MONITORING DISEASE AND THERAPY IN DYSTROPHIN-DEFICIENT MUSCLE USING ULTRASOUND

这项持续工作的长期目标是通过无损成像方式定量表征骨骼肌的结构和功能，并实施可靠的疾病诊断和改善临床管理方法。特别是，我们寻求基于超声与软组织相互作用的独特信息，开发骨骼肌病理生理学的新见解。到目前为止，我们主要关注心肌梗死和特发性心肌病的心脏重塑，以及生理和病理血管结构和功能。我们现在建议扩展从这些努力中获得的知识，以便在动物模型中定量地“表型”骨骼肌遗传疾病中由于肌营养不良蛋白缺乏而导致的重塑过程。最终目标是为肌萎缩症的定量超声组织表征提供一个全面的软件包，这是通过强大的独立措施（例如，强度测试）充分验证的。

The long term goal of this continuing work is to quantitatively characterize skeletal muscle structure and function noninvasively with nondestructive imaging modalities, and to implement robust methods for diagnosing disease and improving clinical management. In particular, we seek to develop novel insights into skeletal muscle pathophysiology based on unique information available from the interaction of ultrasound with soft tissues. Heretofore we have focused on cardiac remodeling in myocardial infarction and idiopathic cardiomyopathy, and on physiologic and pathologic vascular structure and function. We now propose to extend the knowledge gained from these efforts in order to quantitatively “phenotype” the remodeling process in genetic disorders of skeletal muscle as a consequence of dystrophin deficiency in animal models. The ultimate goal is to produce a comprehensive package for quantitative ultrasound tissue characterization of muscular dystrophy, which is fully validated by robust independent measures (e.g., strength testing)