Additional Informatics support on U54 Clinical Core
U54 临床核心的附加信息学支持
基本信息
- 批准号:10890529
- 负责人:
- 金额:$ 6.42万
- 依托单位:
- 依托单位国家:美国
- 项目类别:
- 财政年份:2020
- 资助国家:美国
- 起止时间:2020-07-15 至 2024-06-30
- 项目状态:已结题
- 来源:
- 关键词:Autosomal Dominant Polycystic KidneyBasic ScienceBiocompatible MaterialsBiological MarkersBiomedical ResearchCancer CenterCell modelClinicalClinical ResearchClinical TrialsClinical Trials NetworkCollaborationsCommunitiesComputer softwareData CollectionData SourcesDevelopmentDiseaseEducationEducational workshopEffectivenessEnvironmentFamilyFunctional disorderGoalsGrantInformaticsInfrastructureInstitutionInterdisciplinary StudyKansasLongitudinal cohortMapsMethodsMissionPatient-Focused OutcomesPatientsReagentResearchResearch MethodologyResearch PersonnelResearch PriorityResearch SupportResource SharingResourcesRodent ModelSeriesServicesSiteTechnologyTranslatingTranslationsbasebiomarker discoveryclinical developmentdata formatdata modelingdata standardsdiverse dataimprovedinnovationinstrumentinter-institutionalmeetingsmultidisciplinarynew technologynovel therapeuticspatient engagementpatient orientedprogramsrecruitskillssummer studentsymposiumtherapeutic targettooltranslational pipeline
项目摘要
Project Summary – Overall
The Kansas PKD Research and Translation Core Center (RTCC) is comprised of an Administrative
Core and three innovative Biomedical Research Cores: Core 1: Biomarkers, Biomaterials, and
Cellular Models; Core 2: Rodent Models Core 3: Clinical Research. The Kansas RTCC also offers an
Educational Enrichment Program and an internal Pilot Grant Program in partnership with the KU
Cancer Center. The mission of the PKD Center is to promote PKD research by providing both
innovative and essential reagents, biomaterials, and service to the national PKD research community
for the advancement of our understanding of disease mechanisms, the discovery of biomarkers and
therapeutic targets and the development of clinical trials to improve patient outcomes. The Specific
Aims of the Kansas PKD RTCC are: Aim 1) To support PKD researchers nationally by developing
innovative biomedical research cores. This will be accomplished by developing the expertise,
reagents and resources required to bring the best new technologies to PKD research to meet the
needs of the greater PKD research community; by leveraging existing institutional shared resources
to expand our capability, efficiency, and effectiveness; and by developing tools to support clinical
research and establish collaborations both regionally and nationally to develop a multi-institutional
ADPKD clinical trials network. Aim 2) To develop a skilled, collaborative, multidisciplinary basic
science and clinical PKD research investigator base. This will be accomplished by building on our
broad multidisciplinary research base and continuing to recruit new investigators from outside the
PKD field by offering an innovative pilot grant program that will attract new investigators to the field,
and by providing new investigators, nationally, with research support for core services, new
technologies, educational opportunities, and an open and collaborative research environment that will
help them succeed. Aim 3) To enhance the PKD research environment with educational programs to
support ongoing projects and to develop new research directions. The research environment will be
enhanced through a seminar program, Summer Student Enrichment Program, PKD lab meetings,
PKD methods workshops, Research Methods and Advanced Technologies Program, professional
development seminar series, patient-oriented activities, and by participating in the consortium-wide
annual symposium. Aim 4) To develop partnerships regionally and nationally and to build teams to
serve as a resource for the greater PKD research community. Inter-institutional partnerships will
provide local and national infrastructure support, new research technologies, increased numbers of
patients involved in clinical trials, and research collaborators for the development of new research
opportunities by attracting investigators from other fields into PKD research from collaborating sites
within the PKD Research Consortium and with cooperating institutions nationwide, broadening
opportunities beyond what any single institution can provide.
