Poster PDF: Presented at the 2017 Discovery on Target Meeting

DISEASE MODELING AND DRUG SCREENING IN NEUROLOGICAL DISORDERS VIA NOVEL HUMAN IPSC-BASED TECHNOLOGIES

Human primary cells derived induced pluripotent stem cells (iPSCs) and its differentiated neuronal lineage cells serve as an ideal in vitro disease model for neurological disorders drug screening and other applications. We have established a panel of iPSC lines for neurotoxicity assays and disease modeling applications. These cell lines include: 1) control lines, 2) patient-specific disease lines, 3) lineage-specific knock-in reporter lines, and 4) isogenic lines of single and double knock-outs. We have also developed scalable protocols for the generation of different lineages of differentiated neuronal cells in an assay-ready format. In this study, we demonstrate the applications of the above-mentioned lines for neurotoxicity assays, including assays to determine the specificity of different neural cell types for a small range of chemicals and drugs from the Tox21 library, as well as for neuroprotective assays with dopaminergic neurons.