Combination HSCT and intravenous AAV-mediated gene therapy in a canine model proves pivotal for translation of Krabbe disease therapy
在犬类模型中，联合 HSCT 和静脉 AAV 介导的基因治疗被证明是克拉伯病治疗转化的关键
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2023.11.014
Allison M Bradbury, Jessica Bagel, Gary Swain, Keiko Miyadera, Jill P Pesayco, Charles-Antoine Assenmacher, Becky Brisson, Ian Hendricks, Xiao H Wang, Zachary Herbst, Nettie Pyne, Patricia Odonnell, G Diane Shelton, Michael Gelb, Neil Hackett, Paul Szabolcs, Charles H Vite, Maria Escolar