Project Summary - Clinical Research Core
The overarching challenge in PKD is how to translate the many advances in basic scientific understanding of
the pathophysiology of PKD into the development of effective new drugs. Specific challenges include a
shrinking pool of patients available for clinical trials, lack of biomarkers in early disease, lack of widely-
available, standardized data models to harmonize diverse data sources, and limited engagement of patients
and their families in prioritizing research questions. The goal of the Clinical Research Core is to facilitate
research across the translational spectrum from T1 to T4 by PKD investigators nationwide. This will be
achieved through the development of multiple tools and resources that will be made freely available to the
research community. The specific aims are: 1) to create a consortium of academic centers across the Central
and Western regions to form an ADPKD clinical trial network; 2) to expand and continue to follow a longitudinal
cohort of young patients with early ADPKD and control subjects and collect biospecimens for biomarker
discovery; 3) create a standardized data collection instrument for PKD studies in REDCap and software for
automated mapping into a standard data format required for registration studies; and 4) use our patient
engagement resource to identify patients’ priorities for research questions and use this to inform research
strategy and directions in the PKD community.
项目概要-总体
该堪萨斯PKD研究和翻译核心中心(RTCC)是由一个行政
核心和三个创新的生物医学研究核心:核心1:生物标志物,生物材料,
细胞模型;核心2:啮齿动物模型;核心3:临床研究。堪萨斯RTCC还提供
教育充实计划和与KU合作的内部试点赠款计划
癌症中心PKD中心的使命是通过提供
创新和必要的试剂,生物材料,并为国家PKD研究界提供服务
促进我们对疾病机制的理解,发现生物标志物,
治疗目标和临床试验的发展,以改善患者的结果。具体
堪萨斯PKD RTCC的目标是:目标1)通过开发
创新的生物医学研究核心。这将通过发展专门知识来实现,
为PKD研究带来最佳新技术所需的试剂和资源,以满足
更大的PKD研究社区的需求;通过利用现有的机构共享资源
扩大我们的能力,效率和有效性;并通过开发工具,以支持临床
研究和建立区域和国家合作,以发展多机构
ADPKD临床试验网络。目标2)发展一个熟练的,协作的,多学科的基础
科学和临床PKD研究调查员基地。这将通过在我们的基础上再接再厉来实现
广泛的多学科研究基础,并继续从外部招募新的研究人员,
PKD领域通过提供一个创新的试点赠款计划,将吸引新的调查人员到外地,
通过在全国范围内为新的研究人员提供核心服务的研究支持,
技术,教育机会,以及开放和协作的研究环境,
帮助他们成功。目标3)通过教育计划改善PKD研究环境,
支持正在进行的项目,并开发新的研究方向。研究环境将是
通过研讨会计划,暑期学生充实计划,PKD实验室会议,
PKD方法研讨会,研究方法和先进技术计划,专业
发展研讨会系列,以病人为导向的活动,并通过参与整个财团
年度研讨会。目标4)在区域和国家一级发展伙伴关系,并建立团队,
作为更大的PKD研究社区的资源。机构间伙伴关系将
提供地方和国家基础设施支持,新的研究技术,
参与临床试验的患者,以及开发新研究的研究合作者
通过吸引其他领域的研究人员从合作地点进入PKD研究,
在PKD研究联盟内,并与全国各地的合作机构,扩大
这是任何一个机构都无法提供的机会。
项目总结-临床研究核心
PKD的首要挑战是如何将基础科学认识的许多进展转化为
PKD的病理生理学研究投入到有效新药的开发中。具体挑战包括:
可用于临床试验的患者人数减少,缺乏早期疾病的生物标志物,缺乏广泛的
可用的标准化数据模型,以协调不同的数据源,患者参与有限
和他们的家人优先考虑研究问题。临床研究核心的目标是促进
PKD研究人员在全国范围内进行了从T1到T4的翻译谱研究。这将是
通过开发多种工具和资源,
研究社区。具体目标是:1)在中部地区建立一个学术中心联盟
和西部地区,形成ADPKD临床试验网络; 2)扩大并继续遵循纵向
早期ADPKD年轻患者和对照受试者队列,并收集生物标志物的生物标本
发现; 3)为REDCap中的PKD研究创建标准化的数据收集工具,并为
自动映射到配准研究所需的标准数据格式;以及4)使用我们的患者
参与资源,以确定患者对研究问题的优先级,并利用此为研究提供信息
在PKD社区的战略和方向。
项目成果
期刊论文数量(16)
专著数量(0)
科研奖励数量(0)
会议论文数量(0)
专利数量(0)
PKD1 and PKD2 mRNA cis-inhibition drives polycystic kidney disease progression.
