Our goal is to develop new and effective treatments for diseases by uncovering the fundamental biological principles underlying development and disease. To accomplish our goals, we develop and deploy a broad range of in vitro and in vivo tools that reveal the fundamental mechanisms involved in human disease. These approaches include genetic editing in vitro and in vivo, human induced pluripotent cell (iPS cell) models of disease, haploid cells for genetic as well as compound screening paradigms, mouse and human organoid cultures, as well as genetically engineered mice. Our multidisciplinary techniques enable us to model and study the complexity of diseases.