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Current Trends in Duchenne Muscular Dystrophy Research and Therapy: 3D Cardiac Modelling.
Marta Przymuszała1,2, Marta Białobrzeska1, Józef Dulak1
1Department of Medical Biotechnology, Faculty of Biochemistry, Biophysics and Biotechnology, Jagiellonian University, Kraków, Poland.
Journal of Cachexia, Sarcopenia and Muscle
|January 7, 2026
Summary
Duchenne muscular dystrophy (DMD) treatments face challenges in cardiac targeting and long-term efficacy. Genetic approaches combined with improved cardiac delivery strategies show promise for future DMD therapies.
Area of Science:
- Biomedical Science
- Cardiovascular Research
- Regenerative Medicine
Background:
- Duchenne muscular dystrophy (DMD) causes progressive muscle degeneration and life-threatening cardiac issues due to dystrophin deficiency.
- Current treatments for DMD have limited clinical evidence for efficacy, especially in cardiac targeting and long-term safety.
- Human-induced pluripotent stem cells (hiPSCs) offer a platform for modeling DMD and testing therapies, including patient-specific cardiomyocytes.
Purpose of the Study:
- To review the current landscape of Duchenne muscular dystrophy (DMD) treatments, focusing on challenges and emerging strategies.
- To evaluate the potential of genetic therapies and stem cell-based approaches for DMD, particularly concerning cardiac involvement.
- To highlight the need for optimized cardiac delivery and combination therapies for effective DMD management.
Main Methods:
- Review of current FDA-approved therapies and ongoing research in dystrophin restoration (RNA-based, gene therapy).
- Analysis of human-induced pluripotent stem cell (hiPSC) applications in DMD modeling (in vitro, 3D cardiac models).
- Evaluation of cell-based therapy potential (2D cell sheets, patches, 3D models) and their limitations in DMD.
Main Results:
- Limited clinical evidence exists for current dystrophin-restoring therapies, with significant challenges in cardiac targeting, safety, and scalability.
- hiPSC-derived cardiomyocytes and 3D cardiac models provide valuable in vitro tools for understanding DMD pathophysiology and testing therapeutic interventions.
- Cell-based therapies show potential but face hurdles in engraftment, maturation, and long-term function for DMD applications.
Conclusions:
- Current DMD treatment focuses on genetic approaches, emphasizing the combination of dystrophin restoration with targeted cardiac therapies.
- Optimized cardiac-specific delivery strategies are crucial for improving the efficacy of both genetic and cell-based DMD treatments.
- Further research is needed to overcome challenges in cell integration, maturation, and long-term function for regenerative therapies in DMD.
