Molecular and Tissue Engineering for Understanding Human Brain in Health and Disease

This conference seeks to bring together a multidisciplinary group of geneticists, neuroscientists, and stem cell biologists endeavoring to improve diagnostics, prognostics, and treatment of brain disease. The conference will explore (1) genomics of brain diseases, (2) applications of high-throughput genomic assays and screens, (3) technological advances in reprogramming, differentiation, and genome-engineering; (4) new insights into human brain development and disorders obtained through technologies; and (5) progress towards novel therapeutic targets and interventions. Whereas large-scale genomic studies are uncovering the complex genetic architecture of gene regulation and disease risk, the causal cellular mechanisms of disease risk and progression remain unclear. While the generation of patient-specific neurons, glia, and organoids facilitate the study of human brain development, CRISPR-based engineering approaches facilitate single or genome-wide perturbations in DNA sequence or epigenetic activity; their integration makes it possible to investigate the molecular mechanisms of disease-associated variations. Together, these approaches translate the myriad advances from large-scale genetic studies into clinically actionable information.

This GRC will contribute to improving our understanding the cellular and molecular mechanisms involved in regulating human brain development in health and disease.