Systemic gene therapy for methylmalonic acidemia using the novel adeno-associated viral vector 44.9
| Autor: | Chandler, Randy J., Di Pasquale, Giovanni, Sloan, Jennifer L., McCoy, Samantha, Hubbard, Brandon T., Kilts, Tina M., Manoli, Irini, Chiorini, John A., Venditti, Charles P. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 8 December 2022 27:61-72 |
| Databáze: | ScienceDirect |
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