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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
Published on: April 10, 2019
Molecular, cellular, and pharmacological therapies for Duchenne/Becker muscular dystrophies
Joe V Chakkalakal1, Jennifer Thompson, Robin J Parks
1Department of Cellular and Molecular Medicine and Centre for Neuromuscular Disease, Faculty of Medicine, University of Ottawa, Ontario, Canada.
Abstract:
Although the molecular defect causing Duchenne/Becker muscular dystrophy (DMD/BMD) was identified nearly 20 years ago, the development of effective therapeutic strategies has nonetheless remained a daunting challenge. Over the years, a variety of different approaches have been explored in an effort to compensate for the lack of the DMD gene product called dystrophin. This review not only presents some of the most promising molecular, cellular, and pharmacological strategies but also highlights some issues that need to be addressed before considering their implementation. Specifically, we describe current strategies being developed to exogenously deliver healthy copies of the dystrophin gene to dystrophic muscles. We present the findings of several studies that have focused on repairing the mutant dystrophin gene using various approaches. We include a discussion of cell-based therapies that capitalize on the use of myoblast or stem cell transfer. Finally, we summarize the results of several studies that may eventually lead to the development of appropriate drug-based therapies. In this context, we review our current knowledge of the mechanisms regulating expression of utrophin, the autosomal homologue of dystrophin. Given the complexity associated with the dystrophic phenotype, it appears likely that a combinatorial approach involving different therapeutic strategies will be necessary for the appropriate management and eventual treatment of this devastating neuromuscular disease.
Insights
Developing effective treatments for Duchenne/Becker muscular dystrophy (DMD/BMD) remains challenging. This review explores promising gene, cell, and drug therapies, suggesting a combination approach is key for managing this neuromuscular disease.
Area of Science:
- Neurology
- Genetics
- Biomedical Engineering
Background:
- Duchenne/Becker muscular dystrophy (DMD/BMD) is caused by defects in the dystrophin gene, identified nearly 20 years ago.
- Despite the genetic cause being known, effective therapeutic strategies for DMD/BMD are still under development.
- Current research focuses on compensating for the lack of dystrophin, the key protein missing in DMD/BMD patients.
Purpose of the Study:
- To review promising therapeutic strategies for Duchenne/Becker muscular dystrophy.
- To highlight challenges and necessary considerations for implementing these therapies.
- To discuss the potential of combined therapeutic approaches for DMD/BMD management.
Main Methods:
- Review of current molecular, cellular, and pharmacological strategies for DMD/BMD.
- Analysis of studies on exogenous gene delivery of the dystrophin gene.
- Examination of gene repair approaches for the mutant dystrophin gene.
- Discussion of cell-based therapies, including myoblast and stem cell transfer.
- Summary of research on drug-based therapies and utrophin expression regulation.
Main Results:
- Several promising strategies are being developed, including gene therapy, gene repair, cell transplantation, and drug development.
- Exogenous delivery of healthy dystrophin genes and repair of mutant genes are active areas of research.
- Cell-based therapies using myoblasts or stem cells show potential.
- Drug-based therapies targeting utrophin expression are also being investigated.
Conclusions:
- A combinatorial approach integrating multiple therapeutic strategies is likely necessary for effective management and treatment of DMD/BMD.
- Addressing the complexity of the dystrophic phenotype requires multifaceted therapeutic interventions.
- Further research and development are crucial for translating these strategies into clinical practice for DMD/BMD.
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