Physiological Assessment

对评估小鼠模型的潜在疗法(药理学、基因或细胞疗法)至关重要 肌营养不良是对肌肉功能的敏感和可重复的生理学评估。 因此，我们建议建立一个研究核心，在体外、原位和整个动物中进行 肌肉完整性和功能的评估。本拨款建议的三个项目(项目1-3) 含有需要肌肉生理测量的成分。(项目4依赖于 这些测试用于临床前的合理性。)生理评估核心将满足 这笔赠款内的项目，以及肌营养不良症研究界的需求。此外, 核心将按顺序维护DMD、LGMD和强直性肌营养不良模型的小鼠群体 以支持需要长期测试的研究。这一核心的资源包含在Dr。 斯威尼的实验室，包括所有用于肌肉生理测量的仪器，以及 用于后肢悬挂实验的专用设备、有记录的自由轮运行和跑步机 跑步。巴顿博士是一位肌肉生理学家，在评估小鼠的肌肉功能方面有着丰富的经验。 莫妮卡·扎德尔是进行肌肉力学实验的专家，而佩德罗·阿科斯塔则坚持我们的 繁殖群体，并将监测后肢悬挂和自由轮运行实验。 我们相信，这个核心将提供一个宝贵的资源，将使整个肌肉营养不良 研究界利用这些分析来评估大量方法的潜在好处 不同形式的肌营养不良症的治疗

Critical to the evaluation of potential therapeutics (pharmacological, gene or cell therapies) in mouse models of muscular dystrophy are sensitive and repeatable physiological assessments of muscle function. Therefore, we are proposing to create a research core which performs ex vivo, in situ, and whole animal assessments of muscle integrity and function. Three of the projects (Projects 1-3) proposed in this grant contain components that require muscle physiological measurements. (Project 4 relies on the outcome of these tests for pre-clinical justification.) The Physiological Assessment Core will meet the needs of the projects within this grant, as well as the needs of the muscular dystrophy research community. In addition, the core will maintain in house colonies of mouse models of DMD, LGMD, and myotonic dystrophy in order to support studies where long-term testing is necessary. The resources of this core are contained within Dr. Sweeney's laboratory, which includes all instrumentation for muscle physiological measurements, as well as specialized equipment for hindlimb suspension experiments, documented free wheel running, and treadmill running. Dr. Barton is a muscle physiologist with extensive experience evaluating muscle function in mice. Monica Zadel is an expert at performing muscle mechanical experiments, while Pedro Acosta maintains our breeding colonies and will monitor hindlimb suspension and free wheel running experiments. We believe that this Core will provide 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