Screening PKD biological systems for repurposing targeted therapeutics

National Center For Advancing Translational Sciences

Investigators

Abstract

The goal of this project is to screen existing drugs (in particular oncology focused drugs) for their efficacy in PKD using in vitro model systems. The research team has conducted a high-throughput viability screen of mouse PKD1 null cell lines and their respective wild-type controls against oncology-focused small molecule libraries. Compounds that preferentially disrupt the viability of PKD1 null cells versus controls have been selected for follow-up studies, including cell-based and ex vivo cystogenesis models. Selected compounds will also be tested for efficacy in patient-derived kidney cells.