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Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies

1
Department of Geriatric Medicine, Tokyo Metropolitan Institute of Gerontology, Tokyo 173-0015, Japan
2
Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, Tokyo 187-8502, Japan
3
Department of Child Neurology, National Center Hospital for Neurology and Psychiatry, National Center of Neurology and Psychiatry, Tokyo 187-8502, Japan
4
Department of Paediatrics, University of Oxford, South Parks Road, Oxford OX1 3QX, UK
*
Author to whom correspondence should be addressed.
Cells 2019, 8(9), 1066; https://doi.org/10.3390/cells8091066
Received: 29 July 2019 / Revised: 6 September 2019 / Accepted: 10 September 2019 / Published: 11 September 2019
Muscular dystrophies (MDs) are a group of heterogeneous genetic disorders caused by mutations in the genes encoding the structural components of myofibres. The current state-of-the-art treatment is oligonucleotide-based gene therapy that restores disease-related protein. However, this therapeutic approach has limited efficacy and is unlikely to be curative. While the number of studies focused on cell transplantation therapy has increased in the recent years, this approach remains challenging due to multiple issues related to the efficacy of engrafted cells, source of myogenic cells, and systemic injections. Technical innovation has contributed to overcoming cell source challenges, and in recent studies, a combination of muscle resident stem cells and gene editing has shown promise as a novel approach. Furthermore, improvement of the muscular environment both in cultured donor cells and in recipient MD muscles may potentially facilitate cell engraftment. Artificial skeletal muscle generated by myogenic cells and muscle resident cells is an alternate approach that may enable the replacement of damaged tissues. Here, we review the current status of myogenic stem cell transplantation therapy, describe recent advances, and discuss the remaining obstacles that exist in the search for a cure for MD patients. View Full-Text
Keywords: skeletal muscle; stem cells; regeneration; dystrophy; therapy skeletal muscle; stem cells; regeneration; dystrophy; therapy
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Motohashi, N.; Shimizu-Motohashi, Y.; Roberts, T.C.; Aoki, Y. Potential Therapies Using Myogenic Stem Cells Combined with Bio-Engineering Approaches for Treatment of Muscular Dystrophies. Cells 2019, 8, 1066.

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