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Cell Therapy Strategies on Duchenne Muscular Dystrophy: A Systematic Review of Clinical Applications

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Abstract

Duchenne Muscular Dystrophy (DMD) is an inherited genetic disorder characterized by progressive degeneration of muscle tissue, leading to functional disability and premature death. Despite extensive research efforts, the discovery of a cure for DMD continues to be elusive, emphasizing the need to investigate novel treatment approaches. Cellular therapies have emerged as prospective approaches to address the underlying pathophysiology of DMD. This review provides an examination of the present situation regarding cell-based therapies, including CD133 + cells, muscle precursor cells, mesoangioblasts, bone marrow-derived mononuclear cells, mesenchymal stem cells, cardiosphere-derived cells, and dystrophin-expressing chimeric cells. A total of 12 studies were found eligible to be included as they were completed cell therapy clinical trials, clinical applications, or case reports with quantitative results. The evaluation encompassed an examination of limitations and potential advancements in this particular area of research, along with an assessment of the safety and effectiveness of cell-based therapies in the context of DMD. In general, the available data indicates that diverse cell therapy approaches may present a new, safe, and efficacious treatment modality for patients diagnosed with DMD. However, further studies are required to comprehensively understand the most advantageous treatment approach and therapeutic capacity.

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The datasets used and/or analyzed during the current study are available from the corresponding author on reasonable request.

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  1. Life Park Hospital, Cellular and Biological Products Manufacturing Center, Ragıp Kenan Sok. No:8, Ortakoy, 99010, Nicosia (Lefkosa), Cyprus

    Ayberk Akat

  2. Liv Hospital Ulus, Regenerative Medicine and Stem Cell Center, Istanbul, Turkey

    Erdal Karaöz

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AA: Conceived and designed the analysis, collected data, analyzed results, drafted the article. EK: Study conception and design, revised the article critically for important intellectual content.

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Akat, A., Karaöz, E. Cell Therapy Strategies on Duchenne Muscular Dystrophy: A Systematic Review of Clinical Applications. Stem Cell Rev and Rep 20, 138–158 (2024). https://doi.org/10.1007/s12015-023-10653-8

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