Failed Regeneration in the Muscular Dystrophies: Inflammation, Fibrosis and Fat - Administrative Supplement

摘要 本行政补充的目的是保持UF韦尔斯顿中心的三个核心， 它的一年无费用延长，以服务于行政，培训和资源职能， 中心，以及国家肌肉萎缩症社区。行政核心提供整合 管理中心的活动。它促进了 在国家和地方一级的研究和患者/倡导社区。行政核心 维护我们的网站，以传达中心的使命和培训机会的可用性， 通过我们的共享科学资源核心提供服务。培训核心的总体目标是 增加受过严格训练和有科学能力的研究人员的数量， 肌肉萎缩症的生产性基础科学、转化和/或临床研究项目。满足这一 中心目标，核心将确定和招募合格的基础科学和临床博士前， 博士后候选人谁表现出真正的兴趣，和潜力，独立的基础科学， 用于肌营养不良症的转化和/或临床研究。将特别强调 从在种族、族裔、性别、社会经济和文化方面代表性不足的群体中招聘受训人员 地位和残疾。对潜在治疗方法（药理学、基因或细胞）的评价至关重要 治疗）是应用于小鼠的肌肉功能的灵敏的和可重复的生理评估。 营养不良的模型因此，我们建议继续我们的共享科学资源核心， 进行肌肉完整性和功能的离体、原位和整体动物评估。这个核心不会 只支持中心内项目的需要，在没有成本的扩展，但也将作为一个 对肌营养不良症的潜在疗法进行功能评估的国家资源。的 生理评估核心/共享科学资源核心位于实验室空间 指定给核心主管巴顿博士巴顿博士是一位肌肉生理学家， 评估小鼠的肌肉功能。这包括用于肌肉生理测量的所有仪器， 专门的设备，用于后肢悬吊实验、记录的自由轮跑步和跑步机 运行，以及样品在-80 ° C和液氮冷冻机中的临时储存。我们建议 与邻近的同事合作，评估整个动物的呼吸功能，这将补充前 隔膜的体内评估。我们认为，这一核心已满足了过去未得到满足的需求 15年的存在，并将继续成为一个宝贵的资源，将使整个肌肉 营养不良研究社区利用这些测定来评估大量的潜在益处， 不同形式的肌营养不良症的治疗方法。

Abstract The purpose of this Administrative Supplement is to maintain the three cores of the UF Wellston Center during its one-year no cost extension in order to serve the administrative, training, and resource functions of the Center, as well as the national muscular dystrophy community. The Administrative Core provides integration and management of activities for the Center. It promotes interactions and communications between the research and patient/advocacy communities on both a national and local level. The Administrative Core maintains our website to communicate the Center mission and the availability of training opportunities and services available through our Shared Scientific Resource Core. The overall objective of the Training Core is to increase the number of rigorously trained and scientifically competent researchers capable of sustaining productive basic science, translational and/or clinical research programs in muscular dystrophy. To meet this central objective, the core will identify and recruit qualified basic science and clinical predoctoral and postdoctoral candidates who demonstrate a genuine interest in, and potential for, independent basic science, translational and/or clinical research in muscular dystrophy. Particular emphasis will be placed on the recruitment of trainees from underrepresented groups with respect to race, ethnicity, gender, socio-economic status and disabilities. Critical to the evaluation of potential therapeutics (pharmacological, gene, or cell therapies) are sensitive and repeatable physiological assessments of muscle function applied to mouse models of dystrophy. Therefore, we are proposing to continue our Shared Scientific Resource Core that performs ex vivo, in situ, and whole animal assessments of muscle integrity and function. This core will not only support the needs of the projects within the center during the no cost extension, but will also serve as a national resource for performing functional evaluation of potential therapies for the muscular dystrophies. The Physiological Assessment Core / Shared Scientific Resource Core is housed in the laboratory space designated to Dr. Barton, the Core Director. Dr. Barton is a muscle physiologist with extensive experience evaluating muscle function in mice. This includes all instrumentation for muscle physiological measurements, specialized equipment for hindlimb suspension experiments, documented free wheel running, and treadmill running, as well as interim storage of samples in both –80ºC and liquid nitrogen freezers. We propose to collaborate with neighboring colleagues to assess whole animal respiratory function, which will complement ex vivo assessment of the diaphragm. We believe that this Core has provided a previously unmet need in the past 15 years of its existence, and will continue to be a valuable resource that will enable the entire muscular dystrophy research community to utilize these assays to assess the potential benefits of a large number of approaches to the treatment of different forms of muscular dystrophy.