Mouse Models and Cell Systems Core

Mouse Models and Cell Systems Core (MMCSC) Project Summary/Abstract Mouse models and cell-based systems are invaluable tools for advancing research into Polycystic Kidney Disease (PKD). Mouse models closely replicate the disease's progression, enabling scientists to study how mutations in PKD1 and PKD2 genes drive cyst formation. They also provide a rigorous platform for preclinical testing of potential therapies. Complementing these, cell-based systems—such as kidney organoids and tubuloids—offer a powerful, simplified platform for examining signaling pathways, cellular responses, and the molecular dynamics of cyst formation at high resolution. During the current funding period, there has been a significant demand for these models, which highlights their crucial role in advancing PKD research. The Mouse Models and Cell Systems Core (MMCSC) is committed to meeting this demand by offering centralized, easily accessible resources, including genetically engineered mouse strains, isogenic cell lines, and a range of biospecimens. The distribution of standardized resources ensures consistency and reproducibility, essential for reliable data and meaningful comparisons across studies. In addition to supporting existing research tools, the MMCSC aims to drive innovation by developing new models that will allow researchers to explore previously inaccessible aspects of PKD. Key objectives include: 1) Provide the research community with a comprehensive range of genetically engineered in vivo and in vitro PKD bioresources; 2) Expand and maintaining a biorepository of human and murine PKD biospecimens; 3) Establish disease modeling platforms for preclinical testing of therapeutic candidates; 4) Create and distribute novel Pkd1 and Pkd2 mouse models; and 5) Offer personalized tools and support to investigators working with PKD model systems. In summary, completion of these aims will allow the MMCSC to support a wide range of community researchers in conducting high-quality studies with cutting-edge model systems, thereby driving rapid advancements in polycystic kidney disease research.