624. A Single Neonatal Delivery of an Exon 2 Directed AAV9.U7snRNA Vector Results in Long-Term Dystrophin Expression That Prevents Pathologic Features in the Dup2 Mouse
| Autor: | Wein, Nicolas, Simmons, Tabatha R., Gumienny, Felecia, Yurkoski, Jacqueline, Huang, Nianyuan, Muntoni, Francesco, Flanigan, Kevin M. |
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| Zdroj: | In Molecular Therapy May 2016 24 Supplement 1:S247-S248 |
| Databáze: | ScienceDirect |
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