Repeated dosing of AAV-mediated liver gene therapy in juvenile rat and mouse models of Crigler-Najjar syndrome type I.
在克里格勒-纳贾尔综合征 I 型幼年大鼠和小鼠模型中重复进行 AAV 介导的肝脏基因治疗
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2024.101363
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
