Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression

Duchenne muscular dystrophy (DMD) is a devastating disorder and is considered to be one of the worst forms of inherited muscular dystrophies. DMD occurs as a result of mutations in the dystrophin gene, leading to progressive muscle fiber degradation and weakness. Although DMD pathology has been stud...

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Main Authors: Laura Yedigaryan, Maurilio Sampaolesi
Format: Article
Language:English
Published: Frontiers Media S.A. 2023-02-01
Series:Frontiers in Physiology
Subjects:
Online Access:https://www.frontiersin.org/articles/10.3389/fphys.2023.1130063/full
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author Laura Yedigaryan
Maurilio Sampaolesi
Maurilio Sampaolesi
author_facet Laura Yedigaryan
Maurilio Sampaolesi
Maurilio Sampaolesi
author_sort Laura Yedigaryan
collection DOAJ
description Duchenne muscular dystrophy (DMD) is a devastating disorder and is considered to be one of the worst forms of inherited muscular dystrophies. DMD occurs as a result of mutations in the dystrophin gene, leading to progressive muscle fiber degradation and weakness. Although DMD pathology has been studied for many years, there are aspects of disease pathogenesis and progression that have not been thoroughly explored yet. The underlying issue with this is that the development of further effective therapies becomes stalled. It is becoming more evident that extracellular vesicles (EVs) may contribute to DMD pathology. EVs are vesicles secreted by cells that exert a multitude of effects via their lipid, protein, and RNA cargo. EV cargo (especially microRNAs) is also said to be a good biomarker for identifying the status of specific pathological processes that occur in dystrophic muscle, such as fibrosis, degeneration, inflammation, adipogenic degeneration, and dilated cardiomyopathy. On the other hand, EVs are becoming more prominent vehicles for custom-engineered cargos. In this review, we will discuss the possible contribution of EVs to DMD pathology, their potential use as biomarkers, and the therapeutic efficacy of both, EV secretion inhibition and custom-engineered cargo delivery.
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spelling doaj.art-2ee723a6917f4483b4f700996a4eb61c2023-02-20T13:58:44ZengFrontiers Media S.A.Frontiers in Physiology1664-042X2023-02-011410.3389/fphys.2023.11300631130063Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progressionLaura Yedigaryan0Maurilio Sampaolesi1Maurilio Sampaolesi2Translational Cardiomyology Laboratory, Stem Cell and Developmental Biology, Department of Development and Regeneration, KU Leuven, Leuven, BelgiumTranslational Cardiomyology Laboratory, Stem Cell and Developmental Biology, Department of Development and Regeneration, KU Leuven, Leuven, BelgiumHistology and Medical Embryology Unit, Department of Anatomy, Histology, Forensic Medicine and Orthopaedics, Sapienza University of Rome, Rome, ItalyDuchenne muscular dystrophy (DMD) is a devastating disorder and is considered to be one of the worst forms of inherited muscular dystrophies. DMD occurs as a result of mutations in the dystrophin gene, leading to progressive muscle fiber degradation and weakness. Although DMD pathology has been studied for many years, there are aspects of disease pathogenesis and progression that have not been thoroughly explored yet. The underlying issue with this is that the development of further effective therapies becomes stalled. It is becoming more evident that extracellular vesicles (EVs) may contribute to DMD pathology. EVs are vesicles secreted by cells that exert a multitude of effects via their lipid, protein, and RNA cargo. EV cargo (especially microRNAs) is also said to be a good biomarker for identifying the status of specific pathological processes that occur in dystrophic muscle, such as fibrosis, degeneration, inflammation, adipogenic degeneration, and dilated cardiomyopathy. On the other hand, EVs are becoming more prominent vehicles for custom-engineered cargos. In this review, we will discuss the possible contribution of EVs to DMD pathology, their potential use as biomarkers, and the therapeutic efficacy of both, EV secretion inhibition and custom-engineered cargo delivery.https://www.frontiersin.org/articles/10.3389/fphys.2023.1130063/fullextracellular vesicles (EVs)exosomesmuscle regenerationmiRNAsmuscular dystrophiescardiomyopathy
spellingShingle Laura Yedigaryan
Maurilio Sampaolesi
Maurilio Sampaolesi
Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
Frontiers in Physiology
extracellular vesicles (EVs)
exosomes
muscle regeneration
miRNAs
muscular dystrophies
cardiomyopathy
title Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
title_full Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
title_fullStr Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
title_full_unstemmed Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
title_short Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression
title_sort extracellular vesicles and duchenne muscular dystrophy pathology modulators of disease progression
topic extracellular vesicles (EVs)
exosomes
muscle regeneration
miRNAs
muscular dystrophies
cardiomyopathy
url https://www.frontiersin.org/articles/10.3389/fphys.2023.1130063/full
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