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.

项目摘要/行政核心摘要 UT西南部参议员Paul D. Wellstone肌营养不良专科 研究中心（MDSRC）将负责整个中心的管理和行政工作。 管理核心的主要目标是确保我们的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。 实现该中心的目标将是现代医学的一个变革性时刻， 为肌营养不良症研究领域以及患者护理做出贡献。为了让UT 西南井石MDSRC在其科学努力保持成功，行政核心将 继续领导中心内的各种调查人员，以确保有效的 资源的使用和成功完成本NIH P50补助金申请中概述的目标。的 行政核心将提供一个环境，以促进分享和交流的想法和发现 中心内部以及整个科学界。中心还将积极寻求 将这些发现传播给患者及其家属。存在于UT的培育环境 西南大学将促进下一代临床和科学研究人员的培训和教育 在肌肉萎缩症研究领域。最后，UT西南行政核心将继续其 与NIH和Wellstone Network Consortium进行有效沟通，以共同推进 肌营养不良症的研究领域向前迈进。行政核心设定的目标将是 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 UT西南韦尔斯通肌肉营养不良专门研究内的科学互动 中心具体目标2：促进传播与以下方面有关的知识、资源和发现： 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属宣传新的治疗方法和进展， 临床护理在行政核心的持续有效领导下，UT西南 参议员保罗·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， 期待已久的DMD治疗方法因此，拟议的NIH P50补助金申请与 国家卫生研究院的使命。