High-throughput screening to identify modulators of PKD2 function

National Center For Advancing Translational Sciences

Investigators

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is caused by mutations in transmembrane proteins PKD1 (PC1) or PKD2 (PC2). PC1 and PC2 subunits form a cation channel at the plasma membrane, and function can be disrupted by disease-causing mutations During this period, the team worked to design and implement a high-throughput screening amenable assay capable of identifying small molecule activators of the PC1/PC2 heteromeric complex. A novel cell-based assay of PC1/PC2 activity was developed by the Delling lab.