Long-Term Therapeutic Efficacy of Intravenous AAV-Mediated Hamartin Replacement in Mouse Model of Tuberous Sclerosis Type 1
静脉注射 AAV 介导的 Hamartin 替代疗法对 1 型结节性硬化症小鼠模型的长期治疗效果
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:
doi:10.1016/j.omtm.2019.08.003
Shilpa Prabhakar, Pike See Cheah, Xuan Zhang, Max Zinter, Maria Gianatasio, Eloise Hudry, Roderick T Bronson, David J Kwiatkowski, Anat Stemmer-Rachamimov, Casey A Maguire, Miguel Sena-Esteves, Bakhos A Tannous, Xandra O Breakefield