Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy
肌肉定向 AAV 基因治疗治疗大鼠模型中的婴儿型庞贝氏症
期刊:Molecular Metabolism
影响因子:7
doi:10.1016/j.molmet.2024.101899
Sergio Muñoz, Joan Bertolin, Veronica Jimenez, Maria Luisa Jaén, Miquel Garcia, Anna Pujol, Laia Vilà, Victor Sacristan, Elena Barbon, Giuseppe Ronzitti, Jihad El Andari, Warut Tulalamba, Quang Hong Pham, Jesus Ruberte, Thierry VandenDriessche, Marinee K Chuah, Dirk Grimm, Federico Mingozzi, Fatima