Core C - Resource Core

摘要 评价潜在治疗（药理学、基因或细胞疗法）的关键是敏感性和 应用于营养不良小鼠模型的肌肉功能的可重复生理学评估。因此，我们认为， 我们建议继续进行离体、原位和整个动物评估的研究核心 肌肉的完整性和功能。这个核心不仅将支持中心内项目的需求， 还将作为国家资源，对潜在的治疗方法进行功能评估， 肌肉萎缩症生理评估核心资源位于实验室空间 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 ℃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 隔膜的离体评估。 我们认为，这一核心在其存在的过去15年中提供了以前未得到满足的需求，并将 继续是一个宝贵的资源，将使整个肌肉萎缩症研究界， 利用这些检测来评估大量治疗方法的潜在益处， 不同形式的肌肉萎缩症。

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 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.