UCLA Muscular Dystrophy Core Center

描述(由申请者提供)：杜兴肌营养不良中心(“CDMD”)在洛杉矶提供了一个学术中心，用于肌肉营养不良的翻译研究。虽然CDMD支持所有与肌肉和肌肉营养不良相关的研究，但其主要关注的是Duchenne肌营养不良症(DMD)，这是儿童最常见的致命性遗传病。CDMD将基础和临床肌肉营养不良研究领域公认的领导者与加州大学洛杉矶分校庞大的技术和教育基础设施结合在一起，以促进退行性肌肉疾病新疗法的发现和开发。我们位于洛杉矶，美国人口最多的县，使我们能够进入一个巨大的集水区，从那里招募CDMD研究的罕见肌肉疾病患者。这些资源，再加上我们现有中心的成功，使CDMD即使不是全国的，也将成为西海岸领先的翻译性肌营养不良中心。自2009年CDMD核心中心获得第一笔资金以来，该中心成功地扩大了专注于肌营养不良症研究的多学科合作数量，并加快了潜在疗法的发现和测试。在过去的四年里，我们已经向17个实验室授予了65万美元的试点和可行性种子赠款，其中12个授予了肌肉或肌肉营养不良研究的新手。CDMD的成员增加了一倍，达到近45名成员。隶属于本中心的研究人员已经启动了13项临床试验和四个治疗靶点的临床前试验。在这项应用中，我们建议进一步扩展我们的基础设施和服务，以创建一个支持肌肉和肌肉营养不良研究发现转化的生态系统。通过一个行政核心、一个浓缩核心和三个基础科学核心，CDMD将为基础科学发现、临床前试验和临床试验的所有方面提供必要的机构资源。TH基础科学核心包括高通量筛选和细胞库核心(核心B)、肌肉表型和成像核心(核心C)和生物信息学和基因组学核心(核心D)，旨在促进发现新的治疗靶点、新化合物并创建模型来测试这些化合物和与肌肉疾病相关的靶点。

DESCRIPTION (provided by applicant): The Center for Duchenne Muscular Dystrophy ("CDMD") provides an academic home in Los Angeles for the conduct of translational research for the muscular dystrophies. Although CDMD supports all muscle and muscular dystrophy related research, its primary focus is Duchenne Muscular Dystrophy (DMD), the most common lethal genetic disorder in children. CDMD unites recognized leaders in basic and clinical muscular dystrophy research with the immense technological and educational infrastructure of UCLA to facilitate discovery and development of new therapeutics for degenerative muscle disease. Our location in Los Angeles, the most populous county in the United States, gives us access to a huge catchment from which to recruit patients with rare muscle diseases studied at CDMD. These resources, coupled with the successes of our existing Center, position CDMD to become the leading translational muscular dystrophy center on the West Coast, if not the nation. Since the first funding of the CDMD Core Center in 2009, the Center has successfully expanded the number of multidisciplinary collaborations focused on muscular dystrophy research and accelerated the discovery and testing of potential therapeutics. In the past four years, we have awarded $650,000 in pilot and feasibility seed grants to 17 labs, and 12 of which went to investigators new to muscle or muscular dystrophy research. Membership in CDMD has doubled to nearly 45 members. Researchers affiliated with our Center have initiated 13 clinical trials and preclinical tests of four therapeutic targets. In this application, we propose to furthe expand our infrastructure and services to create an ecosystem that supports translation of discoveries in muscle and muscular dystrophy research. Through an Administrative Core, an Enrichment Core and 3 basic science cores, CDMD will provide the institutional resources necessary for all aspects of basic science discovery, pre-clinical testing, and clinical trials. Th basic science cores, which include a High Throughput Screening and Cell Repository Core (Core B), a Muscle Phenotyping and Imaging Core (Core C), and a Bioinformatics and Genomics Core (Core D) are designed to facilitate discovery of new therapeutic targets, novel compounds and create models to test these compounds and targets related to muscle disease.