Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy
CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in m...
| Main Authors: | , , , , , , , , |
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| Format: | Article |
| Language: | English |
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Nature Portfolio
2017-02-01
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| Series: | Nature Communications |
| Online Access: | https://doi.org/10.1038/ncomms14454 |
| _version_ | 1818343312532176896 |
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| author | Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain |
| author_facet | Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain |
| author_sort | Niclas E. Bengtsson |
| collection | DOAJ |
| description | CRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in mice. |
| first_indexed | 2024-12-13T16:28:35Z |
| format | Article |
| id | doaj.art-42b84f4d2d7e485cb3c0f8f3e7eaf353 |
| institution | Directory Open Access Journal |
| issn | 2041-1723 |
| language | English |
| last_indexed | 2024-12-13T16:28:35Z |
| publishDate | 2017-02-01 |
| publisher | Nature Portfolio |
| record_format | Article |
| series | Nature Communications |
| spelling | doaj.art-42b84f4d2d7e485cb3c0f8f3e7eaf3532022-12-21T23:38:33ZengNature PortfolioNature Communications2041-17232017-02-018111010.1038/ncomms14454Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophyNiclas E. Bengtsson0John K. Hall1Guy L. Odom2Michael P. Phelps3Colin R. Andrus4R. David Hawkins5Stephen D. Hauschka6Joel R. Chamberlain7Jeffrey S. Chamberlain8Department of Neurology, University of WashingtonDepartment of Neurology, University of WashingtonDepartment of Neurology, University of WashingtonDepartment of Pathology, University of WashingtonDepartment of Medicine, University of WashingtonDepartment of Medicine, University of WashingtonSenator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of WashingtonSenator Paul D. Wellstone Muscular Dystrophy Cooperative Research Center, University of WashingtonDepartment of Neurology, University of WashingtonCRISPR/Cas9-mediated gene editing is an emerging strategy to treat Duchenne muscular dystrophy. Here the authors develop multiple CRISPR/Cas9-based approaches to correct different dystrophin gene mutations, and show significant restoration of dystrophin expression in skeletal and cardiac muscle in mice.https://doi.org/10.1038/ncomms14454 |
| spellingShingle | Niclas E. Bengtsson John K. Hall Guy L. Odom Michael P. Phelps Colin R. Andrus R. David Hawkins Stephen D. Hauschka Joel R. Chamberlain Jeffrey S. Chamberlain Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy Nature Communications |
| title | Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy |
| title_full | Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy |
| title_fullStr | Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy |
| title_full_unstemmed | Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy |
| title_short | Muscle-specific CRISPR/Cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for Duchenne muscular dystrophy |
| title_sort | muscle specific crispr cas9 dystrophin gene editing ameliorates pathophysiology in a mouse model for duchenne muscular dystrophy |
| url | https://doi.org/10.1038/ncomms14454 |
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