Shin Jin-Hong, Takeda Shin'ichi
Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-higashi-machi, Kodaira, Tokyo, Japan.
Brain Nerve. 2007 Apr;59(4):415-24.
A successful cure for muscular dystrophies has yet to be demonstrated, but we could notice spectacular progress for the past few years. Advances have been made in methods of delivering foreign genes into muscle cells, either as naked plasmid DNA or via viral vectors. Among the virus-based methods, results with adeno-associated viral vectors have been the most promising. Systemic administration of anti-sense oligonucleotides has shown incredible success in inducing dystrophin expression and ameliorating muscle pathology via exon skipping in canine models of muscular dystrophy. Biochemical and functional rescue of dystrophic muscle has also been demonstrated with the transplantation of normal or genetically modified myogenic stem cells, as well as with a number of pharmacological approaches in animal models. Here we review the current researches on the treatment of muscular dystrophies and clinical trials on Duchenne muscular dystrophy.
目前尚未证明能成功治愈肌肉萎缩症，但在过去几年里我们可以看到显著进展。将外源基因导入肌肉细胞的方法取得了进展，这些方法包括使用裸质粒DNA或通过病毒载体。在基于病毒的方法中，腺相关病毒载体的效果最有前景。在犬类肌肉萎缩症模型中，全身给予反义寡核苷酸通过外显子跳跃诱导抗肌萎缩蛋白表达并改善肌肉病理状况，已取得了惊人的成功。正常或基因改造的成肌干细胞移植以及动物模型中的一些药理学方法也证明了对营养不良肌肉的生化和功能挽救作用。在此，我们综述了目前关于肌肉萎缩症治疗的研究以及杜氏肌营养不良症的临床试验。
