Administrative Core

行政核心(J.Mendell，PI)将为MDCRC方案提供科学和财政监督和指导。核心将建立在国家儿童医院(NCH)研究所神经肌肉临床研究计划的现有专业知识之上。已经由国际翻译协会召集的翻译研究人员，门德尔博士，在设计和实施方面具有证明的专业知识 前沿临床研究项目。工作人员在协调大型多中心合作项目方面的经验与魏尔斯通MDCRC的管理直接相关，包括协调多个地点之间的通信、质量控制和文件跟踪、数据管理、数据挖掘和报告、里程碑和任务管理以及财政问责方面的经验。联合营养不良症项目将促进患者的接触，这是一个由NIH资助的大型多中心基因/表型和自然历史联盟，由最近加入NCH基因治疗中心的Kevin Flanigan博士领导。门德尔博士在DMD临床试验的设计和执行方面拥有丰富的经验，包括在基因转移试验的执行方面的独特经验，他和弗拉尼根博士都曾担任目前正在进行的基因转移或基因纠正(外显子跳过和无稽之谈抑制)试验的设计顾问。 该核心将负责在NCH MDCRC内部和更广泛的Weilstone网络之间建立和保持调查人员之间的有效沟通与合作。在这一角色中，它将制定明确的内部和外部程序，以监测和评估研究项目和核心设施；建立机制，在研究人员之间管理和共享数据、动物模型和其他资源；并为外部资助和行业赞助的翻译研究项目的发展以及千年发展目标中心的教育核心活动提供行政支助。 行政核心还将设立一个中心咨询委员会，并制定框架，以确保该委员会对监督中心的研究和翻译活动作出贡献。

The Administrative Core (J. Mendell, PI) will provide scientific and fiscal oversight of and direction to the MDCRC Program. The Core will build upon the existing expertise of the Neuromuscular Clinical Research Program at the Research Institute at Nationwide Children's Hospital (NCH). The translational research staff already assembled by the PI, Dr. Mendell, has a demonstrated expertise in designing and implementing cutting-edge clinical research projects. The staff's experience in coordinating large multi-center collaborative programs is directly relevant to administration of a Weilstone MDCRC, and includes experience in coordination of communication among multiple sites; quality control and document tracking; data management; data mining and reporting; milestone and task management; and fiscal accountability. Access to patients will be facilitated by the United Dystrophinopathy Project, a large NIH-funded multicenter genotype/phenotype and natural history consortium directed by Dr. Kevin Flanigan, who has recently joined the NCH Center for Gene Therapy. Dr. Mendell has extensive experience in the design and performance of DMD clinical trials, including unique experience in the performance of gene transfer trials, and both he and Dr. Flanigan have served as consultants for the design of trials of gene transfer or gene correction (exon skipping and nonsense suppression) trials that are currently underway. The Core will be responsible for establishing and maintaining effective communications and cooperation among investigators both inside the NCH MDCRC and among the wider Weilstone network. In this role, it will develop clear internal and external procedures for monitoring and evaluating the research projects and core facilities; establish mechanisms for managing and sharing data, animal models, and other resources among investigators; and provide administrative support for the development of both extramural funded and industry-sponsored translational research projects, and for the Educational Core activities of the MDCRC. The Administrative Core will also establish a Center Advisory Committee, and develop the framework to ensure the contribution of this Committee to the oversight of the Center's research and translational activities.