Rescue of myocytes and locomotion through AAV2/9-2YF intracisternal gene therapy in a rat model of creatine transporter deficiency
通过 AAV2/9-2YF 脑池内基因治疗挽救肌酸转运蛋白缺乏症大鼠的肌细胞和运动能力
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2024.101251
Gabriella Fernandes-Pires, Marcelo Duarte Azevedo, Marc Lanzillo, Clothilde Roux-Petronelli, Pierre-Alain Binz, Cristina Cudalbu, Carmen Sandi, Liliane Tenenbaum, Olivier Braissant
