Administrative Core

行政核心

• 批准号: 10261403
• 负责人: ERIC N Olson
• 金额: $ 16.28万
• 依托单位: UT SOUTHWESTERN MEDICAL CENTER
• 依托单位国家: 美国
• 财政年份: 2015
• 资助国家: 美国
• 起止时间: 2015-09-15 至 2025-08-31
• 项目状态: 未结题
• 来源: https://reporter.nih.gov/project-details/10261403
• 关键词: Achievement; Advocate; Applications Grants; Charge; Clinical; Communication; Communities; Duchenne muscular dystrophy; Dystrophin; Ensure; Environment; Family; Fostering; Foundations; Goals; Information Resources; International; Journals; Knowledge; Leadership; Maintenance; Medicine; Methods; Mission; Modern Medicine; Muscle Cells; Muscular Dystrophies; Patient Care; Patients; Public Domains; Public Health; Publishing; Reagent; Research; Research Personnel; Resources; Science; Skeletal Muscle; Training and Education; United States National Institutes of Health; Update; Work; biobank; clinical care; genome editing; innovation; meetings; member; next generation; novel therapeutics; outreach; parent project; regenerative; symposium; web site

Project Summary/Abstract for the Administrative Core The Administrative Core of the UT Southwestern Senator Paul D. Wellstone Muscular Dystrophy Specialized Research Center (MDSRC) will be responsible for the management and administration of the overall Center. The primary goal of the Administrative Core is to ensure our Center remains successful in optimizing genome editing or myoediting as a feasible and practical approach to treating Duchenne muscular dystrophy (DMD). Achievement of the Center’s goal will be a transformative moment in modern medicine and will be a significant contribution to the field of muscular dystrophy research as well as to patient care. In order for the UT Southwestern Wellstone MDSRC to remain successful in its scientific endeavors, the Administrative Core will continue to provide leadership amongst the various investigators within the Center in order to ensure efficient use of resources and the successful completion of the goals outlined in this NIH P50 Grant Application. The Administrative Core will provide an environment to facilitate the sharing and exchange of ideas and discoveries within the Center as well as with the scientific community at-large. The Center will also actively seek to disseminate these discoveries to patients and their families. The nurturing environment that exists at UT Southwestern will foster the training and education of the next generation of clinical and scientific investigators in the field of muscular dystrophy research. Finally, the UT Southwestern Administrative Core will continue its effective communication with the NIH and Wellstone Network Consortium in order to collectively advance the field of muscular dystrophy research forward. The goals set out by the Administrative Core will be accomplished by pursuing the following three specific aims: Specific Aim 1: To facilitate and enhance clinical and scientific interactions within the UT Southwestern Wellstone Muscular Dystrophy Specialized Research Center. Specific Aim 2: To promote the dissemination of knowledge, resources, and discoveries related to genome editing and Duchenne muscular dystrophy. Specific Aim 3: To serve as an advocate for and promote outreach to muscular dystrophy patients and their families regarding novel therapeutics and advances in clinical care. Under the continued effective leadership of the Administrative Core, the UT Southwestern Senator Paul D. Wellstone MDSRC will strive to achieve its overarching goal of optimizing genome editing to be an effective method of permanently restoring dystrophin expression within myocytes and thus provide a long awaited therapy for DMD. Thus, the proposed NIH P50 Grant Application is relevant to and in keeping with the mission of the NIH.

行政核心项目摘要/摘要 德克萨斯大学西南参议员 Paul D. Wellstone 肌肉萎缩症专业的行政核心 研究中心（MDSRC）将负责整个中心的管理和行政工作。 行政核心的主要目标是确保我们中心在优化基因组方面保持成功 编辑或肌编辑作为治疗杜氏肌营养不良症 (DMD) 的可行且实用的方法。 该中心目标的实现将成为现代医学的一个变革时刻，也将是一个重大的里程碑。 对肌营养不良症研究领域以及患者护理的贡献 西南 Wellstone MDSRC 为了在其科学事业中保持成功，行政核心将 继续在中心内的各个调查人员中提供领导，以确保高效 使用资源并成功完成本 NIH P50 拨款申请中概述的目标。 行政核心将提供一个促进思想和发现的共享和交流的环境 该中心还将积极寻求与整个科学界的合作。 将这些发现传播给患者及其家人 UT 的培育环境。 西南航空将促进下一代临床和科学研究人员的培训和教育 最后，德州大学西南行政核心将继续其肌肉营养不良研究领域的研究。 与 NIH 和 Wellstone 网络联盟进行有效沟通，共同推进 行政核心制定的目标将是肌肉营养不良研究领域的发展。 通过追求以下三个具体目标来实现： 具体目标 1：促进和加强临床 德克萨斯大学西南威尔斯通肌营养不良症专业研究中的科学互动 中心具体目标 2：促进相关知识、资源和发现的传播 基因组编辑和杜氏肌营养不良症具体目标 3：倡导和促进。 向肌营养不良症患者及其家人宣传新的治疗方法和进展 在行政核心的持续有效领导下，UT西南大学。 参议员 Paul D. Wellstone MDSRC 将努力实现其优化基因组编辑的总体目标 是永久恢复肌细胞内肌营养不良蛋白表达的有效方法，从而提供 因此，拟议的 NIH P50 拨款申请与 DMD 相关且保持一致。 NIH 的使命。