AAV hamartin gene therapy in a stochastic, cerebral mouse model of tuberous sclerosis type 1
在随机性脑结节性硬化症1型小鼠模型中应用AAV hamartin基因疗法
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2025.101556
Abou Haidar, Edwina; Prabhakar, Shilpa; Geffrey, Alexandra L; Mahamdeh, Mohammed; Tomeh, Tomeh; Breyne, Koen; Roumieh, Elie; Gurevich, Mia; Soberman, Roy J; Wojtkiewicz, Gregory R; Chen, John W; Han, Justin Seungkyu; Stemmer-Rachamimov, Anat; Breakefield, Xandra O