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

基本信息

批准号：
8333446
负责人：
RICHARD J SAMULSKI
金额：
$ 139.17万
依托单位：
UNIV OF NORTH CAROLINA CHAPEL HILL
依托单位国家：
美国
项目类别：
财政年份：
2008
资助国家：
美国
起止时间：
2008-09-05 至 2014-02-28
项目状态：
已结题

项目摘要

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).

NIH参议员Paul B. Wellstone中心是该国努力降低肌肉疾病发病率和死亡率的核心，是发现和开发更有效的预防，诊断和治疗方法的主要来源。 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用于Duchenne肌肉营养不良（DMD）和其他遗传肌肉疾病。该中心的主要主题是将基于基因的新型疗法推向肌肉疾病的诊所。这项工作的推动力源于近年来UNC基因治疗中心（GTC）取得了重大进步，以建立基础，该基金会将使能够发展全面的基准 - 卧铺工作，从而使DMD的基因治疗使DMD更接近现实（例如GMP Vector Production Production Facity，DMD Dog Model Core）。本着这项任务的精神，在MDA的支持下，UNC使用新设计的AAV载体（萨默斯基博士）和一个迷你肌营养物（DYS）基因（Xiao博士）独立启动了基于基因的DMD疗法。从我们的第一阶段研究和提案的主要重点中出现的一个共同主题，与基于基因的治疗剂治疗后对安全性和有效性的清晰了解有关，并将这些努力推进了精选的人类临床试验，这将显示出潜在的治疗。继续与FDA讨论最有效，最谨慎的方式，以推动我们基于基因治疗的努力对具有最低风险和最大收益的DMD患者的努力，这导致了拟议的MDCRC Wellstone Center应用。 MDCRC将由3个项目（项目1 Pi- Powers Copi博士沃尔夫 - 沃尔夫 - Xiao博士，项目3 Project 3 Pi- samulski博士，（项目1-3）和四个核心：行政核心（Pi-Dr。JudeSamulski，co-pi-dr。co-pi-dr。

项目成果

期刊论文数量（8）

专著数量（0）

科研奖励数量（0）

会议论文数量（0）

专利数量（0）

K137R mutation on adeno-associated viral capsids had minimal effect on enhancing gene delivery in vivo.

腺相关病毒衣壳上的 K137R 突变对增强体内基因递送的影响极小。

DOI：
10.1089/hgtb.2013.176
发表时间：
2014
期刊：
Human gene therapy methods
影响因子：
0
作者：
Qiao,Chunping;Li,Chengwen;Zhao,Chunxia;Li,Jianbin;Bian,Tao;Grieger,Joshua;Li,Juan;Samulski,RJude;Xiao,Xiao
通讯作者：
Xiao,Xiao

Intravenous thrombolysis of basilar artery thrombosis.

基底动脉血栓的静脉溶栓。