AAV-mediated gene therapy for galactosialidosis: A long-term safety and efficacy study
| Autor: | Hu, Huimin, Mosca, Rosario, Gomero, Elida, van de Vlekkert, Diantha, Campos, Yvan, Fremuth, Leigh E., Brown, Scott A., Weesner, Jason A., Annunziata, Ida, d’Azzo, Alessandra |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 10 December 2021 23:644-658 |
| Databáze: | ScienceDirect |
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