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

国家多囊肾病 (PKD) 研究和转化核心中心协调中心 (NCC)

• 批准号: 10218161
• 负责人: Terry J Watnick
• 金额: $ 91.15万
• 依托单位: UNIVERSITY OF MARYLAND BALTIMORE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-20 至 2025-06-30
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10218161
• 关键词: Address; Autosomal Dominant Polycystic Kidney; Belief; Biological Models; Collaborations; Communication; Communities; Critical Thinking; Cyst; Cystic Kidney Diseases; Data; Development; Disease; Educational Activities; Evaluation; Event; Fostering; Functional disorder; Funding; Funding Opportunities; Genes; Genomics; Goals; Human; Information Dissemination; Inherited; Intervention; Joints; Kidney Failure; Leadership; Mission; Patients; Polycystic Kidney Diseases; Positioning Attribute; Reagent; Research; Research Personnel; Resource Development; Resource Sharing; Resources; Science; Structure; Talents; Translational Research; Translations; United States National Institutes of Health; cohesion; cohort; collaborative environment; effective therapy; experience; improved; innovation; insight; material transfer agreement; new therapeutic target; novel therapeutics; programs; public health relevance; success; symposium; tool; web site; web-accessible

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.

项目摘要 多囊肾脏疾病（PKD）是全球肾衰竭的最常见原因。基因 在过去两个 几十年。这些年来的进步已导致了研究囊肿形成和进展的工具，并且 反过 批准的疗法。尽管有这些进展，但是没有干预措施阻止囊性肾脏疾病或 它的系统性表现和对更有效的治疗的迫切需求。为了 促进跨学科和综合方法来识别新型治疗靶标的RFA 已发行以支持PKD研究和翻译核心中心（RTCC）的任务是 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） 对RTCC的高度组织行政支持。我们将实施大胆，创新和外向的 面对计划，该计划将促进RTCC之间的协作和凝聚力并建立机制 用于广泛共享试剂和专业知识。国家协调中心将寻求激动和 通过支持和扩大PKD调查人员的社区，通过促进PKD的研究来激发PKD的研究 数据驱动的科学以及开发鼓励开放和批判性思维的教育活动以及 吸引最聪明的新人才来研究PKD。国家协调中心的领导 由两个调查人员提供了对PKD领域的深入了解，并对 协作科学的重要性。 NCC将支持PKD研究联盟的目标 以下目的：1）召集PKD研究联盟指导委员会开发该计划的 总体方向，组织和执行计划活动，并协助广泛传播 信息2）制定一个多收益的沟通计划，包括用于传播的可访问网站 有关研究资源的信息3）通过建立框架来促进RTCC之间的合作 为了共同开发资源4）促进整个财团的广泛共享5） 一个教育计划，包括一年的科学研讨会，该研讨会将鼓励建设性话语 以及PKD科学的潜水员和创造性方法。 6）发展，支持和执行飞行员和可行性 鼓励新调查人员（早期和成立）进入PKD领域的计划 研究。 7）提供针对早期研究人员的教育节目，以促进 开发新的PKD研究人员和领导者。总之，协作环境 NCC培养将促进我们期望的进步，这将导致对PKD病理学的新见解 以及针对多囊性肾脏疾病患者的新治疗选择。