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Variable rescue of microtubule and physiological phenotypes in mdx muscle expressing different miniaturized dystrophins
Delivery of miniaturized dystrophin genes via adeno-associated viral vectors is one leading approach in development to treat Duchenne muscular dystrophy. Here we directly compared the functionality of five mini- and micro-dystrophins via skeletal muscle-specific transgenic expression in dystrophin-d...
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| 發表在: | Hum Mol Genet |
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| Main Authors: | , , , , , , |
| 格式: | Artigo |
| 語言: | Inglês |
| 出版: |
Oxford University Press
2018
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| 主題: | |
| 在線閱讀: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5985723/ https://ncbi.nlm.nih.gov/pubmed/29618008 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddy113 |
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