Developmental Mechanisms of Human Idiopathic Scoliosis

人类特发性脊柱侧凸的发育机制

• 批准号: 10458399
• 负责人: CAROL A WISE
• 金额: $ 35.7万
• 依托单位: UT SOUTHWESTERN MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2016
• 资助国家: 美国
• 起止时间: 2016-09-01 至 2027-05-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10458399
• 关键词: Achievement; Adherence; Administrator; Adolescent; Advisory Committees; Animals; Area; Award; Budgets; California; Caring; Child; Communication; Data; Data Set; Deformity; Deposition; Development; Ensure; Ethics; Funding; Genomics; Goals; Guidelines; Human; IACUC; Idiopathic scoliosis; Institutional Review Boards; Knowledge; Manuscripts; Measures; Medical center; Modeling; Monitor; Musculoskeletal Diseases; Online Systems; Participant; Pathogenesis; Pediatric Hospitals; Persons; Policies; Preparation; Productivity; Publications; Quality Control; Regulation; Regulator Genes; Research; Research Personnel; Resources; San Francisco; Specific qualifier value; Spinal; Texas; TimeLine; United States National Institutes of Health; Universities; Vertebral column; Visualization; Washington; Work; Zebrafish; data sharing; data submission; gene discovery; human subject protection; innovation; large scale data; medical schools; meetings; multiple omics; programs; relational database; tool; tool development; virtual; web portal

PROJECT SUMMARY/ABSTRACT (ADMINISTRATIVE CORE) We have established an innovative Program entitled “Developmental Mechanisms of Human Idiopathic Scoliosis”. This program combines unbiased gene discovery in humans, modeling and gene discovery in zebrafish, and genomic analysis of gene regulatory sequences that are defining the underlying causes of adolescent idiopathic scoliosis (AIS), the most common musculoskeletal disorder in children. Three research groups are leading these efforts. Project 1 “Human” focuses on AIS gene discovery and is led by Carol Wise, PD, PI, University of Texas Southwestern Medical Center and Texas Scottish Rite Hospital for Children. Project 2 “Zebrafish” is led by Lila Solnica-Krezel, Washington University School of Medicine St. Louis, and her co- investigator Dr. Ryan Gray, University of Texas. Project 3 “Genomics” is led by Nadav Ahituv, University of California San Francisco. The Administrative Core will aid activities of the Program, which is already a working collaborative, and will be ultimately responsible for its execution and productivity. The Program Director Dr. Wise, the Core Administrator, Ms. Liza Nowlin, and web portal administrator Dr. Kidane will oversee the administration of the Program. The Core will facilitate organization, communication, data flow, compliance, and productivity between the research groups. Specific responsibilities of the Core will be to ensure effective and frequent communication between component groups of the project by hosting regular meetings either virtually or in person. These will include regular meetings with the Advisory Committee. Primary purposes of the meetings will be to rigorously interpret results ensuring quality control, identify progress toward specified goals, and to interact with the Advisory Committee. The Core will be responsible for confirming that requisite resources are available for each Project, defining timelines for achieving specified goals, monitoring progress toward those goals, and helping to guide any necessary programmatic changes. A major goal of our collaborative is to generate knowledge, tools, and resources that will spur the field of spine deformity research forward. The Administrative Core will also oversee deposition of multi-omics Programmatic data into SpineSource, a new tool that was developed in the prior award cycle to facilitate and centralize communication and data sharing. We expect that SpineSource will become an important resource for not only our team but other researchers and stakeholders in the area of spine and spinal deformity. Otherwise the Administrative Core will be responsible for regularly monitoring budgets and ensuring that funding is disbursed appropriately, and for compliance with ethical guidelines, with institutional and federal requirements (e.g. institutional review boards, institutional animal care and use committees), and with data-sharing policies. Finally, the Core will facilitate the preparation and submission of manuscripts, ensuring fairness and consistency with NIH public access policies. In these ways the Core will promote synergistic interaction within the Program and hasten the goal to decipher the pathogenesis of AIS.

项目摘要/摘要（行政核心） 我们已经建立了一个创新的计划，名为“人类特发性的发展机制 脊柱侧弯”。该程序结合了人类中公正的基因发现，建模和基因发现 斑马鱼和基因调节序列的基因组分析，这些序列定义了根本原因 青春期特发性脊柱侧弯（AIS）是儿童最常见的肌肉骨骼疾病。三项研究 小组正在领导这些努力。项目1“人类”专注于AIS基因发现，并由Carol Wise领导， PD，PI，德克萨斯大学西南医学中心和德克萨斯州苏格兰儿童礼仪医院。项目 2“斑马鱼”由华盛顿大学医学院圣路易斯及其共同的Lila Solnica-Krezel领导 得克萨斯大学的研究者瑞安·格雷博士。项目3“基因组学”由大学纳达夫·阿希图夫（Nadav Ahituv）领导 加利福尼亚旧金山。行政核心将有助于该计划的活动，这已经是一个工作 协作，最终将对其执行和生产力负责。计划主任Wise博士， 核心管理员Liza Nowlin女士和Web Portal管理员Kidane博士将监督管理 程序。核心将托管组织，沟通，数据流，合规性和生产力 在研究小组之间。核心的特定责任将是确保有效和频率 通过虚拟或在项目组件组之间进行沟通。 人。这些将包括与咨询委员会的定期会议。会议的主要目的将是 严格解释结果确保质量控制，确定朝着指定目标的进步，并与 咨询委员会。核心将负责确认每个项目都需要资源， 定义实现指定目标，监视这些目标的进度并帮助指导任何时间表的时间表 必要的程序化更改。我们合作的主要目标是生成知识，工具和资源 将刺激脊柱畸形研究的领域。行政核心还将监督多派的沉积 编程数据到Spinesource，这是一种新工具，是在先前的奖励周期中开发的，以促进和集中 通信和数据共享。我们预计，不仅是我们的团队的刺刺都将成为重要的资源 但是，脊柱和脊柱畸形领域的其他研究人员和利益相关者。否则行政核心 将负责定期监控预算，并确保适当支付资金，并为 遵守道德准则，以及机构和联邦要求（例如机构审查委员会， 机构动物护理和使用委员会）以及数据共享政策。最后，核心将有助于 准备和提交手稿，确保与NIH公共访问政策的公平性和一致性。 通过这些方式，核心将促进程序中的协同互动，并加快破译的目标 AIS的发病机理。