REVIEW article
Front. Cell Dev. Biol.
Sec. Stem Cell Research
Volume 13 - 2025 | doi: 10.3389/fcell.2025.1640275
This article is part of the Research TopicStem Cell Therapy for Hereditary Neuromuscular DiseasesView all 3 articles
Stem/Progenitor Cell-Based Therapy for Duchenne Muscular Dystrophy
Provisionally accepted
- Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (Japan), Tokyo, Japan
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Duchenne muscular dystrophy is a genetic disease where loss of sarcolemma-associated protein, dystrophin, leads to progressive muscle wasting, and eventual loss of life from complications linked to cardiac deficits. Currently, numerous molecular therapies to restore dystrophin have entered clinical trials. However, the therapeutic benefits of these strategies in promoting tissue regeneration and reducing fibrosis remain limited. Stem/progenitor cell-based therapy in DMD patients is a promising strategy to promote muscle regeneration, though the conditions of transplantation and pre-treatments of numerous cell types are still being optimized. Several cell types with different properties and origins, such as myogenic stem cells, mesenchymal stem cells (MSCs), and induced pluripotent stem cells (iPSCs), have been studied for treating DMD.Myogenic stem cells derived from healthy donors are expected to restore the number of myofibers as well as dystrophin expression in DMD muscles. MSCs derived from various tissues, including umbilical cord, have immunosuppressive properties and are expected to ameliorate DMD phenotypes in combination with other gene therapies. In this review, we will summarize the challenges that must be overcome to allow for successful DMD muscle tissue regeneration and review the latest findings in stem cell-based DMD therapy. We will focus on the pre-conditioning of cells for replacement therapies and treatment of the disease niche to improve muscle fiber integration.
Keywords: Duchenne muscular dystrophy (DMD), Dystrophin, Stem cell therapies, skeletal muscle, cardiac muscle
Received: 03 Jun 2025; Accepted: 11 Aug 2025.
Copyright: © 2025 Tominari, Sathyaprakash and Aoki. This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) or licensor are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms.
* Correspondence: Yoshitsugu Aoki, Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry (Japan), Tokyo, Japan
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