Ontology highlight
ABSTRACT:
SUBMITTER: Sung EA
PROVIDER: S-EPMC5689620 | biostudies-literature | 2017 Oct
REPOSITORIES: biostudies-literature
Sung Eun-Ah EA Yu Kyung-Rok KR Shin Ji-Hee JH Seo Yoojin Y Kim Hyung-Sik HS Koog Myung Guen MG Kang Insung I Kim Jae-Jun JJ Lee Byung-Chul BC Shin Tae-Hoon TH Lee Jin Young JY Lee Seunghee S Kang Tae-Wook TW Choi Soon Won SW Kang Kyung-Sun KS
Oncotarget 20170807 49
Niemann-Pick disease type C (NPC) is a neurodegenerative and lysosomal lipid storage disorder, characterized by the abnormal accumulation of unesterified cholesterol and glycolipids, which is caused by mutations in the <i>NPC1</i> genes. Here, we report the generation of human induced neural stem cells from NPC patient-derived fibroblasts (NPC-iNSCs) using only two reprogramming factors <i>SOX2</i> and <i>HMGA2</i> without going through the pluripotent state. NPC-iNSCs were stably expandable and ...[more]