Project Summary
P5 aims at identifying a novel therapeutic approach for polycystic kidney disease by applying the concept of synthetic lethality to the treatment of ADPKD. P5 developed a robust cell-based assay for high-throughput screening of synthetic lethality in ADPKD. Based on preliminary data from a chemical library screen of >50,000 compounds and a proposed genome-wide shRNA screen to identify genetic synthetic lethal interactions with Pkd1, P5 will validate identified promising compounds in cell-based 2D and 3D models of ADPKD, followed by testing in ADPKD mouse models.
