Neurologic Recovery in MPS I and MPS II Mice by AAV9-Mediated Gene Transfer to the CNS After the Development of Cognitive Dysfunction
认知功能障碍发生后,通过 AAV9 介导的中枢神经系统基因转移,MPS I 和 MPS II 小鼠的神经功能得以恢复
期刊:Human Gene Therapy
影响因子:3.9
doi:10.1089/hum.2022.162
Kelly M Podetz-Pedersen, Kanut Laoharawee, Sajya Singh, Tam T Nguyen, Miles C Smith, Alexa Temme, Karen Kozarsky, R Scott McIvor, Lalitha R Belur