Improved MECP2 Gene Therapy Extends the Survival of MeCP2-Null Mice without Apparent Toxicity after Intracisternal Delivery
改进的MECP2基因疗法可延长MeCP2基因敲除小鼠的生存期，且在脑池内给药后未观察到明显毒性。
期刊:Molecular Therapy-Methods & Clinical Development
影响因子:4.7
doi:10.1016/j.omtm.2017.04.006
Sinnett, Sarah E; Hector, Ralph D; Gadalla, Kamal K E; Heindel, Clifford; Chen, Daphne; Zaric, Violeta; Bailey, Mark E S; Cobb, Stuart R; Gray, Steven J
