Development of the next-generation medical treatment for muscularDystrophy

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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.