Autosomal dominant polycystic kidney disease (ADPKD) is the most common, lethal, human genetic disease inherited as a dominant trait as a result of mutation in a single gene. With an estimated prevalence of 1:750, it affects 500,000 individuals in the United States. In recent years, substantial progress has been made towards understanding the molecular genetics and biology of this disease that now makes it possible to design translational studies to improve prognosis and therapies for this group of patients. We are interested in pre-clinical testing of glycogen synthase kinase-3 beta inhibitors in polycystic kidney disease models to slow cyst progression. Clinical studies include polycystic kidney disease patient database for longitudinal studies of impact of various clinical variables on disease progression; biomarker analysis to monitor disease progression and response to therapies; as well as clinical trials of therapies as they become available.