New Therapies Targeting Cystogenesis in Autosomal Polycystic Kidney Disease

Abstract

Autosomal dominant polycystic kidney disease is the most common inherited kidney disease and results from mutations in the polycystin 1 gene (PKD1) or the polycystin 2 gene (PKD2). The disease is characterised by the progressive development of fluid-filled cysts derived from renal tubular epithelial cells that destroy the architecture of the renal parenchyma and lead to kidney failure. Until recently, the causes and the molecular pathways that lead to cystogenesis remained obscure. In the last decade, enormous progress has been made in understanding the pathogenesis of autosomal dominant polycystic kidney disease and developing new therapies. The purpose of this review is to provide an update on the promising therapies that are being developed and tested, based on knowledge of recent advances in molecular and cellular targets involved in cystogenesis.