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DOI | 10.1038/s41467-018-08232-4 |
Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy | |
Bengtsson, Niclas E.1,2; Hall, John K.1,2; Odom, Guy L.1,2; Phelps, Michael P.3; Andrus, Colin R.4,5; Hawkins, R. David4,5; Hauschka, Stephen D.2,6; Chamberlain, Joel R.2,4; Chamberlain, Jeffrey S.1,2,4,6 | |
2019-01-16 | |
发表期刊 | NATURE COMMUNICATIONS
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ISSN | 2041-1723 |
出版年 | 2017 |
卷号 | 8 |
文章类型 | Article |
语种 | 英语 |
国家 | USA |
英文摘要 | Gene replacement therapies utilizing adeno-associated viral (AAV) vectors hold great promise for treating Duchenne muscular dystrophy (DMD). A related approach uses AAV vectors to edit specific regions of the DMD gene using CRISPR/Cas9. Here we develop multiple approaches for editing the mutation in dystrophic mdx(4cv) mice using single and dual AAV vector delivery of a muscle-specific Cas9 cassette together with single-guide RNA cassettes and, in one approach, a dystrophin homology region to fully correct the mutation. Muscle-restricted Cas9 expression enables direct editing of the mutation, multiexon deletion or complete gene correction via homologous recombination in myogenic cells. Treated muscles express dystrophin in up to 70% of the myogenic area and increased force generation following intramuscular delivery. Furthermore, systemic administration of the vectors results in widespread expression of dystrophin in both skeletal and cardiac muscles. Our results demonstrate that AAV-mediated muscle-specific gene editing has significant potential for therapy of neuromuscular disorders. |
领域 | 资源环境 |
收录类别 | SCI-E |
WOS记录号 | WOS:000393859600001 |
WOS关键词 | MDX MICE ; CRISPR-CAS9 ; EXPRESSION ; SKELETAL ; VECTORS |
WOS类目 | Multidisciplinary Sciences |
WOS研究方向 | Science & Technology - Other Topics |
URL | 查看原文 |
引用统计 | |
文献类型 | 期刊论文 |
条目标识符 | http://119.78.100.173/C666/handle/2XK7JSWQ/204169 |
专题 | 资源环境科学 |
作者单位 | 1.Univ Washington, Dept Neurol, Seattle, WA 98195 USA; 2.Univ Washington, Senator Paul D Wellstone Muscular Dystrophy Coope, Seattle, WA 98195 USA; 3.Univ Washington, Dept Pathol, Seattle, WA 98195 USA; 4.Univ Washington, Dept Med, Seattle, WA 98195 USA; 5.Univ Washington, Dept Genome Sci, Seattle, WA 98195 USA; 6.Univ Washington, Dept Biochem, Seattle, WA 98195 USA |
推荐引用方式 GB/T 7714 | Bengtsson, Niclas E.,Hall, John K.,Odom, Guy L.,et al. Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy[J]. NATURE COMMUNICATIONS,2019,8. |
APA | Bengtsson, Niclas E..,Hall, John K..,Odom, Guy L..,Phelps, Michael P..,Andrus, Colin R..,...&Chamberlain, Jeffrey S..(2019).Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy.NATURE COMMUNICATIONS,8. |
MLA | Bengtsson, Niclas E.,et al."Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy".NATURE COMMUNICATIONS 8(2019). |
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