Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy
Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal m...
| Main Authors: | , , , |
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| Format: | Article |
| Language: | English |
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The Company of Biologists
2015-03-01
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| Series: | Disease Models & Mechanisms |
| Subjects: | |
| Online Access: | http://dmm.biologists.org/content/8/3/195 |
| _version_ | 1818429826607874048 |
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| author | Joe W. McGreevy Chady H. Hakim Mark A. McIntosh Dongsheng Duan |
| author_facet | Joe W. McGreevy Chady H. Hakim Mark A. McIntosh Dongsheng Duan |
| author_sort | Joe W. McGreevy |
| collection | DOAJ |
| description | Duchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal models of DMD have been developed over the last 30 years, ranging from invertebrate to large mammalian models. mdx mice are the most commonly employed models in DMD research and have been used to lay the groundwork for DMD gene therapy. After ~30 years of development, the field has reached the stage at which the results in mdx mice can be validated and scaled-up in symptomatic large animals. The canine DMD (cDMD) model will be excellent for these studies. In this article, we review the animal models for DMD, the pros and cons of each model system, and the history and progress of preclinical DMD gene therapy research in the animal models. We also discuss the current and emerging challenges in this field and ways to address these challenges using animal models, in particular cDMD dogs. |
| first_indexed | 2024-12-14T15:23:41Z |
| format | Article |
| id | doaj.art-e4bc73bf374343ccb8c9d259858dea64 |
| institution | Directory Open Access Journal |
| issn | 1754-8403 1754-8411 |
| language | English |
| last_indexed | 2024-12-14T15:23:41Z |
| publishDate | 2015-03-01 |
| publisher | The Company of Biologists |
| record_format | Article |
| series | Disease Models & Mechanisms |
| spelling | doaj.art-e4bc73bf374343ccb8c9d259858dea642022-12-21T22:56:06ZengThe Company of BiologistsDisease Models & Mechanisms1754-84031754-84112015-03-018319521310.1242/dmm.018424018424Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapyJoe W. McGreevyChady H. HakimMark A. McIntoshDongsheng DuanDuchenne muscular dystrophy (DMD) is a progressive muscle-wasting disorder. It is caused by loss-of-function mutations in the dystrophin gene. Currently, there is no cure. A highly promising therapeutic strategy is to replace or repair the defective dystrophin gene by gene therapy. Numerous animal models of DMD have been developed over the last 30 years, ranging from invertebrate to large mammalian models. mdx mice are the most commonly employed models in DMD research and have been used to lay the groundwork for DMD gene therapy. After ~30 years of development, the field has reached the stage at which the results in mdx mice can be validated and scaled-up in symptomatic large animals. The canine DMD (cDMD) model will be excellent for these studies. In this article, we review the animal models for DMD, the pros and cons of each model system, and the history and progress of preclinical DMD gene therapy research in the animal models. We also discuss the current and emerging challenges in this field and ways to address these challenges using animal models, in particular cDMD dogs.http://dmm.biologists.org/content/8/3/195Duchenne muscular dystrophyDystrophinAnimal modelCanine DMDGene therapy |
| spellingShingle | Joe W. McGreevy Chady H. Hakim Mark A. McIntosh Dongsheng Duan Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy Disease Models & Mechanisms Duchenne muscular dystrophy Dystrophin Animal model Canine DMD Gene therapy |
| title | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| title_full | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| title_fullStr | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| title_full_unstemmed | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| title_short | Animal models of Duchenne muscular dystrophy: from basic mechanisms to gene therapy |
| title_sort | animal models of duchenne muscular dystrophy from basic mechanisms to gene therapy |
| topic | Duchenne muscular dystrophy Dystrophin Animal model Canine DMD Gene therapy |
| url | http://dmm.biologists.org/content/8/3/195 |
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