Development of the next-generation medical treatment for muscularDystrophy

疾病虽然基因治疗部分处于试验阶段，但标准化治疗仍在研究中。最近，已经建立了人iPS细胞的制备程序。通过使用这种策略，各种新的治疗方法的新研究变得可行。例如，促进疾病责任区外显子跳跃或恢复内在脆弱卫星细胞存活的小分子的研究。最近，除了小鼠iPS细胞外，我们还开发了人iPS细胞中的成熟肌细胞沿着未成熟肌细胞（或卫星细胞作为干细胞）的分化程序（Awaya T，Heike T et al.PloS One 7：e51638 2012）。与此同时，我们正在制造肌营养不良症特异性iPS细胞的储备。

diseases. Although gene therapy is partly in trial, the standardized therapies are still under investigation. Recently, the preparation procedures for human iPS cells have been set up. By using this strategy, various new studies for new treatments become practicable. For example, the research of small molecules which facilitate exon skipping in disease-responsible region, or which restore the survival of intringically-fragile satellite cells. Recently, we have developed the differentiation procedures intomature muscle cells along with immature muscle cells (or satellite cells as stem cells) in human iPS cells, in addition to in mouse iPS cells (Awaya T, Heike T et al. PloS One 7:e51638 2012）. Simultaneously, we are on the way to make stock of muscular dystrophy-specific iPS cells.