Our overall objective is to understand mechanisms of pathogenesis, and to discover therapeutic targets and innovative treatments for human genetic diseases. We make use of human specimens and animal models to understand the molecular basis of a disease, perform drug screening and evaluate the activity of candidate drugs. Our drug screening platforms offer a rapid, effective, and economical system to validate chemical compounds for their utility as drugs in the studied pathological conditions. Our long term aspiration is the application of our research findings and the transformation of biomedical knowledge into products or services for the society. For that purpose, we are pursuing collaboration with spin-offs and start-ups.