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Repeated Dosing of AAV-mediated Liver Gene Therapy in Juvenile Rat and Mouse Models of Crigler-Najjar Syndrome Type I

Xiaoxia Shi, Giulia Bortolussi, Fanny Collaud, Pierre-Romain Lebrun, Lysbeth ten Bloemendaal, Nicolas Guerchet, Dirk Rudi de Waart,Pauline Sellier, Suzanne Duijst,Philippe Veron,Federico Mingozzi, Takashi Kei Kishimoto,Giuseppe Ronzitti,Piter Bosma, Andrés F. Muro

Molecular Therapy - Methods &amp Clinical Development（2024）

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