Cell therapeutic approaches using multipotent mesenchymal stromal cells for muscular dystrophy

  • Nitahara-Kasahara Yuko
    Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan
  • Takeda Shin'ichi
    Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan
  • Okada Takashi
    Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo, Japan Department of Biochemistry and Molecular Biology, Division of Gene Therapy Research, Center for Advanced Medical Technology, Nippon Medical School, Tokyo, Japan

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Abstract

Multipotent mesenchymal stromal cells (MSCs) have potential therapeutic uses owing to their ability to differentiate in situ into various cell types with immunosuppressive properties. Clinically, MSCs have been used to treat inflammatory diseases, such as steroid-resistant graft-versus-host disease. We previously reported a strategy to expand MSC cultures and to induce these cells to undergo myogenic differentiation, which is promising for the treatment of muscular diseases. Muscular dystrophy is an incurable genetic disease with early mortality and causes skeletal muscle weakness with chronic inflammation. Here, we focused on the beneficial properties of MSCs, namely, they can undergo mesoderm differentiation, have the ability to fuse with dystrophic muscles, and have anti-inflammatory activities. In this review, we highlight and discuss MSC-based therapeutic approaches for muscular dystrophy.

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