Filling the holes in cystic kidney disease research

Abstract

Kidney disease is a significant medical and public health problem. The National Institute of Diabetes and Digestive and Kidney Diseases recently asked the community to identify research objectives, which, if addressed, could improve understanding of basic kidney function and aid in prevention, treatment, and reversal of kidney disease. The Kidney Research National Dialogue invited interested parties to submit, discuss, and prioritize ideas using an interactive website; 1600 participants posted more than 300 ideas covering all areas of kidney disease, including the cystic kidney diseases. Although much is known about the genetics and pathogenesis of cystic diseases, there remain challenges to our understanding of the fundamental mechanisms of cyst formation, what genes act as modifiers to cause variable responses in different people, and how to detect and monitor disease progression. This article summarizes key research questions for cystic kidney diseases.

Keywords: cilia; cysts; kidney disease; polycystic kidney disease.

Figure 1.

There is a continued need for basic research into cyst formation and growth, the role of cilia, and the genes that modify the course of disease. Earlier biomarkers are needed to predict prognosis and monitor disease progression. They, in turn, will help select patients for clinical trials and optimize therapeutic end points. PKD, polycystic kidney disease.