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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...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Frontiers Media S.A.
2023
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9987248/ https://www.ncbi.nlm.nih.gov/pubmed/36891137 http://dx.doi.org/10.3389/fphys.2023.1130063 |
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author | Yedigaryan, Laura Sampaolesi, Maurilio |
author_facet | Yedigaryan, Laura Sampaolesi, Maurilio |
author_sort | Yedigaryan, Laura |
collection | PubMed |
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. |
format | Online Article Text |
id | pubmed-9987248 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2023 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-99872482023-03-07 Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression Yedigaryan, Laura Sampaolesi, Maurilio Front Physiol Physiology 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. Frontiers Media S.A. 2023-02-14 /pmc/articles/PMC9987248/ /pubmed/36891137 http://dx.doi.org/10.3389/fphys.2023.1130063 Text en Copyright © 2023 Yedigaryan and Sampaolesi. https://creativecommons.org/licenses/by/4.0/This is an open-access article distributed under the terms of the Creative Commons Attribution License (CC BY). The use, distribution or reproduction in other forums is permitted, provided the original author(s) and the copyright owner(s) are credited and that the original publication in this journal is cited, in accordance with accepted academic practice. No use, distribution or reproduction is permitted which does not comply with these terms. |
spellingShingle | Physiology Yedigaryan, Laura Sampaolesi, Maurilio Extracellular vesicles and Duchenne muscular dystrophy pathology: Modulators of disease progression |
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 | Physiology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9987248/ https://www.ncbi.nlm.nih.gov/pubmed/36891137 http://dx.doi.org/10.3389/fphys.2023.1130063 |
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