Senator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center

参议员 Paul D. Wellstone 肌营养不良症合作研究中心

• 批准号: 7684829
• 负责人: RICHARD J SAMULSKI
• 金额: $ 140.81万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2008
• 资助国家: 美国
• 起止时间: 2008-09-05 至 2013-08-31
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7684829
• 关键词: Senator; Paul; D.; Wellstone; Muscular

DESCRIPTION (provided by applicant): The NIH Senator Paul B. Wellstone Centers are the centerpiece of the nation's effort to reduce morbidity and mortality from muscular disorders and are a major source of discovery and development of more effective approaches to prevention, diagnosis, and therapy. The University of North Carolina (UNC) "goal" of establishing a premier Wellstone Center in the state of North Carolina has assembled a highly interactive research group creating a dynamic program comprised of experienced clinical investigators (Drs. Powers & Wolff), expert basic laboratories (Drs. Samulski, Xiao, and Beecham), and large animal models (Dr. Kornegay) focused on developing, testing, and establishing therapeutic treatments for Duchenne Muscular Dystrophy (DMD) and other genetic muscle disorders. A major theme of the Center is to advance novel gene based therapies into the clinic for muscle disorders. The impetus for this effort stems from the fact that UNC Gene Therapy Center (GTC) has made significant strides in recent years to build the foundation that will enable development of a full-scale bench-to-bedside effort bringing gene therapy for DMD closer to a reality (e.g. GMP vector production facility, DMD dog model core). In the spirit of this mission, UNC with support from MDA has independently initiated the first gene-based therapy for DMD using a newly engineered AAV vector (Dr. Samulski) and a mini-dystrophin (dys) gene (Dr. Xiao). A common theme emerging from our Phase I studies and a primary focus of the proposal, relates to establishing a clear understanding of safety and efficacy after treatment with gene based therapeutics and advancing these efforts into selected human clinical trials that will show potential for treatment. Continued discussion with FDA for most efficient and prudent manner to advance our gene therapy based efforts to DMD patients with minimum risk and maximum benefit has lead to the proposed MDCRC Wellstone Center application. The MDCRC will consist of 3 projects (Project 1 PI- Dr. Powers coPI Dr. Wolff -, Project 2 PI-Dr. Xiao, Project 3 PI- Dr. Samulski, (Projects 1-3) and four cores: an Administrative core (PI-Dr. Jude Samulski, Co-PI-Dr. Powers) Training Core, Vector Core (PI Dr. Jeffrey Beecham) and Large animal core (PI- Dr. Kornegay).

描述（由申请人提供）：NIH 参议员 Paul B. Wellstone 中心是国家降低肌肉疾病发病率和死亡率努力的核心，也是发现和开发更有效的预防、诊断和治疗方法的主要来源。北卡罗来纳大学 (UNC) 的“目标”是在北卡罗来纳州建立首屈一指的 Wellstone 中心，该研究小组组建了一个高度互动的研究小组，创建了一个由经验丰富的临床研究人员（Powers 和 Wolff 博士）、专家基础实验室（Samulski、Xiao 和 Beecham 博士）以及大型动物模型（Kornegay 博士）组成的动态项目，专注于开发、测试和建立治疗方法 用于杜氏肌营养不良症 (DMD) 和其他遗传性肌肉疾病。该中心的一个主要主题是将新型基因疗法推进肌肉疾病的临床。这一努力的动力源于这样一个事实：北卡罗来纳大学基因治疗中心 (GTC) 近年来在建立基础方面取得了重大进展，该基础将能够开发全面的从实验室到临床的工作，使 DMD 的基因治疗更接近现实（例如 GMP 载体生产设施、DMD 狗模型核心）。本着这一使命的精神，北卡罗来纳大学在 MDA 的支持下，使用新设计的 AAV 载体（Samulski 博士）和迷你肌营养不良蛋白（dys）基因（Xiao 博士）独立启动了第一个基于基因的 DMD 治疗。我们的第一阶段研究中出现的一个共同主题和该提案的主要焦点涉及对基因疗法治疗后的安全性和有效性建立清晰的认识，并将这些努力推进到选定的人体临床试验中，以显示治疗的潜力。与 FDA 继续讨论以最有效和审慎的方式推进我们基于基因治疗的努力，以最小的风险和最大的效益向 DMD 患者提供帮助，从而促成了拟议的 MDCRC Wellstone 中心申请。 MDCRC 将由 3 个项目组成（项目 1 PI-Dr. Powers coPI Dr. Wolff -、项目 2 PI-Dr.Xiao、项目 3 PI-Dr.Samulski（项目 1-3）和四个核心：管理核心（PI-Dr. Jude Samulski、Co-PI-Dr. Powers）、培训核心、Vector 核心（PI Dr. Jeffrey Beecham）和大型动物核心（PI-Dr. Samulski）。科内盖）。