Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse
| Autor: | Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., Flanigan, Kevin M. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 8 September 2022 26:279-293 |
| Databáze: | ScienceDirect |
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