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

Dorothea Maetzel, Sovan Sarkar, Haoyi Wang, Lina Abi-Mosleh, Ping Xu, Albert W. Cheng, Qing Gao, Maisam Mitalipova, Rudolf Jaenisch

Research output: Contribution to journalArticlepeer-review

118 Scopus citations

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Chemical Compounds

Medicine & Life Sciences