Human iPSC-Based Disease Modeling & Drug Screening For Neurodegenerative Disorders

Human induced pluripotent stem cell (iPSC) technology combines the advantages of cell lines with the use of human primary cells, offering versatile tools for research and drug development. These iPSCs can be genetically edited and differentiated into various cell types, which enables the creation of highly relevant disease models for basic research, toxicity screening, drug discovery, and potential therapeutic applications.

Here, we discuss a panel of engineered iPSC lines designed specifically for neurotoxicity assays and disease modeling. The panel includes control lines, patient-specific lines, lineage-specific knock-in reporters, and isogenic controls with single and double gene knockouts. Scalable protocols have been developed to generate differentiated cells in an assay-ready format, streamlining their use in high-throughput screens. The discussion further emphasizes how these iPSC-derived models can assess the neurotoxicity of various chemicals and drugs, using a subset of compounds from the Tox21 library.

Watch the presentation below to review their application in neuroprotective assays, particularly using dopaminergic neurons, demonstrating their utility for studying neural specificity and protective mechanisms relevant to neurodegenerative diseases.