Novel MECP2 gene therapy is effective in a multicenter study using two mouse models of Rett syndrome and is safe in non-human primates
新型 MECP2 基因疗法在一项使用两种雷特综合征小鼠模型的多中心研究中表现出疗效,且在非人类灵长类动物中安全
期刊:Molecular Therapy
影响因子:12.1
doi:10.1016/j.ymthe.2023.07.013
Samantha Powers, Shibi Likhite, Kamal K Gadalla, Carlos J Miranda, Amy J Huffenberger, Cassandra Dennys, Kevin D Foust, Pablo Morales, Christopher R Pierson, Federica Rinaldi, Stephanie Perry, Brad Bolon, Nicolas Wein, Stuart Cobb, Brian K Kaspar, Kathrin C Meyer