Novel Therapeutic Strategies Targeting Molecular Pathways of Cystogenesis in Autosomal Polycystic Kidney Disease

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most common inherited kidney disease that results from mutations in PKD1 or PKD2. The disease is characterized 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 ADPKD and the development of new therapies. The purpose of this review is to 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.

An Overview of In Vivo and In Vitro Models for Autosomal Dominant Polycystic Kidney Disease: A Journey from 3D-Cysts to Mini-Pigs

“…PC-1: polycystin-1; PC-2: polycystin-2; CTT: C-terminal tail (of polycystin-1); ECM: extracellular matrix; ER: endoplasmic reticulum; M: mitochondrion; PC: primary cilium; PM: plasma membrane; TNG: trans-Golgi network. [ 19 , 21 , 22 , 23 , 24 , 25 , 26 ].…”

New Therapies Targeting Cystogenesis in Autosomal Polycystic Kidney Disease

“…In addition, other agents targeting different molecules or pathways involved in cystogenesis have been used in preclinical studies and some of them are ongoing in early clinical trials in humans. 66 All these agents are summarised in Table 1.…”