GENE TRANSFER TO SKELETAL MUSCLE FIBERS AND ENDOTHELIUM

描述：（改编自申请人摘要）本申请是 继续提供资金，以开发骨骼肌中最佳的转导效率， 肌毛细血管内皮和肌纤维在血管隔离过程中的作用 后肢，并确定这些方法的有效性，以实现 适当的重组蛋白（VEGF，eNOS）的治疗浓度 和肌营养不良蛋白），并最终确定这是否 可以使用基于导管的技术来实现该方法。 在 从该组的初步数据来看，高转导效率已经被证明是有效的。 没有肌肉损伤或坏死的证据。 这些实验 旨在优化方法，而不是获得 治疗效果。

DESCRIPTION: (Adapted from the applicant's abstract) This application is for continued funding to develop optimal transduction efficiency in skeletal muscle capillary endothelium and muscle fibers during vascular isolation of the hindlimb, and to determine the efficacy of these approaches to achieve therapeutic concentrations of appropriate recombinant proteins (VEGF, eNOS and dystrophin) in animal models, and finally to determine whether this approach can be accomplished using a catheter-based technique. In preliminary data from this group, high transduction efficiencies have been obtained without evidence of muscle injury or necrosis. These experiments are directed at optimizing the methods rather than at obtaining a therapeutic result.