Large-scale Genetic Investigation and Drug Discovery to Counteract Down Syndrome Neural Deficits

This proposal directly addresses the need for new pharmacological treatments to prevent intellectual disability in Down syndrome. Defining how human chromosome 21 genes interact synergistically to drive abnormal brain development and function will reveal critical underlying pathways and new protein targets. Using the high- throughput larval zebrafish will support systematic characterization of many genetic contributions and discovery of lead molecules through whole-organism compound screening.