UCLA Muscular Dystrophy Core Center

描述（由申请人提供）：杜氏肌营养不良症中心（“CDMD”）在洛杉矶提供了一个学术之家，用于进行肌营养不良症的转化研究。虽然CDMD支持所有肌肉和肌营养不良症相关的研究，但其主要重点是杜氏肌营养不良症（DMD），这是儿童中最常见的致命遗传疾病。CDMD将基础和临床肌营养不良研究领域的公认领导者与加州大学洛杉矶分校的巨大技术和教育基础设施结合起来，以促进退行性肌肉疾病新疗法的发现和开发。我们在洛杉矶，在美国人口最多的县的位置，使我们能够进入一个巨大的集水区，从那里招募在CDMD研究的罕见肌肉疾病的患者。这些资源，加上我们现有中心的成功，使CDMD成为西海岸领先的转化型肌营养不良症中心，如果不是全国的话。自2009年CDMD核心中心首次获得资助以来，该中心已成功扩大了专注于肌营养不良研究的多学科合作的数量，并加速了潜在疗法的发现和测试。在过去的四年里，我们已经向17个实验室提供了65万美元的试点和可行性种子赠款，其中12个实验室用于肌肉或肌肉萎缩症研究的新研究人员。荒漠化和干旱理事会的成员增加了一倍，达到近45个成员。我们中心的研究人员已经启动了四个治疗靶点的13项临床试验和临床前试验。在此应用中，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萎缩症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，临床前测试和临床试验的各个方面提供必要的机构资源。这些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）以及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、新型化合物，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.