Utilizing hiPSC-Derived Neurons and Cardiomyocytes For Drug Screening

Patient specific induced pluripotent stem cells (iPSC) complement traditional cell-based assays used in drug discovery and could aid in the development of clinically useful compounds. They allow interrogation of differentiated features of human cells not reflected by immortalized lines, and importantly they carry disease-specific traits in complex genetic backgrounds that can impact disease phenotypes. Development of technology platforms to perform compound screens of iPSC with relatively high-throughput will be essential to realize their potential for disease modeling and drug discovery. Towards this goal, we have been working to develop a standardized battery of assays against which iPSC-derived neurons can be screened for specific phenotypes. We will discuss our screening results and development of patient cell specific and hiPSC based models for testing of drugs on disease relevant cell types.