Effective knockdown-replace gene therapy in a novel mouse model of DNM1 developmental and epileptic encephalopathy
DNM1 发育性癫痫性脑病新型小鼠模型中有效的敲减-替换基因治疗
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2024.08.009
Devin J Jones, Divya Soundararajan, Noah K Taylor, Osasumwen V Aimiuwu, Pranav Mathkar, Amy Shore, Jia Jie Teoh, Wanqi Wang, Tristan T Sands, Matthew C Weston, Scott Q Harper, Wayne N Frankel