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Systemic AAV Micro-dystrophin Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by dystrophin gene mutation. Conceptually, replacing the mutated gene with a normal one would cure the disease. However, this task has encountered significant challenges due to the enormous size of the gene and the distribution of m...
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| Publicado no: | Mol Ther |
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| Autor principal: | |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
American Society of Gene & Cell Therapy
2018
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC6171037/ https://ncbi.nlm.nih.gov/pubmed/30093306 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.ymthe.2018.07.011 |
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