Baylor College of Medicine Intellectual and Developmental Disabilities Research Center

贝勒医学院智力与发育障碍研究中心

• 批准号: 10085940
• 负责人: David Loren Nelson
• 金额: $ 127.85万
• 依托单位: BAYLOR COLLEGE OF MEDICINE
• 依托单位国家: 美国
• 财政年份: 2020
• 资助国家: 美国
• 起止时间: 2020-07-22 至 2025-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10085940
• 关键词: Advanced Development; Age; Basic Science; Biological Markers; Blood; Caring; Cells; Clinical; Clinical Research; Clinical Trials; Collaborations; Communication; Communities; Core Facility; Cost Effectiveness Analysis; Coupled; DNA; Development; Disease; Education; Environment; Fostering; Future; Gene Dosage; Generations; Genetic; Goals; Human Genetics; Individual; Influentials; Infrastructure; Institution; Intellectual and Developmental Disabilities Research Centers; Intellectual functioning disability; International; Intervention; Investigation; Laboratories; Leadership; Medicine; Mentors; Mentorship; Mission; Molecular; Molecular Genetics; Neurons; Neurosciences; Oligonucleotides; Outcome; Pathogenesis; Pediatric Hospitals; Pediatrics; Personal Satisfaction; Phase; Phenotype; Philosophy; Physicians; Play; Population; Psychiatry; Quality of life; Readiness; Reproducibility; Research; Research Activity; Research Infrastructure; Research Personnel; Research Project Grants; Role; Safety; Science; Scientist; Services; Shapes; Study models; Techniques; Texas; Therapeutic; Tissues; Training; Training and Education; Translational Research; Vision; Whole Organism; animal model development; base; biomarker panel; clinical heterogeneity; college; community partnership; cost effective; dissemination research; drug discovery; education planning; educational atmosphere; gene therapy; improved; in vivo Model; innovation; learning community; multidisciplinary; next generation; novel diagnostics; novel therapeutic intervention; operation; pre-clinical; preclinical study; programs; recruit; specific biomarkers; synergism; therapeutic development

PROJECT SUMMARY/ABSTRACT: The Eunice Kennedy Shriver Intellectual and Developmental Disabilities Research Center at Baylor College of Medicine (BCM IDDRC) has been instrumental in advancing basic science, translational, and clinical endeavors to improve the lives of individuals with intellectual and developmental disability (IDD). Beyond discoveries, the Center has mentored more than two generations of scientists and physicians engaged in research and the care and treatment of individuals with IDD. The mission of the BCM IDDRC is to identify as many causes of IDD as possible, to understand their pathogenesis, and to develop novel diagnostic and therapeutic approaches. To realize this mission, accelerate the research activities of our Investigators and advance development of therapeutics for IDD, we will carry out the following aims: 1) Provide Core facilities and services to advance IDD research. Six cores are proposed to provide innovative, high-quality and cost-effective research services to assist investigators in studies of molecules (Molecular and Expression Analysis), cells and tissues (Cell and Tissue Pathogenesis), circuits (Circuit Analysis and Modulation), and whole organisms (Preclinical and Clinical Outcomes). The Clinical Translational Core will provide services specific for clinical research infrastructure and the Center Administration Core will coordinate overall Center operations along with stakeholder engagement, communication and education; 2) Promote and enhance collaborative efforts and dissemination activities with a comprehensive engagement, communication, and education plan. The Admin Core will promote interactions locally, nationally, and internationally, will implement best practices for community partnerships and dissemination of research findings, and will enhance the training of next-generation IDD researchers; 3) Conduct a multidisciplinary signature research project that leads to clinical trial readiness. The emergence of DNA-based therapies, coupled with exciting discoveries and preclinical studies from the BCM IDDRC, provides exciting opportunities to treat IDDs, but the fact that many IDD-causing genes are dosage sensitive (too much or too little is detrimental) poses a serious challenge requiring robust biological markers meaningful for the individual rather than the population. The Signature Project seeks to develop multidimensional biomarkers (molecules and circuits) for target engagement, safety, and efficacy in six gene dosage dependent IDDs. The Center will support 75 investigators and 72 research projects. For 30+ years the BCM IDDRC has had a profound impact on IDD, elucidating causes, determining mechanisms, and developing interventions. It has fostered an environment that welcomes and supports additional investigators and emphasizes training. As we enter the next decade, Center investigators, their collaborators, and trainees are poised to transform dozens of exciting discoveries into safe therapeutics that will improve the quality of life and well-being of individuals with IDD.

项目摘要/摘要：Eunice Kennedy Shriver知识和发育障碍 贝勒医学院（BCM IDDRC）的研究中心在推进基本方面发挥了作用 科学，翻译和临床努力，以改善具有智力和知识分子的人的生活 发育障碍（IDD）。除了发现之外，该中心还指导了两代以上 科学家和医生从事IDD患者的研究以及护理和治疗。任务 BCM IDDRC是尽可能多的IDD原因，了解其发病机理，并 开发新颖的诊断和治疗方法。要实现这一任务，请加速研究 我们的调查人员的活动并提高IDD治疗学的开发，我们将进行以下 目的：1）提供核心设施和服务以推进IDD研究。建议六个核心提供 创新，高质量和具有成本效益的研究服务，以帮助研究人员进行分子研究 （分子和表达分析），细胞和组织（细胞和组织发病机理），电路（电路 分析和调节）以及整个生物（临床前和临床结果）。临床 转化核心将为临床研究基础设施和中心提供特定的服务 管理核心将协调整体中心运营以及利益相关者的参与， 沟通和教育； 2）促进和加强协作努力和传播活动 通过全面的参与，沟通和教育计划。管理员核心将促进 在本地，国内和国际上的互动将实施社区伙伴关系的最佳实践 并传播研究结果，并将增强下一代IDD的培训 研究人员； 3）进行多学科签名研究项目，导致临床试验 准备。基于DNA的疗法的出现，再加上令人兴奋的发现和临床前研究 来自BCM IDDRC，提供了治疗IDD的激动人心的机会，但是许多引起IDD的基因的事实 剂量敏感（太多或太少是有害的）是否构成了一个严重的挑战，需要强大的生物学 标记对个人而不是人口有意义。签名项目旨在发展 六个基因的目标参与，安全性和功效的多维生物标志物（分子和电路） 剂量依赖IDD。该中心将支持75名调查人员和72个研究项目。 30多年 BCM IDDRC对IDD，阐明原因，确定机制和发展有深远的影响 干预措施。它培养了一个欢迎和支持其他调查人员和的环境 强调培训。当我们进入接下来的十年时，中心调查员，他们的合作者和学员是 准备将数十种令人兴奋的发现转变为安全的治疗剂，以改善生活质量和 患有IDD的人的福祉。