Physiological Assessment

在小鼠模型中评价潜在治疗（药理学、基因或细胞疗法）的关键 肌营养不良症是敏感的和可重复的肌肉功能的生理评估。 因此，我们建议创建一个研究核心， 评估肌肉完整性和功能。本补助金中提出的三个项目（项目1 - 3） 包含需要肌肉生理测量的组件。（项目4依赖于 这些试验用于临床前论证。）生理评估核心将满足 项目在此赠款，以及肌肉萎缩症研究界的需求。此外，本发明还提供了一种方法， 核心将维持DMD、LGMD和强直性肌营养不良小鼠模型的室内群体， 以支持需要长期测试的研究。该核心的资源包含在博士。 斯威尼的实验室，其中包括所有的肌肉生理测量仪器，以及 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.巴顿博士是一位肌肉生理学家，在评估小鼠肌肉功能方面拥有丰富的经验。 莫妮卡·扎德尔是进行肌肉力学实验的专家，而佩德罗·阿科斯塔则坚持认为， 繁殖群体，并将监测后肢悬挂和自由轮运行实验。 我们相信，这一核心将提供一个宝贵的资源，将使整个肌肉萎缩症 研究界利用这些测定来评估大量方法的潜在益处 治疗不同形式的肌肉萎缩症

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