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Therapeutic Approach of iPS Cell Technology for Treating Muscular Dystrophy

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Translational Research in Muscular Dystrophy

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Abstract

Most genetic disorders of skeletal muscle do not have curative treatment and are limited to palliative care that fails to curb progression. Additionally, for many myopathies, the pathology remains unclear, although the causative genes have been identified. Thus, research attempts are underway to develop new treatments for these intractable myopathies, including those based on induced pluripotent stem (iPS) cells. In general, iPS cell strategies can be divided into two groups: those focused on cell transplant therapies in the hope of finding a curative treatment and those seeking to elucidate the underlying pathology to develop effective drugs.

The transplantation of muscle progenitor cells derived from mouse iPS cells promotes muscle regeneration in a muscular dystrophy mouse model and restores muscle strength. Similarly, research using human iPS cells has led to several methods for inducing muscle engraftable progenitors. Though reports on pathology studies using patient-derived iPS cells are few, they have shown successful recapitulation of the disease. Here I explain how the induction of differentiation of human iPS cells into muscle cells with exceptional efficiency and high reproducibility will boost drug development and therapeutic efforts along with providing detailed understanding of myopathies.

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  1. Department of Clinical Application, Center for iPS Cell Research and Application (CiRA), Kyoto University, 53 Shogoin-Kawahara-cho, Sakyo-ku, Kyoto, 606-8507, Japan

    Hidetoshi Sakurai

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  1. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Shin'ichi Takeda

  2. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Yuko Miyagoe-Suzuki

  3. National Institute of Neuroscience, Kodaira, Tokyo, Tokyo, Japan

    Madoka Mori-Yoshimura

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Sakurai, H. (2016). Therapeutic Approach of iPS Cell Technology for Treating Muscular Dystrophy. In: Takeda, S., Miyagoe-Suzuki, Y., Mori-Yoshimura, M. (eds) Translational Research in Muscular Dystrophy. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55678-7_9

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