Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I
| Autor: | Shi, Xiaoxia 1, 5, 7, Bortolussi, Giulia 2, 7, Collaud, Fanny 3, 6, 7, Le Brun, Pierre-Romain 3, Bloemendaal, Lysbeth ten 1, Guerchet, Nicolas 3, Rudi de Waart, Dirk 1, Sellier, Pauline 3, 6, Duijst, Suzanne 1, Veron, Philippe 3, Mingozzi, Federico 3, Kishimoto, Takashi Kei 4, Ronzitti, Giuseppe 3, 6, 8, ∗, Bosma, Piter 1, 8, ∗∗, Muro, Andrés F. 2, 8, ∗∗∗ |
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| Zdroj: | In Molecular Therapy - Methods & Clinical Development 12 December 2024 32(4) |
| Databáze: | ScienceDirect |
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