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中文摘要
翻译
评价潜在疗法(药理学、基因或细胞疗法)的关键是 应用于小鼠模型的肌肉功能的灵敏和可重复的生理评估 营养不良因此,我们建议继续进行离体、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要, 在中心内,但也将作为一个国家资源,用于执行潜在的功能评估, 肌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。巴顿博士是肌肉生理学家, 在评估小鼠肌肉功能方面有丰富的经验。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,免费记录 轮跑和跑步机跑,以及样品在-8OC和液氮中的临时储存 冰箱我们建议将我们的措施扩展到包括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5年中提供了以前未得到满足的需求,并将 confinue是一个宝贵的资源,将使enfire肌营养不良症研究界, 利用这些检测方法来评估大量治疗方法的潜在益处, 不同形式的肌肉萎缩症。
英文摘要
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.
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The Chloroplast Expression System as a platform for orally bioavailable muscle therapeutics
  • 批准号:
    9904474
  • 项目类别:
  • 资助金额:
    $16.86万
  • 财政年份:
    2019
  • 负责人:
    Elisabeth R Barton
  • 依托单位:
The Sarcoglycan Complex in Skeletal Muscle Mechanotransduction
  • 批准号:
    9903225
  • 项目类别:
  • 资助金额:
    $33.0万
  • 财政年份:
    2016
  • 负责人:
    Elisabeth R Barton
  • 依托单位:
The Sarcoglycan Complex in Skeletal Muscle Mechanotransduction
  • 批准号:
    9247122
  • 项目类别:
  • 资助金额:
    $39.04万
  • 财政年份:
    2016
  • 负责人:
    Elisabeth R Barton
  • 依托单位:
Modulation of muscle regeneration by growth factors
  • 批准号:
    8122854
  • 项目类别:
  • 资助金额:
    $6.5万
  • 财政年份:
    2011
  • 负责人:
    Elisabeth R Barton
  • 依托单位:
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