nephropathy, with an esti-mated prevalence of 1:1000. The disease is characterized by the development of multiple cysts from all nephron segments leading to the enlargement of both kidneys and replacement of normal parenchyma (see [1]). Change in total kidney volume over time is the strongest predictor of renal function decline in ADPKD [2]. Glomerular filtra-tion rate remains preserved up to the age of 40 years in most patients because glomerular hyperfiltration in functioning nephrons compensates for the ongoing loss of renal tissue, until end-stage renal failure ensues in>50 % of patients, usually in their fifth decade. Mutations in the PKD1 gene account for ~85 % of the affected families, whereas the remaining cases are caused by mutations in PKD2. PKD1 encodes polycystin-1, an integral membrane protein with a large extracellular domain that probably functions as a re-ceptor and/or an adhesion molecule, whereas PKD2 enco-des polycystin-2, a non-selective cation channel belonging to the family of transient receptor potential channels. The polycystins are located in the primary cilium and interact to form a mechanosensory complex that is involved in intra-cellular Ca21 homeostasis and various signalling pathways. Disruption of the complex leads to cyst development and enlargement resulting from tubular cell proliferation and transepithelial fluid secretion. The progressive understand-ing of these pathways has led to spectacular advances in the prospective treatment for ADPKD, including the blockade of vasopressin 2 receptor (V2R) to decrease the intracellu-lar level of 3#-5#-cyclic adenosine monophosphate (cAMP) in cyst-lining tubular cells [1]
