Autosomal Dominant Polycystic Kidney Disease (ADPKD) is one of the most common genetic diseases with an incidence of 1 in 1000 in the general population. The hallmark of this slowly progressive disease is bilateral renal cysts formation. The cysts increase in size and number throughout an individual's lifetime and approximately half of all patients progress to renal failure, requiring dialysis or transplant. There are no therapies to date to cure or even to slow the progress of the disease. Mutations in two genes have been to date associated with the disease: PKD1 and PKD2. The former is responsible for 85% of all cases, while the latter for the remaining 15%. PKD1 is predicted to encode a large transmembrane receptor (Polycystin-1) involved in cell/cell or cell/matrix interactions, while the PKD2 gene product (Polycystin-2) is a non selective cation channel. The two proteins interact through their intracellular C-termini and are believed to function in a common signaling pathway, thus explaining the almost identical phenotype observed in patients with mutation in PKD1 or PKD2.