Clinical Translational Core

临床转化核心

基本信息

批准号：
10085126
负责人：
Christina Gurnett
金额：
$ 19.15万
依托单位：
WASHINGTON UNIVERSITY
依托单位国家：
美国
项目类别：
财政年份：
资助国家：
美国
起止时间：
至
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10085126
关键词：
Affect Award Behavior Behavioral Biological Models Brain Child Abuse Childhood Clinical Clinical Data Clinical Research Clinical Trials Clinical Trials Unit Complement Core Facility Data Development Disease Electrophysiology (science)Faculty Family Fragile X Syndrome Funding Genes Genetic Genetic Counseling Genome Genomics Genotype Health Human Impairment Individual Infant Development Informatics Infrastructure Institutes Institutional support resources Insurance Intellectual and Developmental Disabilities Research Centers Intellectual functioning disability Intervention Intervention Trial Investments K-Series Research Career Programs Laboratories Link Medical Natural History Near-Infrared Spectroscopy Neurodevelopmental Disorder Outcome Pathogenesis Pathogenicity Patient Participation Patient Recruitments Patient observation Patients Phenotype Physicians Populations at Risk Prevention Research Research Personnel Research Support Resources Risk Role Science Secure Services Structure Syndrome Technology Testing Therapeutic Training Universities Variant Washington base behavioral genomics biomarker development brain circuitry cost data acquisition design early detection biomarkers follow-up genomic data human subject innovation medical schools neuroimaging neuropsychiatry novel strategies patient population personalized intervention premature prevent programs psychosocial social visual tracking

项目摘要

Clinical Translational Core Project Summary / Abstract Major advances in our understanding of the genetic and environmental structure of neurodevelopmental disorders present new opportunities to prevent or ameliorate intellectual and developmental disabilities (IDD). The Clinical Translational Core (CTC) of the IDDRC@WUSTL will serve as an essential institutional resource for supporting research that applies scientific discoveries on pathogenic mechanisms of IDD to benefit actual patients through the development of high-impact personalized intervention that is guided by information generated from natural history studies, biomarker development, and clinical trials. The CTC will serve as an integrative scientific hub of the IDDRC@WUSTL linking patients along with their clinical and behavioral data to clinical studies in the CTC and to research opportunities with the Model Systems Core and the Developmental Neuroimaging Core. The Specific Aims of the CTC, designed to accelerate translational advances in IDD, are as follows: 1) To provide investigators with the expertise, resources, and assessment services needed to conduct comprehensive clinical, behavioral, and genomic characterization of human subjects for IDD-related research and clinical trials through the Human Genomic and Phenotypic Characterization Unit (HGPCU) and Clinical Trials Unit (CTU), and to leverage this activity with established, state-of-the-art genome technology services at WUSTL to contribute to understanding of the pathogenicity and functional significance of variation in genes influencing the developing human brain. 2) To strategically harness services and expertise of allied WUSTL facilities that critically and cost-effectively complement the IDDRC@WUSTL scientific cores to contribute to a discovery pipeline for higher-impact intervention and clinical trials for individuals affected by or at risk for IDD. Initial implications-for-treatment of new discoveries in IDD pathogenesis are often first discovered through advances in the Model Systems Core and made ready for translational applications utilizing CTC services. Similarly, brain circuitry data from the Developmental Neuroimaging Core are assimilated with behavioral and genomic data collected using CTC resources to identify early biomarker risk profiles and inform developmental timing of intervention and prevention, 3) To promote bidirectional interchange between scientific activity in the discovery of pathogenic mechanisms and the testing of novel approaches to IDD prevention and treatment in human subjects and among populations at risk. This will occur in the context of a dynamic interface with the other two scientific core facilities of the IDDRC@WUSTL (the Developmental Neuroimaging and Model Systems Cores), facilitated by dedicated faculty liaisons whose roles are to cultivate cross-disciplinary science.

临床转化核心项目总结/摘要我们对神经发育的遗传和环境结构的理解取得了重大进展疾病为预防或改善智力和发育障碍（IDD）提供了新的机会。 IDDRC@WUSTL 的临床转化核心 (CTC) 将作为重要的机构资源支持将科学发现应用于 IDD 致病机制的研究，以造福于实际通过开发以信息为指导的高影响力的个性化干预措施来帮助患者由自然历史研究、生物标志物开发和临床试验产生。 CTC 将作为 IDDRC@WUSTL 的综合科学中心将患者及其临床和行为数据与 CTC 的临床研究以及模型系统核心和发展的研究机会神经影像核心。 CTC 旨在加速 IDD 转化进展的具体目标是如下： 1) 为研究者提供开展研究所需的专业知识、资源和评估服务人类受试者的全面临床、行为和基因组特征，用于 IDD 相关研究以及通过人类基因组和表型表征单元 (HGPCU) 进行的临床试验和临床试验单位 (CTU)，并利用 WUSTL 现有的最先进的基因组技术服务来利用这项活动有助于理解影响基因变异的致病性和功能意义正在发育的人类大脑。 2) 战略性地利用 WUSTL 联盟设施的服务和专业知识，关键且经济高效地补充 IDDRC@WUSTL 科学核心，为发现做出贡献为受 IDD 影响或面临 IDD 风险的个人提供更具影响力的干预和临床试验。最初的 IDD发病机制新发现对治疗的影响通常是通过进展首先发现的位于模型系统核心中，并为利用 CTC 服务的转化应用程序做好了准备。同样，大脑来自发育神经影像核心的电路数据与行为和基因组数据同化使用 CTC 资源收集来识别早期生物标志物风险状况并告知发育时间干预和预防，3）促进发现中科学活动之间的双向交流人类IDD预防和治疗的致病机制和新方法的测试受试者和高危人群。这将发生在与其他两个的动态接口的上下文中 IDDRC@WUSTL（发育神经成像和模型系统核心）的科学核心设施，由专门的教员联络员协助，其作用是培养跨学科科学。