吴侠副教授博士生导师

学   院：美加墨世界杯官方网站

电子邮箱：wuxia@ecust.edu.cn

通讯地址：上海市徐汇区梅陇路130号美加墨世界杯官方网站科辅助七楼

研究方向与成果：

        主要从事基因治疗药物的研发和临床转化研究。以单基因遗传罕见病、关节疾病、老年退行性疾病、癌症等为研究对象，利用腺病毒和腺相关病毒作为递送工具，探讨疾病的作用机制，设计、优化药物载体，进行药理学、毒理学研究，进而推进基因治疗药物的临床转化。

       2019年与中国医学科学院天津血液病医院合作，开展了亚洲首个B型血友病基因治疗药物临床研究。2025年该药物成功上市，成为亚洲首个研发并上市的罕见病基因治疗药物。该成果在The Lancet Haematology（2022），Nature Medicine（2025、2026）发表高水平研究论文，两次被评为中国血液学十大研究进展（2022、2025），先后获得天津市科技进步一等奖（2024）和中华医学科技一等奖（2025）。开发了法布雷病等10余个罕见病基因治疗药物，其中6个药物实现临床转化。在大病基因治疗方面，治疗骨性关节炎和HPV感染的药物也进入注册临床阶段。

       主持国家重点研发计划、国家“863计划”、国家自然基金、省部级基金项目等10余项；申请国内国际发明专利30余项，9项获得授权；获得省部级科技一等奖2项；在The Lancet Haematology、Nature Medicine、Gene & Disease等发表研究论文30余篇，获得多项省部级人才类称号。

获奖情况：

天津市科技进步一等奖（2024）

中华医学科技一等奖（2025）

发表论文:

1. Xu Feng^#, Zhang Ai^#, Ju Mankai^#, Wu Xia^#, Xu Bingqi^#, Pei Xiaolei, Wang Yuhua, Liu Wei, Liu Aiguo, Jiang Wei, Dai Xinyue, Chen Yunfei, Liu Xiaofan, Fu Rongfeng, Sun Ting, Gu Wenjing, Zheng Jing, Du Zengmin, Yang Caifeng, Xiao Xiao^*, Yang Renchi^*, Hu Qun^*, Zhang Lei^*. Factor IX Padua AAV gene therapy in adolescents with hemophilia B: a phase 1 trial. Nature Medicine. 2026 Sept.17

2. Xue Feng^#, Ju Mankai^#, Zhu Tienan^#, Zhou Zeping^#, Sun Jing, Yang Linhua, Yan Zhenyu, Zhou Hu, Du Xin, Zheng Changcheng, Zheng Jing, Wu Xia, Du Zengmin, Jiang Wei, Yang Caifeng, Xiao Xiao, Liu Wei, Yang Renchi, Zhang Lei^*. Factor IX-Padua AAV gene therapy in hemophilia B: phases 1/2 and 3 trials. Nature Medicine. 2026. 32: 93-102

3. Tang Siqi^#, Luo Wenshu, Wu Shihao, Yuan Meng, Wen Jiashuo, Zhong Guoshen, Shen Leshan, Jiang Wei, Cheng Cheng^*, Wu Xia*, Xiao Xiao^*. Hippocampus-targeted BDNF gene therapy to rescue cognitive impairments of Alzheimer's disease in multiple mouse models. Genes & Diseases. 2026 13, 101649

4. Zhang Feixu^#, Zhou Xinyue, Hua Baolai, He Xinyi, Li Zhanao, Xiao Xiao, Wu Xia*. Activated factor X delivered by adeno-associated virus significantly inhibited bleeding and alleviated hemophilic synovitis in hemophilic mice. Gene Therapy. 2024, 31: 544-552

5. Zhang Yanan^#, Tang Dingyue^#, Wang Li, Yang Jing, Wu Xia*, Xiao Xiao^*, Wang Jianshe^*. Prevention of Portal-Tract Fibrosis in Zfyve19-/- Mouse Model with Adeno-Associated Virus Vector Delivering ZFYVE19. Hum Gene Ther. 2023, 34: 1219-1229

6. Zhou Kaiyi^#, Yuan Meng, Sun Jiabao, Zhang Feixu, Zong Xiaoying, Li Zhanao, Tang Dingyue, Zhou Lichen, Zheng Jing, Xiao Xiao^*, Wu Xia*. Sildenafil increases AAV9 transduction after a systemic administration and enhances AAV9-dystrophin therapeutic effect in mdx mice. Gene Ther. 2024,31: 19-30

7 Zhou Kaiyi^#, Yuan Meng, Sun Jiabao, Zhang Feixu, Li Xinting, Xiao Xiao^*, Wu Xia*. Co-delivery of IL-1Ra and SOX9 via AAV inhibits inflammation and promotes cartilage repair in surgically induced osteoarthritis animal models. Gene Therapy. 2025, 32: 211-222

8. Xue Feng^#, Li Huiyuan^#, Wu Xia^#, Liu Wei^#, Zhang Feixu, Tang Dingyue, Chen Yunfei, Wang Wentian, Chi Ying, Zheng Jing, Du Zengmin, Jiang Wei, Zhong Chen, Wei Jun, Zhu Ping, Fu Rongfeng, Liu Xiaofan, Chen Lingling, Pei Xiaolei, Sun Junjiang, Cheng Tao^*, Yang Renchi^*, Xiao Xiao^*, Lei Zhang^*. Safety and activity of an engineered, liver-tropic adeno-associated virus vector expressing a hyperactive Padua factor IX administered with prophylactic glucocorticoids in patients with haemophilia B: a single-centre, single-arm, phase 1, pilot trial. Lancet Haematology. 2022, 9: e504-e513

9. Zhong Chen^#, Chen Zifeng, Xia Yong, Wu Jun, Zhang Feixu, Cheng Cheng, Wu Xia*, Zhuang Yingping, Xiao Xiao^*. Treatment of experimental autoimmune encephalomyelitis using AAV gene therapy by blocking T cell costimulatory pathways. Mol Ther Methods Clin Dev. 2022, 27: 461-475

10. Zhong Chen^#, Jiang Wei, Wang Yefan, Sun Junjiang, Wu Xia*, Zhuang Yingping, Xiao Xiao^*.  Repeated systemic dosing of AAV vectors in immunocompetent mice after  blockade of T-cell costimulatory pathways, Human Gene Therapy, 2022, 33:  290-300