Core C: PHYSIOLOGICAL ASSESSMENT CORE

摘要 对潜在疗法(药物疗法、基因疗法或细胞疗法)的评估至关重要 并将肌肉功能的可重复生理评估应用于营养不良的小鼠模型。 因此，我们建议继续进行体外、原位和整个动物的研究核心 肌肉完整性和功能的评估。这个核心不仅将支持以下项目的需求 该中心，但也将作为执行潜力功能评估的国家资源 肌肉营养不良症的治疗方法。生理评估核心资源位于 指定给核心主管巴顿博士的实验室空间。巴顿博士是一位肌肉生理学家 评估小鼠肌肉功能的丰富经验。这包括用于肌肉的所有器械 生理测量，后肢悬吊实验专用设备，免费文件 车轮运行和跑步机运行，以及在零下80C和液氮中临时存储样品 冰柜。我们建议与邻近的同事合作评估整个动物的呼吸道 功能，这将补充体外评估的横隔膜。 我们认为，这一核心在其存在的过去10年中提供了以前没有得到满足的需求，并且 将继续是一个宝贵的资源，将使整个肌肉营养不良研究界 利用这些检测来评估大量治疗糖尿病的方法的潜在益处。 不同形式的肌肉营养不良。

ABSTRACT 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 a research 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, but will also serve as a national resource for performing functional evaluation of potential therapies for the muscular dystrophies. The Physiological Assessment Core resources are 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 –80C 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 10 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.