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: 7932344
• 负责人: RICHARD J SAMULSKI
• 金额: $ 5.23万
• 依托单位: UNIV OF NORTH CAROLINA CHAPEL HILL
• 依托单位国家: 美国
• 财政年份: 2009
• 资助国家: 美国
• 起止时间: 2009-09-25 至 2010-09-24
• 项目状态: 已结题
• 来源: https://reporter.nih.gov/project-details/7932344
• 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）：美国国立卫生研究院参议员保罗B。Wellstone中心是国家努力降低肌肉疾病发病率和死亡率的核心，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。北卡罗来纳州大学（University of North Carolina）的“目标”是在北卡罗来纳州建立一个首屈一指的威尔斯通中心，该大学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组成的动态项目（鲍尔斯和沃尔夫博士），专家基础实验室（Samulski，Xiao和Beecham博士）和大型动物模型Kornegay博士专注于开发，测试和建立杜氏肌营养不良症（DMD）和其他遗传性肌肉疾病的治疗方法。该中心的一个主要主题是将新的基于基因的疗法推进到肌肉疾病的临床中。这一努力的动力源于这样一个事实，即近年来，DMD基因治疗中心（GTC）在建立基础方面取得了重大进展，这将使全方位的实验室到床边的努力得以发展，使DMD的基因治疗更接近现实（例如GMP载体生产设施，DMD犬模型核心）。本着这一使命的精神，在MDA的支持下，Zynga独立启动了第一个基于基因的DMD治疗，使用新工程的AAV载体（Samulski博士）和微型肌营养不良蛋白（dys）基因（Xiao博士）。我们的I期研究中出现的一个共同主题和提案的主要焦点涉及建立对基于基因的疗法治疗后的安全性和有效性的明确理解，并将这些努力推进到选定的人类临床试验中，这些试验将显示出治疗潜力。继续与FDA讨论以最有效和谨慎的方式推进我们对DMD患者的基因治疗，以最小的风险和最大的获益，这导致了拟议的MDCRC Wellstone中心申请。MDCRC将包括3个项目（项目1 PI-Powers博士，共同PI Wolff博士，项目2 PI-Xiao博士，项目3 PI-Samulski博士，（项目1-3）和4个核心：管理核心（PI-Jude Samulski博士，共同PI-Powers博士）培训核心，载体核心（PI-Jeffrey Beecham博士）和大型动物核心（PI-Kornegay博士）。