UCLA Muscular Dystrophy Core Center

描述（由申请人提供）：加州大学洛杉矶分校的肌营养不良核心中心由一组对退行性肌肉疾病治疗方法的开发感兴趣的科学家组成。该中心的目标是通过提供资源，试点资金和智力丰富的环境来改善和促进肌营养不良症的发现和临床前测试过程，以促进研究进展，并鼓励肌肉研究人员与其他研究人员之间的富有成效的互动校园内相关学科。该中心的存在将吸引既有肌肉研究人员，也有以前没有进行肌肉研究的科学家，并通过这种联系，为该小组增加新的专业知识和经验。Cores将使用基于新型细胞测定和新型转基因小鼠模型的高通量和非侵入性分析。中心科学家使用的高通量和非侵入性策略与肌肉疾病传统上无关，因此可能会促进新的进步和治疗方法的快速发展。该中心的成员反映了各种不同的科学专业知识，将促进这一奋进。行政核心的目的是协调中心的活动，并促进这些目标。 具体而言，行政核心的目标是：目标1：为核心和中心的所有活动提供行政支持。目标二：促进中心科学家之间的合作，并通过组织丰富活动，如研讨会系列，年度务虚会和网站的维护，鼓励核心的使用。目标3：监督试点项目和可行性项目的审查和供资情况。目标4：促进营养不良研究者社区的数据共享和核心的使用，以便核心将为肌营养不良研究提供国家资源。

DESCRIPTION (provided by applicant): The Muscular Dystrophy Core Center at UCLA is comprised of a group of scientists interested in the development of therapeutics for degenerative muscle disease. The goal of the Center is to improve and facilitate the process of discovery and pre-clinical testing for muscular dystrophy by providing resources, pilot funding and an intellectually rich environment that promotes research progress and encourages productive interactions between muscle researchers and other researchers in related disciplines on campus. The existence of the Center will attract scientists that are both established muscle researchers and those that have not previously conducted muscle research and, with this association, add new expertise and experience to the group. The Cores will use high throughput and non-invasive analyses based on novel cellular assays and novel genetically modified mouse models. The high throughput and non-invasive strategies being used by Center Scientists are not traditionally associated with muscle disease and may thereby foster novel advancements and rapid development of therapeutics. The Center membership reflects a variety of different scientific expertise that will facilitate this endeavor. The aims of the Administrative Core are to coordinate the activities of the Center and to facilitate these goals. Specifically the Aims of the Administrative Core are: Aim 1: To provide administrative support to the Cores and all activities of the Center. Aim 2: To facilitate collaboration between Center Scientists and encouragement of Core usage by organizing enrichment activities such as a seminar series, annual retreat and maintenance of a website. Aim 3: To oversee review and funding of pilot and feasibility projects. Aim 4: To facilitate data sharing and use of the cores from the greater community of dystrophy researchers so that the cores will provide a national resource for muscular dystrophy research.