Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update
Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the...
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doaj-06d1bef7e1b14260b09ef0516949d8102020-11-25T03:27:58ZengMDPI AGGenes2073-44252020-07-011183783710.3390/genes11080837Therapeutic Strategies for Duchenne Muscular Dystrophy: An UpdateChengmei Sun0Luoan Shen1Zheng Zhang2Xin Xie3Zhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, ChinaZhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, ChinaZhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, ChinaZhejiang University-University of Edinburgh Institute, School of Medicine, Zhejiang University, Haining 314400, ChinaNeuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked <i>dystrophin</i> gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy.https://www.mdpi.com/2073-4425/11/8/837Duchenne muscular dystrophypathogenesisdystrophin restorationgene therapycell transplantation |
collection |
DOAJ |
language |
English |
format |
Article |
sources |
DOAJ |
author |
Chengmei Sun Luoan Shen Zheng Zhang Xin Xie |
spellingShingle |
Chengmei Sun Luoan Shen Zheng Zhang Xin Xie Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update Genes Duchenne muscular dystrophy pathogenesis dystrophin restoration gene therapy cell transplantation |
author_facet |
Chengmei Sun Luoan Shen Zheng Zhang Xin Xie |
author_sort |
Chengmei Sun |
title |
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update |
title_short |
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update |
title_full |
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update |
title_fullStr |
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update |
title_full_unstemmed |
Therapeutic Strategies for Duchenne Muscular Dystrophy: An Update |
title_sort |
therapeutic strategies for duchenne muscular dystrophy: an update |
publisher |
MDPI AG |
series |
Genes |
issn |
2073-4425 |
publishDate |
2020-07-01 |
description |
Neuromuscular disorders encompass a heterogeneous group of conditions that impair the function of muscles, motor neurons, peripheral nerves, and neuromuscular junctions. Being the most common and most severe type of muscular dystrophy, Duchenne muscular dystrophy (DMD), is caused by mutations in the X-linked <i>dystrophin</i> gene. Loss of dystrophin protein leads to recurrent myofiber damage, chronic inflammation, progressive fibrosis, and dysfunction of muscle stem cells. Over the last few years, there has been considerable development of diagnosis and therapeutics for DMD, but current treatments do not cure the disease. Here, we review the current status of DMD pathogenesis and therapy, focusing on mutational spectrum, diagnosis tools, clinical trials, and therapeutic approaches including dystrophin restoration, gene therapy, and myogenic cell transplantation. Furthermore, we present the clinical potential of advanced strategies combining gene editing, cell-based therapy with tissue engineering for the treatment of muscular dystrophy. |
topic |
Duchenne muscular dystrophy pathogenesis dystrophin restoration gene therapy cell transplantation |
url |
https://www.mdpi.com/2073-4425/11/8/837 |
work_keys_str_mv |
AT chengmeisun therapeuticstrategiesforduchennemusculardystrophyanupdate AT luoanshen therapeuticstrategiesforduchennemusculardystrophyanupdate AT zhengzhang therapeuticstrategiesforduchennemusculardystrophyanupdate AT xinxie therapeuticstrategiesforduchennemusculardystrophyanupdate |
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1724586069746253824 |