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Current Pharmacological Strategies for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of mus...
Autores principales: | , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
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Frontiers Media S.A.
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8417245/ https://www.ncbi.nlm.nih.gov/pubmed/34490244 http://dx.doi.org/10.3389/fcell.2021.689533 |
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author | Yao, Shanshan Chen, Zihao Yu, Yuanyuan Zhang, Ning Jiang, Hewen Zhang, Ge Zhang, Zongkang Zhang, Baoting |
author_facet | Yao, Shanshan Chen, Zihao Yu, Yuanyuan Zhang, Ning Jiang, Hewen Zhang, Ge Zhang, Zongkang Zhang, Baoting |
author_sort | Yao, Shanshan |
collection | PubMed |
description | Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. There is still no cure for DMD so far and the standard of care is principally limited to symptom relief through glucocorticoids treatments. Current therapeutic strategies could be divided into two lines. Dystrophin-targeted therapeutic strategies that aim at restoring the expression and/or function of dystrophin, including gene-based, cell-based and protein replacement therapies. The other line of therapeutic strategies aims to improve muscle function and quality by targeting the downstream pathological changes, including inflammation, fibrosis, and muscle atrophy. This review introduces the important developments in these two lines of strategies, especially those that have entered the clinical phase and/or have great potential for clinical translation. The rationale and efficacy of each agent in pre-clinical or clinical studies are presented. Furthermore, a meta-analysis of gene profiling in DMD patients has been performed to understand the molecular mechanisms of DMD. |
format | Online Article Text |
id | pubmed-8417245 |
institution | National Center for Biotechnology Information |
language | English |
publishDate | 2021 |
publisher | Frontiers Media S.A. |
record_format | MEDLINE/PubMed |
spelling | pubmed-84172452021-09-05 Current Pharmacological Strategies for Duchenne Muscular Dystrophy Yao, Shanshan Chen, Zihao Yu, Yuanyuan Zhang, Ning Jiang, Hewen Zhang, Ge Zhang, Zongkang Zhang, Baoting Front Cell Dev Biol Cell and Developmental Biology Duchenne muscular dystrophy (DMD) is a lethal, X-linked neuromuscular disorder caused by the absence of dystrophin protein, which is essential for muscle fiber integrity. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. There is still no cure for DMD so far and the standard of care is principally limited to symptom relief through glucocorticoids treatments. Current therapeutic strategies could be divided into two lines. Dystrophin-targeted therapeutic strategies that aim at restoring the expression and/or function of dystrophin, including gene-based, cell-based and protein replacement therapies. The other line of therapeutic strategies aims to improve muscle function and quality by targeting the downstream pathological changes, including inflammation, fibrosis, and muscle atrophy. This review introduces the important developments in these two lines of strategies, especially those that have entered the clinical phase and/or have great potential for clinical translation. The rationale and efficacy of each agent in pre-clinical or clinical studies are presented. Furthermore, a meta-analysis of gene profiling in DMD patients has been performed to understand the molecular mechanisms of DMD. Frontiers Media S.A. 2021-08-19 /pmc/articles/PMC8417245/ /pubmed/34490244 http://dx.doi.org/10.3389/fcell.2021.689533 Text en Copyright © 2021 Yao, Chen, Yu, Zhang, Jiang, Zhang, Zhang and Zhang. 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 | Cell and Developmental Biology Yao, Shanshan Chen, Zihao Yu, Yuanyuan Zhang, Ning Jiang, Hewen Zhang, Ge Zhang, Zongkang Zhang, Baoting Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title | Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title_full | Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title_fullStr | Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title_full_unstemmed | Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title_short | Current Pharmacological Strategies for Duchenne Muscular Dystrophy |
title_sort | current pharmacological strategies for duchenne muscular dystrophy |
topic | Cell and Developmental Biology |
url | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8417245/ https://www.ncbi.nlm.nih.gov/pubmed/34490244 http://dx.doi.org/10.3389/fcell.2021.689533 |
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