My laboratory uses a stem cell based approach to understand neurodegenerative diseases, particularly orphan diseases such as Spinal Muscular Atrophy, so that we can identify potentially new therapeutic targets. Our work is based on using disease relevant cells that we can produce in large numbers from stem-like cells. However, because our fundamental interest relates to drug discovery, we have configured a high throughput screening laboratory that combines stem cells, particular sets of drug-like small molecules, and automated confocal imaging. This combination is quite versatile and has allowed us to identify reagents that regulate cell differentiation, reprogramming, transdifferentiation, proliferation and survival. In addition, we have gained a great deal of information concerning pathways that regulate fate and behavior of many different types of cells. Along the way, we have established collaborations with many labs in the Harvard community who share our interest in stem cells, differentiation and disease.