Physiological Assessment Core

评估潜在疗法(药物疗法、基因疗法或细胞疗法)的关键是 灵敏和可重复的肌肉功能生理学评估应用于小鼠模型 营养不良。因此，我们建议继续进行体外、原位和整体的研究核心 肌肉完整性和功能的动物评估。这一核心不仅将支持项目的需求 在该中心内，但也将作为执行潜力的功能评估的导航资源 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，跑步机运行，以及在零下8摄氏度和液氮中临时存储样品 冰柜。我们建议将我们的措施扩展到包括对整个动物呼吸功能的评估， 这将补充对横隔膜的体外评估。 我们认为，这一核心在其存在的过去5年中提供了以前没有得到满足的需求，并将 限制成为一个有价值的资源，使Enfire肌营养不良症研究社区能够 利用这些化验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。

Critical to the evaluation of potential therapeufics (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 funcfion. This core will not only support the needs of the projects within the center, but will also serve as a nafional resource for performing funcfional evaluation of potenfial 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 -8OC and liquid nitrogen freezers. We propose to extend our measures to include evaluation of whole animal respiratory funcfion, which will complement ex vivo assessment of the diaphragm. We believe that this Core has provided a previously unmet need in the past 5 years of its existence, and will confinue to be a valuable resource that will enable the enfire muscular dystrophy research community to ufilize these assays to assess the potential benefits of a large number of approaches to the treatment of different forms of muscular dystrophy.