Mayo Translational PKD Center (MTPC)

 描述(由申请人提供)：本建议书是为延续NIH/NIDDK研究和翻译性PKD研究基金提交的，该基金于2010年7月1日开始资助，名称为“梅奥翻译性PKD中心”(MTPC)。MTPC的总体目标是开发核心设施，以支持和鼓励Mayo和非Mayo研究人员进行新的PKD研究，并通过临床试验的实施，促进将基础研究突破转化为临床实践的改进。MTPC生物医学研究的三个核心是在对实现MTPC目标最有效的服务进行深思熟虑的分析后选择的，建立在Mayo PKD调查人员现有优势的基础上，并根据我们过去四年的运作经验进行了更新。分子遗传学和生物标记物核心(Peter Harris博士，Pi)的重点是确定PKD人群的特征，以促进临床试验，帮助基因/表型研究和基因发现，并提供材料和信息以促进对疾病发病机制的理解。模型系统核心(Stephen Ekker博士，Pi)的重点是通过促进获取PKD线虫、斑马鱼和啮齿动物模型来促进PKD研究，为新的生物特征提供专业知识，并测试新的治疗方法。人类成像核心(Dr.Bradley Erickson，PI)的重点是提供准确可靠的成像数据分析，并应用新的和增强的定性和定量方法来衡量ADPKD的疾病进展和治疗结果。试点和可行性计划(Michael Romero博士，PI)的重点是吸引新的研究人员进入PKD研究，并提供资金和指导，以促进发现和产生强大的赠款申请，从而通过培养新一代PKD研究人员来加强研究基础。在其存在的四年中，MTPC根据调查和反馈改进了中心的活动，以确定成员需求，加强了其内部研究基础，并增加了一个大型的PKD调查人员外部研究基地。三个频繁使用的生物医学研究核心提供的服务和尖端技术得到了扩展，重点放在需求和兴趣较高的领域。到目前为止，试点和可行性计划已经为12个项目提供了出色的投资回报，我们已经实施了一个成功的科学丰富计划，促进了其成员之间的协同作用，并通过与机构设施、其他梅奥和非梅奥国立卫生研究院资助的中心和产业界合作，建立了研究能力。因此，MTPC已经实现了NIDDK为其PKD研究和翻译核心中心计划设定的目标，并处于有利地位，将在未来五年取得这些成功。

 DESCRIPTION (provided by applicant): This proposal is submitted for renewal of the NIH/NIDDK Research and Translational PKD Research grant entitled "Mayo Translational PKD Center" (MTPC) that began funding on July 1, 2010. The overall goal of MTPC is to develop Core facilities to support existing and stimulate new PKD research by Mayo and non-Mayo investigators and to facilitate the translation of basic research breakthroughs into improvements in clinical practice through the implementation of clinical trials. The three MTPC Biomedical Research Cores were chosen after thoughtful analysis of the services that would be most effective in achieving the MTPC goals, build on the existing strengths of the PKD investigators at Mayo, and updated in response to our experiences over the past four years of operation. The focus of the Molecular Genetics and Biomarker Core (Dr. Peter Harris, PI) is to characterize PKD populations to facilitate clinical trials, to aid genotype/phenotype studies and gene discovery, and to provide materials and information to catalyze understanding of disease pathogenesis. The focus of the Model Systems Core (Dr. Stephen Ekker, PI) is to empower PKD research through facilitating access to PKD C. elegans, zebrafish and rodent models, to provide expertise for novel organism characterization, and to test novel therapeutics. The focus of the Human Imaging Core (Dr. Bradley Erickson, PI) is to provide accurate and reliable analysis of imaging data and to apply new and enhanced qualitative and quantitative methodologies to measure disease progression and therapeutic outcomes in ADPKD. The focus of the Pilot and Feasibility Program (Dr. Michael Romero, PI) is to attract new investigators into PKD research and provide funds and guidance to foster discovery and the generation of strong grant applications, thus strengthening the research base by developing a new generation of PKD researchers. During its four years of existence, MTPC has refined the activities of the Center in response to surveys and feedback to ascertain membership needs, strengthened its Internal Research Base and added a large External Research Base of PKD investigators. The services and cutting-edge technologies provided by the three heavily used Biomedical Research Cores have been expanded, focusing on areas of high demand and interest. The Pilot and Feasibility Program has so far funded 12 projects with an excellent return for investment, we have implemented a successful Scientific Enrichment Program that promotes synergy among its members, and built research capacity by partnering with Institutional Facilities, other Mayo and non-Mayo NIH funded Centers, and industry. Thus, MTPC has fulfilled the goals set forth by NIDDK for its PKD Research and Translation Core Centers program and is well positioned to build on these successes in the next five years.