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Purification and Transplantation of Myogenic Progenitor Cell Derived Exosomes to Improve Cardiac Function in Duchenne Muscular Dystrophic Mice
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Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression

Laura Yedigaryan1, Maurilio Sampaolesi1,2

  • 1Translational Cardiomyology Laboratory, Stem Cell and Developmental Biology, Department of Development and Regeneration, KU Leuven, Leuven, Belgium.

Frontiers in Physiology
|March 9, 2023
PubMed

Insights

Extracellular vesicles (EVs) may play a role in Duchenne muscular dystrophy (DMD) progression. This review explores their contribution to DMD pathology, potential as biomarkers, and therapeutic applications.

Area of Science:

  • Biochemistry
  • Molecular Biology
  • Genetics

Background:

  • Duchenne muscular dystrophy (DMD) is a severe inherited muscle-wasting disorder caused by dystrophin gene mutations.
  • Despite extensive research, key aspects of DMD pathogenesis and progression remain underexplored, hindering therapeutic development.

Purpose of the Study:

  • To review the potential role of extracellular vesicles (EVs) in DMD pathology.
  • To discuss the utility of EV cargo as biomarkers for DMD-related conditions.
  • To explore therapeutic strategies involving EV modulation and engineered cargo delivery.

Main Methods:

  • Literature review of studies investigating extracellular vesicles in muscular dystrophy.
  • Analysis of EV cargo, including microRNAs, in relation to DMD hallmarks.
  • Evaluation of therapeutic approaches targeting EV function.

Main Results:

  • Extracellular vesicles (EVs) are implicated in contributing to DMD pathology through their secreted cargo.
  • EV-derived microRNAs show promise as biomarkers for diagnosing and monitoring DMD-associated fibrosis, inflammation, and cardiac complications.
  • EVs offer potential as vehicles for targeted therapeutic cargo delivery.

Conclusions:

  • Extracellular vesicles represent a significant factor in Duchenne muscular dystrophy pathogenesis.
  • EVs hold potential as diagnostic biomarkers and therapeutic delivery systems for DMD.
  • Further research into EV biology is crucial for advancing DMD treatment strategies.
Keywords:
cardiomyopathyexosomesextracellular vesicles (EVs)induced pluripotent stem cells (iPSCs)miRNAsmuscle regenerationmuscular dystrophiessatellite cells

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