National Coordinating Center (NCC) for the Polycystic Kidney Disease (PKD) Research and Translation Core Centers

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Project Summary Polycystic Kidney Disease (PKD) is the most common inherited cause of renal failure worldwide. The genes responsible for the most frequent forms of PKD (PKD1, PKD2 and PKHD1) were identified over the past two decades. Progress over these years has led to improved tools for studying cyst formation and progression and these in turn have resulted in an improved understanding of PKD pathophysiology and, most recently, an approved therapy. Despite these advances, however, there is no intervention to halt cystic kidney disease or its systemic manifestations and there remains a pressing need for more effective treatments. In order to promote interdisciplinary and integrated approaches to the identification of novel therapeutic targets, an RFA has been issued to support PKD Research and Translation Core Centers (RTCCs) whose mission will be to develop innovative research tools. Here we establish a new National Coordinating Center (NCC) to provide highly organized administrative support to the RTCCs. We will implement a bold, innovative and outward- facing program that will facilitate collaboration and cohesion between the RTCCs and establish mechanisms for sharing of reagents and expertise broadly. The National Coordinating Center will seek to invigorate and energize research in PKD by supporting and expanding the community of PKD investigators, by promoting data driven science and by developing educational activities that encourage open and critical thinking and attract the brightest new talent to the study of PKD. Leadership of the National Coordinating Center will be provided by two investigators with an in-depth understanding of the PKD field and a deep belief in the importance of collaborative science. The NCC will support the goals of the PKD Research Consortium with the following Aims: 1) Convene the PKD Research Consortium Steering Committee to develop the program’s overall direction, to organize and execute program activities and to assist with broad dissemination of information 2) Develop a multipronged communication plan including an accessible web site for disseminating information about research resources 3) Facilitate collaboration between RTCCs by establishing a framework for joint development of resources 4) Facilitate broad sharing of resources across the consortium 5) Develop an educational program, including an annual scientific symposium that will encourage constructive discourse and diverse and creative approaches to PKD science. 6) Develop, support and execute a pilot and feasibility program that encourages new investigators (both early stage and established) to enter the field of PKD research. 7) Provide educational programming geared toward early stage investigators to foster the development of a new cohort of PKD researchers and leaders. In summary, the collaborative environment fostered by the NCC will catalyze advances that we expect will result in new insights into PKD pathobiology and new therapeutic options for patients with polycystic kidney disease.