Boston Children's Hospital/HMS Intellectual and Developmental Disabilities Research Center

波士顿儿童医院/HMS 智力与发育障碍研究中心

基本信息

批准号：
10239463
负责人：
SCOTT Loren POMEROY
金额：
$ 132.6万
依托单位：
BOSTON CHILDREN'S HOSPITAL
依托单位国家：
美国
项目类别：
财政年份：
2021
资助国家：
美国
起止时间：
2021-07-22 至 2026-05-31
项目状态：
未结题

来源：
https://reporter.nih.gov/project-details/10239463
关键词：
Address Advocacy Affect Animal Behavior Animal Model Animals Area Basic Science Behavioral Biochemical Bioinformatics Biological Markers Biological Models Biology Boston Brain Caliber Clinic Clinical Clinical Research Clinical Trials Collaborations Collection Communities Complement Core Facility Development Discipline Disease Disease model Drug Screening Effectiveness Electroencephalography Electrophysiology (science)Encapsulated Ensure Etiology Family Fostering Funding Generations Genes Genetic Genotype Goals Hospitals Human Image Image Analysis In Vitro Individual Institution Intellectual and Developmental Disabilities Research Centers Intellectual functioning disability Investigation Investments Laboratories Leadership Measures Mentors Modeling Modification Molecular Mus Neurodevelopmental Disorder Neurons Neurosciences Organ Pathogenesis Patients Pediatric Hospitals Phenotype Physiological Physiology Prevention strategy Research Research Personnel Research Project Grants Research Support Research Training Resources Rodent Role Services Side Technology Therapeutic Time Tissues Training Training Programs Translational Research Translations Underrepresented Minority United States National Institutes of Health Vision Visualization age group awake base behavior test behavioral phenotyping cellular imaging clinical phenotype community engagement design developmental disease drug discovery educational atmosphere genetic analysis genome editing improved in vivo innovation lectures medical schools meetings neurobehavioral next generation next generation sequencing non-genetic novel novel therapeutics outreach patient advocacy group patient population patient stratification pre-clinical programs recruit research facility stem cell model stem cells symposium targeted treatment tool treatment strategy web site

项目摘要

ABSTRACT Our overarching vision of the Boston Children’s Hospital and Harvard Medical School Intellectual and Developmental Disorders Research Center (IDDRC) is to improve the lives of individuals with IDD with timely and efficient translation of scientific research through collaboration among our institutions’ exceptional investigators and clinicians in partnership with the external IDD community. To achieve our broad vision, we organize the Center’s research around four clearly defined themes: 1) Discovery of genetic and non-genetic causes of IDD; 2) Determination of the cellular bases of IDDs using advanced imaging and analysis tools; 3) Identification of translational phenotypes in animal models of IDD to validate therapeutics; 4) Accelerated translation of research discoveries into new prevention and treatment strategies for IDDs. The Center currently supports 106 research projects and 68 investigators through the Administrative Core and the four scientific Cores. The Administrative Core is the hub of the Center as it provides both scientific and administrative leadership which promotes synergistic, interdisciplinary interactions that address IDD-related issues at multiple levels, trains the next generation of young investigators and facilitates outreach and dissemination of IDD research to diverse audiences. The Genetic Analysis and Editing Core (GAEC) provides access to the latest technological advances both in genetic analysis and in gene editing. The Cellular Imaging Core (CIC) facilitates the study of cellular and circuit biology through state-of-the-art imaging and image analysis services which enable visualization of fixed tissue, in vitro organ explants and in vivo awake behaving model organisms. The Animal Behavior and Physiology (AB&P) Core provides investigators with access to a wide range of validated technologies and scientific expertise for in vivo rodent behavioral, biochemical and physiological measures in a well-controlled and rigorous preclinical setting. Finally, the Clinical Translational Core (CTC) provides full access for IDDRC PIs to all services required for translation of research discoveries into clinical innovation; this ranges from biosample collection and storage, generation of patient-derived stem cell models of diseases, drug screening platforms on the preclinical side to neurobehavioral and electrophysiological assessment of IDD patients of different age groups as well as statistical and regulatory support of clinical trials. The Cores all interact through shared projects, providing complementary expertise and tools to address unique aspects of central scientific questions, and the Core directors meet to exchange ideas and optimize resource utilization in the monthly Executive Committee meetings. This integrated approach aims to enhance the translational potential of basic research in IDD by putting patients at the center of the drug discovery cycle starting with genetic and molecular screens through clinical trials. The Center engages frequently with patient- advocacy groups and the IDD community in a bi-directional manner to ensure that their needs and concerns steer the Center’s efforts.

抽象的我们对波士顿儿童医院和哈佛医学院知识分子的总体愿景发育障碍研究中心（IDDRC）是及时改善患有IDD的人的生活通过我们机构杰出的合作，对科学研究的有效翻译研究人员和临床医生与外部IDD社区合作。为了实现我们的广泛视野，我们组织中心的研究围绕四个明确定义的主题：1）发现遗传和非遗传 IDD的原因； 2）使用高级成像和分析工具确定IDD的细胞基础； 3） IDD动物模型中转化表型的鉴定以验证治疗； 4）加速将研究发现转化为IDD的新预防和治疗策略。当前中心通过行政核心和四个科学支持106个研究项目和68位研究人员内核。行政核心是中心的枢纽，因为它提供了科学和行政领导促进协同的跨学科互动，以解决与IDD相关的问题的多个问题关卡，培训下一代年轻调查人员，并促进IDD的外展和传播对潜水受众的研究。遗传分析和编辑核心（GAEC）提供了对最新的访问遗传分析和基因编辑中的技术进步。细胞成像核（CIC）通过最先进的成像和图像分析服务促进细胞和电路生物学的研究这可以使固定组织，体外器官外植体和体内清醒的行为模型生物的可视化。动物行为和生理（AB＆P）核心为研究人员提供了广泛的范围经体内啮齿动物行为，生化和生理学的经过验证的技术和科学专业知识在控制良好且严格的临床前环境中进行的措施。最后，临床翻译核心（CTC）为IDDRC PI提供了将研究发现转化为临床所需的所有服务的完整访问权限创新;这范围从生物样品收集和存储，生成患者来源的干细胞模型疾病，神经行为和电生理学的临床前的药物筛查平台评估不同年龄组的IDD患者以及临床试验的统计和调节支持。核心都通过共享项目进行交互，提供完整的专业知识和工具来解决独特中心科学问题的各个方面，核心主管会面以交换想法并优化资源在每月执行委员会会议上的利用。这种综合方法旨在增强通过使患者处于药物发现周期的中心，IDD基础研究的翻译潜力通过临床试验从遗传和分子筛选开始。该中心经常与患者合作 - 倡导团体和IDD社区以双向方式确保他们的需求和关注点引导中心的努力。