AAVDFNB9

Jieyu Qi1,2,3, Fangzhi Tan1, Liyan Zhang1

  • 1State Key Laboratory of Digital Medical Engineering, Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Jiangsu Province High-Tech Key Laboratory for Bio-Medical Research, Southeast University, Nanjing, 210096, China.

概括

使用腺相关病毒OTOF的基因治疗成功地恢复了聋子儿童的听力. 这种OTOF基因替代疗法在治疗遗传性聋症方面显示出有前途的安全性和有效性.