Administrative Core

行政核心的项目摘要/摘要 德克萨斯州西南部参议员保罗·D·威尔斯通的行政核心--肌肉营养不良专科 研究中心(MDSRC)将负责整个中心的管理和行政。 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 编辑或肌肉编辑是治疗Duchenne肌营养不良症(DMD)的一种可行和实用的方法。 该中心目标的实现将是现代医学的一个变革性时刻，也将是一个重要的 对肌肉营养不良研究领域以及对患者护理的贡献。为了让德克萨斯大学 西南威尔斯通MDSRC为了保持其科学努力的成功，行政核心将 继续在中心内的不同调查员之间提供领导，以确保高效 资源的使用和成功完成本NIH P50拨款申请中概述的目标。这个 管理核心将提供一个环境，以促进思想和发现的分享和交流 在中心内部以及与整个科学界的合作。该中心还将积极寻求 将这些发现传播给患者及其家属。存在于德克萨斯大学的培育环境 西南大学将培养和教育下一代临床和科学研究人员 在肌肉营养不良研究领域。最后，德克萨斯大学西南行政核心将继续其 与NIH和Wellstone Network财团进行有效沟通，以共同推动 肌营养不良症领域的研究进展。行政核心制定的目标将是 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 与科学互动的德克萨斯大学西南部韦尔斯通肌营养不良专科研究 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 基因组编辑和杜兴氏肌营养不良症。具体目标3：倡导和促进 向肌营养不良症患者及其家人介绍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，德克萨斯大学西南 参议员保罗·D·威尔斯通MDSRC将努力实现其首要目标，即优化基因组编辑以 是一种永久恢复肌细胞内肌营养不良蛋白表达的有效方法，从而提供一种 期待已久的DMD治疗。因此，建议的NIH P50拨款申请是相关的，并保持 美国国立卫生研究院的使命。

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.