping, Gao, Qing, Mitalipova, Maisam, Jaenisch, Rudolf, Cheng, Albert Wu
Другие авторы: Massachusetts Institute of Technology. Department of Biology
Формат: Статья
Язык:en_US
Опубликовано: Elsevier B.V. 2015
Online-ссылка:http://hdl.handle.net/1721.1/96366
https://orcid.org/0000-0001-9724-6776

Internet

http://hdl.handle.net/1721.1/96366
https://orcid.org/0000-0001-9724-6776

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