- DOI:10.1038/s41467-022-32543-2
- 发表时间:2022-08-15
- 期刊:
- 影响因子:16.6
- 作者:
- 通讯作者:
The lonidamine derivative H2-gamendazole reduces cyst formation in polycystic kidney disease.
孤胺衍生物H2-gamendazole降低了多囊性肾脏疾病中的囊肿形成。
- DOI:10.1152/ajprenal.00095.2022
- 发表时间:2022-10-01
- 期刊:
- 影响因子:0
- 作者:
- 通讯作者:
Prognostic Value of Fibroblast Growth Factor 23 in Autosomal Dominant Polycystic Kidney Disease.
- DOI:10.1016/j.ekir.2021.01.004
- 发表时间:2021-04
- 期刊:
- 影响因子:6
- 作者:El Ters M;Lu P;Mahnken JD;Stubbs JR;Zhang S;Wallace DP;Grantham JJ;Chapman AB;Torres VE;Harris PC;Bae KT;Landsittel DP;Rahbari-Oskoui FF;Mrug M;Bennett WM;Yu ASL
- 通讯作者:Yu ASL
Polycystin-1 Interacting Protein-1 (CU062) Interacts with the Ectodomain of Polycystin-1 (PC1).
Polycystin-1相互作用的蛋白-1(CU062)与多囊这1(PC1)的外生域相互作用。
- DOI:10.3390/cells12172166
- 发表时间:2023-08-29
- 期刊:
- 影响因子:6
- 作者:
- 通讯作者:
Antioxidant enzyme peroxiredoxin 5 regulates cyst growth and ciliogenesis via modulating Plk1 stability.
- DOI:10.1096/fj.202101270rr
- 发表时间:2022-01
- 期刊:
- 影响因子:0
- 作者:
- 通讯作者:
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{{ truncateString('DARREN P. WALLACE', 18)}}的其他基金
Kansas PKD Research and Translation Core Center
堪萨斯 PKD 研究与翻译核心中心
- 批准号:
10475035 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Biomedical Research Core 1 - Biomarkers, Biomaterials, and Cellular Models Core
生物医学研究核心 1 - 生物标志物、生物材料和细胞模型核心
- 批准号:
10475041 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Biomedical Research Core 1 - Biomarkers, Biomaterials, and Cellular Models Core
生物医学研究核心 1 - 生物标志物、生物材料和细胞模型核心
- 批准号:
10214614 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Biomedical Research Core 1 - Biomarkers, Biomaterials, and Cellular Models Core
生物医学研究核心 1 - 生物标志物、生物材料和细胞模型核心
- 批准号:
10059766 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Kansas PKD Research and Translation Core Center
堪萨斯 PKD 研究与翻译核心中心
- 批准号:
10686046 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Biomedical Research Core 1 - Biomarkers, Biomaterials, and Cellular Models Core
生物医学研究核心 1 - 生物标志物、生物材料和细胞模型核心
- 批准号:
10686060 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
Additional informatics support on U54 Clinical Core
U54 临床核心的附加信息学支持
- 批准号:
10685724 - 财政年份:2020
- 资助金额:
$ 6.42万 - 项目类别:
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