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

项目概要 多囊肾病 (PKD) 是全球肾衰竭最常见的遗传原因。基因 过去两年中确定了导致最常见 PKD 形式（PKD1、PKD2 和 PKHD1）的原因 几十年。这些年来的进步导致了研究囊肿形成和进展的工具得到改进， 这些反过来又提高了对 PKD 病理生理学的理解，最近， 批准的治疗。然而，尽管取得了这些进展，但还没有任何干预措施可以阻止囊性肾病或 其全身表现，仍然迫切需要更有效的治疗方法。为了 促进跨学科和综合方法来识别新的治疗靶点，RFA 已发布用于支持 PKD 研究和翻译核心中心 (RTCC)，其使命是 开发创新的研究工具。我们在这里建立了一个新的国家协调中心（NCC）来提供 为 RTCC 提供高度组织化的行政支持。我们将实施大胆、创新、外向的 面临的计划将促进 RTCC 之间的合作和凝聚力并建立机制 广泛共享试剂和专业知识。国家协调中心将努力激发和 通过支持和扩大 PKD 研究人员社区、促进 数据驱动科学并开展鼓励开放和批判性思维的教育活动 吸引最聪明的新人才来研究 PKD。国家协调中心的领导 由两位对 PKD 领域有深入了解并对 PKD 领域深信不疑的研究人员提供 协作科学的重要性。 NCC 将支持 PKD 研究联盟的目标 目标如下： 1) 召集 PKD 研究联盟指导委员会来制定该计划的 总体方向，组织和执行计划活动并协助广泛传播 信息 2) 制定多管齐下的沟通计划，包括用于传播信息的无障碍网站 有关研究资源的信息 3) 通过建立框架促进 RTCC 之间的合作 共同开发资源 4) 促进整个联盟的资源广泛共享 5) 开发 教育计划，包括鼓励建设性讨论的年度科学研讨会 以及 PKD 科学的多样化和创造性方法。 6) 开发、支持和执行试点和可行性 鼓励新研究者（早期和成熟）进入 PKD 领域的计划 研究。 7) 提供针对早期研究人员的教育计划，以培养 培养一批新的 PKD 研究人员和领导者。总而言之，协作环境 NCC 培育的项目将促进进展，我们预计这将带来对 PKD 病理学的新见解 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.