Cell therapy strategies in Duchenne muscular dystrophy: a systematic review of clinical applications
Cell Therapy Strategies on Duchenne Muscular Dystrophy: A Systematic Review of Clinical Applications 2023
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.
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.
What is Duchenne muscular dystrophy (DMD)?
Duchenne muscular dystrophy (DMD) is an inherited genetic disorder characterized by progressive degeneration of muscle tissue, leading to functional disability and early death.
Despite extensive research efforts, the discovery of a cure for DMD remains elusive, emphasizing the need to explore new therapeutic approaches. Cellular therapies have emerged as promising approaches to address the underlying pathophysiology of DMD.
This evaluation included an examination of limitations and potential advances in this particular area of research, along with an evaluation of the safety and efficacy of cell-based therapies in the context of DMD.
Available data suggest that diverse cell therapy approaches may provide a new, safe, and effective treatment modality for patients with DMD.

Book reference:
Akat A, Karaöz E. Cell therapy strategies on Duchenne muscular dystrophy: A systematic review of clinical applications. Stem Cell Reviews and Reports. 2023 Nov 13:1-21