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

项目摘要 多囊肾病(PKD)是世界范围内最常见的遗传性肾功能衰竭原因。基因 在过去的两年中发现了最常见的PKD形式(PKD1、PKD2和PKHD1) 几十年。这些年的进步导致了研究囊肿形成和发展的工具的改进，以及 这些反过来又导致了对PKD病理生理学的更好理解，最近， 批准的治疗方法。然而，尽管取得了这些进展，仍没有干预措施来阻止囊性肾脏疾病或 它的全身性表现，仍然迫切需要更有效的治疗。为了 促进确定新的治疗靶点的跨学科和综合方法 已经发布，以支持PKD研究和翻译核心中心(RTCC)，其使命将是 开发创新的研究工具。在这里，我们建立了一个新的国家协调中心(NCC)，以提供 向区域协调中心提供组织严密的行政支助。我们将实施大胆、创新、外向的-- 面向将促进区域协调中心之间的协作和凝聚力并建立机制的方案 广泛分享试剂和专业知识。国家协调中心将寻求振兴和 通过支持和扩大PKD研究人员社区，通过促进 数据驱动的科学，并通过发展鼓励开放和批判性思维的教育活动 吸引最优秀的新人才加入到PKD的研究中。国家协调中心的领导将是 由两名调查人员提供，他们对PKD领域有深入的了解，并对 协作科学的重要性。NCC将支持PKD研究联盟的目标， 以下目标：1)召开PKD研究联盟指导委员会，以制定该计划的 总体指导，组织和执行方案活动，并协助广泛传播 信息2)制定多管齐下的传播计划，包括一个可访问的网站，用于传播 关于研究资源的信息3)通过建立框架促进区域合作伙伴之间的合作 为了联合开发资源4)促进财团之间的资源广泛共享5)开发 教育计划，包括一年一度的科学研讨会，将鼓励建设性的讨论 以及对PKD科学的多样化和创造性的方法。6)开发、支持和执行试点和可行性 鼓励新的研究人员(包括早期和成熟的)进入PKD领域的计划 研究。7)提供针对早期调查人员的教育方案，以培养 发展一批新的PKD研究人员和领导者。总而言之，协作环境 由NCC培育的将催化进展，我们预计将导致对PKD病理生物学的新见解 以及多囊肾病患者的新治疗选择。

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.