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肾功能衰竭的遗传性原因。的基因 在过去的两年中，已经确定了负责最常见形式的PKD（PKD 1，PKD 2和PKHD 1）的基因。 几十年这些年来的进展导致了用于研究囊肿形成和进展的改进的工具， 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。然而，尽管有这些进展，但没有干预措施来阻止囊性肾病， 它的全身表现，仍然迫切需要更有效的治疗。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，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， 向区域培训中心提供高度有组织的行政支助。我们将实施大胆、创新、外向的- 面向计划，将促进RTCC之间的合作和凝聚力，并建立机制 广泛分享试剂和专业知识。国家协调中心将努力振兴和 通过支持和扩大PKD研究人员的社区，通过促进 数据驱动的科学和发展教育活动，鼓励开放和批判性思维， 吸引最聪明的新人才到PKD的研究。国家协调中心的领导将是 由两名研究人员提供，他们对PKD领域有着深入的了解，并对 科学合作的重要性。NCC将支持PKD研究联盟的目标， 以下目标：1）召集PKD研究联盟指导委员会，制定该计划的 全面指导，组织和执行方案活动，并协助广泛传播 2）制定一项多管齐下的宣传计划，包括建立一个便于查阅的网站， 3）通过建立一个框架，促进区域合作中心之间的合作 4）促进在联合体内广泛分享资源5）发展 一个教育计划，包括一个年度科学研讨会，将鼓励建设性的话语 和多样性和创造性的方法来PKD科学。6)开发、支持和执行一项试点和可行性研究 一个鼓励新的研究者（包括早期和成熟的）进入PKD领域的计划 research. 7)提供面向早期调查人员的教育计划，以培养 培养一批新的PKD研究人员和领导者。总之，协作环境 NCC的支持将促进我们预期将导致对PKD病理生物学的新见解的进展 多囊肾的治疗方法有哪些

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.