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Safe and bodywide muscle transduction in young adult Duchenne muscular dystrophy dogs with adeno-associated virus
The ultimate goal of muscular dystrophy gene therapy is to treat all muscles in the body. Global gene delivery was demonstrated in dystrophic mice more than a decade ago using adeno-associated virus (AAV). However, translation to affected large mammals has been challenging. The only reported attempt...
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| Published in: | Hum Mol Genet |
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| Main Authors: | , , , , , , , , |
| Format: | Artigo |
| Language: | Inglês |
| Published: |
Oxford University Press
2015
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4581611/ https://ncbi.nlm.nih.gov/pubmed/26264580 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/hmg/ddv310 |
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