Development of the next-generation medical treatment for muscularDystrophy

疾病。虽然基因疗法部分处于试验阶段，但标准化疗法仍在研究中。最近，人类iPS细胞的制备程序已经建立。通过使用这一策略，各种新的治疗方法的新研究变得可行。例如，研究促进致病区域外显子跳跃的小分子，或恢复本质上脆弱的卫星细胞存活的小分子。最近，除了在小鼠iPS细胞中，我们还在人类iPS细胞中开发了成熟肌肉细胞和未成熟肌肉细胞（或卫星细胞作为干细胞）的分化过程（Awaya T， Heike T等）。《科学通报》，2012)。同时，我们正在制造肌肉萎缩症特异性iPS细胞。

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.