S4: Drug Toxicity Screening using human stem cells

Drug Toxicity Screening using human stem cells

S4: Drug Toxicity Screening using human stem cells, OpenTox Asia 2017
PRESENTING AUTHOR: 

Kyun-Sun Kang 

INSTITUTION / COMPANY : 

Adult Stem Cell Research Center, College of Veterinary Medicine, Seoul National University

POSITION: 

Director/Professor

ABSTRACT CONTENT / DETAILS: 

Human stem cells including induced pluripotent stem cells and induced adult stem cells are thought to be useful for the prediction of drug-induced toxicity and for the alternative methods substitute of animal testing. Recently, human induced neural stem cells (iNSC) from skin fibroblasts of a Niemann-Pick disease type C (NPC) patient that suffers from a neurodegenerative and lysosomal lipid storage disorders were generated. Established NPC-iNSCs were stably expandable and differentiated into neurons, astrocytes, and oligodendrocytes. However, importantly, NPC-iNSCs retained the main features of NPC, displaying defects in self-renewal and neuronal differentiation accompanied by abnormal cholesterol accumulation. For the use as a drug screening platform, these NPC-iNSCs were analyzed to treat valproic acid, a well-known therapeutic molecule, and shown that impaired phenotypes were recapitulated comparable to WT-iNSCs. Moreover, graphene could restore the cholesterol accumulation and neuronal differentiation in NPC-iNSCs. Subsequently, graphene provided its therapeutic role in a NPC1-kockout mouse model. Taken together, these findings suggest that NPC-iNSCs can serve as an unlimited source of neural cells for pathological study and also drug screening in a patient specific manner.