The human brain is the most complex organ in the known universe; it contains approximately 100 billion neurons of many different subtypes. These neurons form an integrated network of trillions of connections that allows the brain to process, compute, and store information at unprecedented levels. The research of my group is focused on understanding how the brain develops at the cellular and molecular level. We are particularity interested in how molecular and cellular processes go awry in neuropsychiatric and neurodegenerative disease. We would like to understand the details of how neurons function at the molecular, cellular, and network level, in order to rationally design drugs.
We are taking a technology-driven approach that combines: 1) the ability to generate human pluripotent stem cells for normal and patient populations, 2) the ability to engineer these cells at nucleotide precision to correct disease mutations and/or generate reporter assays, 3) the ability to direct these cells to become different types of disease-relevant neurons, and 4) the ability to perform high-throughput (HTP) genomic and physiological measurements on these cells to identify cellular phenotypes that correlate with disease state. We are employing a modular pipeline approach where we standardize, industrialize, and automate key aspects of the technology to build a best-in-class platform to model cellular aspects of human brain development and disease in a dish. This pipeline will be used to improve our understanding of the human brain biology, for HTP drug screening/drug discovery, and to increase our odds for drug success in the clinic.