Systemic gene therapy using an AAV44.9 vector rescues a neonatal lethal mouse model of propionic acidemia
| Autor: | Chandler, Randy J., Di Pasquale, Giovanni, Choi, Eun-Young, Chang, David, Smith, Stephanie N., Sloan, Jennifer L., Hoffmann, Victoria, Li, Lina, Chiorini, John A., Venditti, Charles P. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 14 September 2023 30:181-190 |
| Databáze: | ScienceDirect |
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