Polycystin Function Resource Development Core

PROJECT SUMMARY The Polycystin Function Resource Development Core (RDC) will establish an “incubator” with the expertise, resources, and collaborative environment for development of cutting-edge resources to accelerate research on the polycystin proteins. The RDC Team will be led by 3 established scientists with extensive experience in the polycystins, cilia function, and PKD progression, and technological expertise in genetic engineering and cell and mouse lines. The RDC will operate under the guidance of the Steering Committee of the PKD RRC and will solicit input from the research community on the needs for reagents and model systems that are critical for advancing the polycystin-PKD field. We will prioritize these requests and plan and budget projects through consultation and approval with the Kansas PKD RTCC and the PKD RRC Steering Committee. The initial aims of the project are (1) Develop a RDC with the scientific and technical expertise to generate research resources for the investigation of the polycystin proteins, (2) Develop a suite of in vivo reagents to precisely control expression of polycystin-1 (PC1), and (3) Develop tools for investigating PC1 domains and interacting proteins. These innovative reagents will optimize genetic control of Pkd1 gene expression, allow for rapid and reversible depletion of PC1 protein, detect PC1 localization, and identify components of the polycystin complex in vivo. Novel molecular reagents will allow screening of the effects of various domain mutations on PC1 functional read- outs and for testing of intragenic complementation of Pkd1 mutations.