In this application we seek to renew funding for The Baltimore PKD Research and Clinical Core Center (B-PKD Center). The overarching objective of this Center of Excellence is to provide state of the art resources to a national and international Research Base with the goal of advancing innovative translational science in the field of Polycystic Kidney Disease (PKD). The Center capitalizes on a long tradition of cutting edge research by B- PKD Center Investigators who have been at the forefront of PKD science for the past two decades. The objectives of the Center will be implemented in 6 specific Aims: 1) To provide critical state of the art reagents and expertise to a national and international group o PKD investigators 2) To provide critical state of the art 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 members of the research base and particularly between Basic and Clinical PKD investigators 4) To establish enrichment activities that encourage open and critical thought and yield creative approaches to research questions 5) To provide Pilot & Feasibility Funds to junior investigators, to support innovative lines of inquiry and to recruit new expertise to the PKD field. The B-PKD Center Research Base consists of 25 investigators in Baltimore at The University of Maryland School of Medicine and at the Johns Hopkins School of Medicine. In addition, the Center serves a robust extended National and International Research Base of 48 investigators. In order to support these investigators the Center will provide a Tool Kit for PKD Research developed by 4 biomedical core resources: 1. Antibody Validation and Vectorology (Core B), 2. Mouse Model and Biobank (Core C), 3. Cell Engineering (Core D), and 4) the Clinical Translational Core (Core E). In addition the Administrative Core (Core A) will continue to organize innovative Enrichment Activities that will encourage open, interactive and creative solutions to research questions. The well-crafted Pilot Proposals chosen for this year are focused around Core resources and recruit 2 outstanding investigators, at early scientific career stages, to the PKD field. Both proposals will use innovative methods to address mechanistic questions relevant to polycystic kidney disease. Through expanded interdisciplinary collaborations, we hope to catalyze the translation of biomedical discoveries that prevent progression of polycystic kidney disease and its consequences.
Autosomal dominant polycystic kidney disease (ADPKD) is the most common genetic form of kidney disease. The disorder is characterized by progressive renal cyst formation, which ultimately causes end stage renal disease (ESRD) in a significant proportion of those affected. There is no cure for Polycystic Kidney Disease (PKD) and complications are treated as they arise. During the past 5 years several large clinical trials testing therapies for polycystic kidney disease have been reported. Despite a large investment of resources, these therapies have not reversed nor cured the disease. There remains a need for an improved understanding of the PKD signaling nexus in order to identify new therapeutic targets. The next generation of prospective therapeutics will have the highest chance of clinical success if they are informed by basic discovery research. This will require synergy between all corners of the 'PKD Universe', Basic and Clinical investigators alike. The overarching objective of this Center of Excellence is to provide state of the art resources and expertise to a national and international Research Base of investigators with the goal of advancing innovative translational science in the field of Polycystic Kidney Disease. Through expanded interdisciplinary collaborations, we hope to catalyze the translation of biomedical discoveries that will prevent progression of polycystic kidney disease and its consequences.
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