In this proposal we will establish The Johns Hopkins PKD Research and Clinical Core Center (JH-PKD Center), which will provide unique resources and expertise to a national and international Research Base. The Center capitalizes on a long tradition of PKD research at Johns Hopkins and on our unique status as a comprehensive PKD center. The goals of the center are 1) To provide state of the art reagents and expertise to a national and international group of investigators in order to facilitate PKD related research, 2) To provide reagents and expertise to non-PKD investigators in order to remove barriers to PKD related research, 3) To continue to support a vibrant PKD research community that will lead to collaborative interactions between investigators, 4) To establish enrichment activities that encourage open and critical thought and yield creative approaches to research questions, 5) To provide Pilot and Feasibility Funds to junior investigators, to support innovative lines of inquiry and to recruit new expertise to the PKD field and 6) To partner with other PKD Centers and the NIDDK to ensure that resources are used efficiently with a goal of stimulating Translational PKD Research. The JH-PKD Center will contain an Administrative Core (Core A) and 4 biomedical core resources including 1) Antibody Validation and Vectorology (Core B), 2) Mouse models and Biobank (Core C) 3) Cell Engineering (Core D) and 4) the Clinical and Translational Core Resource (Core E). Pilot studies chosen for the first year are tightly focused around Core resources and recruit 3 new investigators at different career stages to the PKD field. Pilot 1 will examine Racial Disparities in Attitudes towards Screening, Treatment, and Participation in Clinical Research for PKD. Pilot 2 will apply cutting edge molecular tools for live cell imaging to examine the role of polycystin 1 and 2 in renal tubuologenesis. Pilot 3 proposes to test a novel glycolipid Inhibitor using the animal models and expertise provided by Core C. Through expanded interdisciplinary science collaborations, we hope to catalyze the translation of biomedical discoveries that slow down or prevent progression of polycystic kidney disease and its consequences. Public Health Relevance: The Polycystic Kidney diseases are a common group of disorders that are a leading cause of end stage renal disease. Despite many advances over the past several years, there is still no cure. We expect that this Core Center will provide the tools and expertise to facilitate collaborative PKD research leading to enhanced understanding of disease pathogenesis and innovative therapeutic approaches.
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