This proposal is submitted for renewal of the NIH/NIDDK Research and Translational PKD Research grant entitled Mayo Translational PKD Center (MTPC) that began funding on July 1, 2010. The overall goal of MTPC is to develop Core facilities to support existing and stimulate new PKD research by Mayo and non-Mayo investigators and to facilitate the translation of basic research breakthroughs into improvements in clinical practice through the implementation of clinical trials. The three MTPC Biomedical Research Cores were chosen after thoughtful analysis of the services that would be most effective in achieving the MTPC goals, build on the existing strengths of the PKD investigators at Mayo, and updated in response to our experiences over the past four years of operation. The focus of the Molecular Genetics and Biomarker Core (Dr. Peter Harris, PI) is to characterize PKD populations to facilitate clinical trials, to aid genotype/phenotype studies and gene discovery, and to provide materials and information to catalyze understanding of disease pathogenesis. The focus of the Model Systems Core (Dr. Stephen Ekker, PI) is to empower PKD research through facilitating access to PKD C. elegans, zebrafish and rodent models, to provide expertise for novel organism characterization, and to test novel therapeutics. The focus of the Human Imaging Core (Dr. Bradley Erickson, PI) is to provide accurate and reliable analysis of imaging data and to apply new and enhanced qualitative and quantitative methodologies to measure disease progression and therapeutic outcomes in ADPKD. The focus of the Pilot and Feasibility Program (Dr. Michael Romero, PI) is to attract new investigators into PKD research and provide funds and guidance to foster discovery and the generation of strong grant applications, thus strengthening the research base by developing a new generation of PKD researchers. During its four years of existence, MTPC has refined the activities of the Center in response to surveys and feedback to ascertain membership needs, strengthened its Internal Research Base and added a large External Research Base of PKD investigators. The services and cutting-edge technologies provided by the three heavily used Biomedical Research Cores have been expanded, focusing on areas of high demand and interest. The Pilot and Feasibility Program has so far funded 12 projects with an excellent return for investment, we have implemented a successful Scientific Enrichment Program that promotes synergy among its members, and built research capacity by partnering with Institutional Facilities, other Mayo and non-Mayo NIH funded Centers, and industry. Thus, MTPC has fulfilled the goals set forth by NIDDK for its PKD Research and Translation Core Centers program and is well positioned to build on these successes in the next five years.
Polycystic Kidney Disease (PKD) affects up to 12 million individuals and is the 4th most common cause for renal replacement therapy worldwide. Basic and translational research on PKD has increased exponentially in the last three decades, particularly after the discovery of the PKD1 and PKD2 genes in 1994 and 1996. Nevertheless, no proven effective therapy exists for this disease at the present time. The mission of the Mayo Translational PKD Center (MTPC) is to foster PKD research and the translation of basic research breakthroughs into improvements in clinical practice with the ultimate goal to reduce or eliminate the disease burden of PKD. Therefore, the leadership of the center chose three Biomedical Research Cores after thoughtful analysis of the services that would be most effective in achieving this goal. The Molecular Genetics and Biomarker Core uses genetic approaches to advance the understanding of disease pathogenesis and characterizes PKD populations to facilitate clinical trials. The Model Systems Core empowers PKD research through access to PKD C. elegans, zebrafish and rodent models, their biological characterization, and their use in identifying new therapeutics. The Human Imaging Core provides accurate, reliable analysis of imaging data and develops enhanced methodologies to facilitate the assessment of disease progression and the outcome of therapies in ADPKD. The Pilot and Feasibility Program attracts new investigators to PKD research, helps them to launch novel PKD focused projects, thus sustaining and expanding the Center Membership. The research capacity of the MTPC is enhanced by a vigorous Scientific Enrichment Program that promotes synergy among its members and and by partnering with Institutional Facilities and other Mayo and non-Mayo NIH funded Centers. In the first four years of their existence, the three MTPC Biomedical Research Cores have provided services to many PKD investigators and made important conceptual and technological advances. Research conducted by MTPC investigators has already led to clinical trials with encouraging results.
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