Gregorevic Paul, Blankinship Michael J, Chamberlain Jeffrey S
The University of Washington, Department of Neurology, Senator Paul D Wellstone Muscular Dystrophy Cooperative Research Center, Seattle, WA 98195, USA.
Curr Opin Mol Ther. 2004 Oct;6(5):491-8.
Recent progress has generated exciting results that are increasing the prospects for gene therapy of a variety of disorders of striated muscle, including the muscular dystrophies and myopathies, acquired and inherited diseases of cardiac muscle, and aging-associated muscle wasting. Numerous viral vector systems are being employed to transfer therapeutic genes to striated muscles, and advances in vector technology are leading to improved gene transfer efficiencies coupled with reduced immunological responses. The current technology in the field of viral vectors as tools for gene delivery to striated muscle is summarized, and recent developments related to gene therapies for skeletal and cardiac muscle disorders are discussed.
最近的进展已产生了令人振奋的成果,这些成果正在增加对多种横纹肌疾病进行基因治疗的前景,包括肌营养不良症和肌病、获得性和遗传性心肌疾病以及与衰老相关的肌肉萎缩。目前正在使用多种病毒载体系统将治疗性基因转移至横纹肌,并且载体技术的进步正带来更高的基因转移效率以及更低的免疫反应。本文总结了作为向横纹肌递送基因工具的病毒载体领域的当前技术,并讨论了与骨骼肌和心肌疾病基因治疗相关的最新进展。