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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice

Published on: April 10, 2019

Transforming Duchenne muscular dystrophy therapy: The multifaceted role of extracellular vesicles and exosomes

Fatemeh Farzi¹, Fatemeh Soltanmohammadi^2,3, Seyed Abolghasem Mohammadi^4,5

¹Department of Medical Biotechnology, Faculty of Advanced Medical Sciences, Tabriz University of Medical Sciences, Tabriz, Iran.

Biochemistry and Biophysics Reports

|April 22, 2026

Abstract:

Duchenne muscular dystrophy (DMD) remains a devastating X chromosome-linked disorder with restricted curative options. Additionally, the existing treatment approaches, such as growth-modulating agents, anti-inflammatory drugs, antisense oligonucleotides with exon-skipping capabilities, stop codon mutation suppressors, vector-mediated gene therapy, CRISPR/Cas9 gene editing, and exogenous cell transplantation, can delay disease progression but are not curative. Extracellular vesicles (EVs), especially exosomes, nanoscale vesicles involved in intercellular communication, have emerged as promising therapeutic tools for DMD due to their low immunogenicity, ability to deliver therapeutic cargos, and potential to modulate inflammation, oxidative stress, and fibrosis. This review explores the transformative role of EVs (including exosomes) as multifunctional tools in DMD management. Natural EVs, enriched with regenerative microRNAs (miRNAs) and anti-fibrotic proteins, modulate inflammation, oxidative stress, and muscle degeneration. Besides, innovative engineering approaches could improve EVs' cargo loading and targeting, assisting efficient delivery of oligonucleotides and CRISPR/Cas9 editing components. Furthermore, we address the capacity of these vesicles to restore dystrophin expression and attenuate pathogenic mechanisms. Lastly, challenges associated with EV isolation, stability, and scalability are critically evaluated to support the development of an integrated cell and gene therapy framework with significant potential to improve clinical outcomes in DMD patients.

Insights

Extracellular vesicles (EVs) show promise for treating Duchenne muscular dystrophy (DMD) by delivering therapeutic molecules and modulating disease pathways. Further research into EV isolation and stability is needed for clinical application in DMD gene therapy.

Area of Science:

Biomedical Engineering
Regenerative Medicine
Molecular Biology

Background:

Duchenne muscular dystrophy (DMD) is a severe X-linked disorder with limited curative treatments.
Current therapies for DMD manage symptoms but do not offer a cure.
Extracellular vesicles (EVs), including exosomes, are emerging as potent therapeutic agents for DMD.

Purpose of the Study:

To review the therapeutic potential of extracellular vesicles (EVs) in managing Duchenne muscular dystrophy (DMD).
To explore how natural and engineered EVs can deliver therapeutic cargos and restore dystrophin expression.
To evaluate the challenges and future directions for EV-based therapies in DMD.

Main Methods:

Review of current literature on extracellular vesicles (EVs) and Duchenne muscular dystrophy (DMD).
Analysis of natural EV properties, including miRNA and protein cargo.
Evaluation of engineered EV strategies for enhanced drug delivery and gene editing components.

Main Results:

Natural EVs possess regenerative and anti-fibrotic properties, modulating inflammation and oxidative stress in DMD.
Engineered EVs can improve cargo loading and targeting for efficient delivery of oligonucleotides and CRISPR/Cas9 components.
EVs demonstrate potential to restore dystrophin expression and mitigate DMD pathogenesis.

Conclusions:

Extracellular vesicles (EVs) offer a multifunctional therapeutic approach for Duchenne muscular dystrophy (DMD).
EVs can be engineered for targeted delivery of gene therapies and regenerative molecules.
Addressing challenges in EV isolation, stability, and scalability is crucial for clinical translation in DMD treatment.

Read full analysis

Keywords:

Cell/gene therapy Duchenne muscular dystrophy (DMD)Exosomes Extracellular vesicles (EVs)

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