Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
| Autor: | Shi, Xiaoxia, Bortolussi, Giulia, Collaud, Fanny, Le Brun, Pierre-Romain, Bloemendaal, Lysbeth ten, Guerchet, Nicolas, Rudi de Waart, Dirk, Sellier, Pauline, Duijst, Suzanne, Veron, Philippe, Mingozzi, Federico, Kishimoto, Takashi Kei, Ronzitti, Giuseppe, Bosma, Piter, Muro, Andrés F. |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4) |
| Databáze: | ScienceDirect |
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