Leveraging Novel Technologies for Human iPSC-based Screening

Luping Huang¹, Andrew Hilmer¹, Ying Pei², Xianmin Zeng³

¹Applied StemCell Inc., Milpitas, CA 95035; ²XCell Science Inc, Novato, CA 94945; ³Scientific Advisory Board, Applied StemCell Inc., Milpitas, CA 95035

Human induced pluripotent stem cell (iPSC) technology offers the benefits of a cell line, coupled with the advantages of using human primary cells. We have developed a panel of iPSC lines for neurotoxicity assays and disease modeling applications. These cell lines include: 1) control lines, 2) patient-specific lines, 3) lineage-specific knock-in reporters, 4) isogenic controls of single and double knock-outs. We have also established scalable protocols for generating differentiated cells in an assay-ready format. Here, we describe the utility of these 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.