» Articles » PMID: 37955832

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

Overview
Publisher Springer
Specialty Cell Biology
Date 2023 Nov 13
PMID 37955832
Authors
Affiliations
Soon will be listed here.
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.

Citing Articles

Safety and Tolerability of Wharton's Jelly-Derived Mesenchymal Stem Cells for Patients With Duchenne Muscular Dystrophy: A Phase 1 Clinical Study.

Lee J, Park S, Kim M, Kim H, Kwon J, Jeon H J Clin Neurol. 2025; 21(1):40-52.

PMID: 39778566 PMC: 11711273. DOI: 10.3988/jcn.2024.0299.


Cell transplantation-mediated dystrophin supplementation efficacy in Duchenne muscular dystrophy mouse motor function improvement demonstrated by enhanced skeletal muscle fatigue tolerance.

Bourgeois Yoshioka C, Takenaka-Ninagawa N, Goto M, Miki M, Watanabe D, Yamamoto M Stem Cell Res Ther. 2024; 15(1):313.

PMID: 39300595 PMC: 11414159. DOI: 10.1186/s13287-024-03922-x.

References
1.
Connuck D, Sleeper L, Colan S, Cox G, Towbin J, Lowe A . Characteristics and outcomes of cardiomyopathy in children with Duchenne or Becker muscular dystrophy: a comparative study from the Pediatric Cardiomyopathy Registry. Am Heart J. 2008; 155(6):998-1005. PMC: 2729548. DOI: 10.1016/j.ahj.2008.01.018. View

2.
Ferrier P, BAMATTER F, Klein D . MUSCULAR DYSTROPHY (DUCHENNE) IN A GIRL WITH TURNER'S SYNDROME. J Med Genet. 1965; 2(1):38-46. PMC: 1012803. DOI: 10.1136/jmg.2.1.38. View

3.
Chelly J, Marlhens F, Le Marec B, Jeanpierre M, Lambert M, Hamard G . De novo DNA microdeletion in a girl with Turner syndrome and Duchenne muscular dystrophy. Hum Genet. 1986; 74(2):193-6. DOI: 10.1007/BF00282093. View

4.
Satre V, Monnier N, Devillard F, Amblard F, Lunardi J . Prenatal diagnosis of DMD in a female foetus affected by Turner syndrome. Prenat Diagn. 2004; 24(11):913-7. DOI: 10.1002/pd.1031. View

5.
Birnkrant D, Bushby K, Bann C, Apkon S, Blackwell A, Brumbaugh D . Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management. Lancet Neurol. 2018; 17(3):251-267. PMC: 5869704. DOI: 10.1016/S1474-4422(18)30024-3. View