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Full-Length Dystrophin Reconstitution with Adeno-Associated Viral Vectors
Duchenne muscular dystrophy (DMD) is the most common lethal muscle disorder in children. It is caused by mutations of the dystrophin gene. Adeno-associated virus (AAV)-mediated gene replacement therapy has been actively pursued to treat DMD. However, this promising therapeutic modality has been chal...
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| Main Authors: | , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
Mary Ann Liebert, Inc.
2014
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| Assuntos: | |
| Acceso en liña: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4064726/ https://ncbi.nlm.nih.gov/pubmed/24580018 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2013.210 |
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