Over the last decade mouse models have become a mainstay of translational polycystic kidney disease (PKD) research and vital to investigators around the world. Mouse models have been used to establish the molecular and cellular basis of PKD, to identify therapeutic targets and to validate treatment targets in vivo. The overarching goal of the Mouse Models and Biobank Core (MMBC) is to dismantle barriers for investigators seeking to use PKD mouse models in their research. The MMBC will facilitate PKD research by distributing and maintaining PKD mouse models and reagents, by sharing expertise and by performing and/or providing assistance with preclinical trials in mice using standardized protocols. In addition, the MMBC will develop innovative mouse models that can be used to understand disease pathogenesis and to test new classes of therapeutic agents. To achieve these goals we propose the following aims: 1) Provide the research community with a genetically engineered live mouse bio-resource 2) Generate and maintain a repository of murine biospecimens 3) Assist in preclinical testing of promising candidate therapies using appropriate PKD models 4) Provide personalized consultation and tools for investigators who use PKD mouse models in their research and 5) Generate and distribute a novel Pkd2 mouse model that can be used for pre-clinical testing of agents that target defective channel processing and/or function. In summary, completion of these aims will allow the Mouse Models and Biobank Core to assist a diverse community of investigators in conducting high quality research using state-of- the-art mouse models thereby accelerating discovery in the field of polycystic kidney disease.
