Lentivirus-modified hematopoietic stem cell gene therapy for advanced symptomatic juvenile metachromatic leukodystrophy: a long-term follow-up pilot study.
慢病毒修饰的造血干细胞基因疗法治疗晚期症状性青少年异染性脑白质营养不良:一项长期随访试点研究
期刊:Protein & Cell
影响因子:12.8
doi:10.1093/procel/pwae037
Zhang Zhao, Jiang Hua, Huang Li, Liu Sixi, Zhou Xiaoya, Cai Yun, Li Ming, Gao Fei, Liang Xiaoting, Tsang Kam-Sze, Chen Guangfu, Ma Chui-Yan, Chai Yuet-Hung, Liu Hongsheng, Yang Chen, Yang Mo, Zhang Xiaoling, Han Shuo, Du Xin, Chen Ling, Hwu Wuh-Liang, Zhuo Jiacai, Lian Qizhou