DFNA15.

Man Wang1,2, Ziyu Zhang1, Xiaohan Wang1

  • 1Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, State Key Laboratory of Digital Medical Engineering, Jiangsu Provincial Key Laboratory of Critical Care Medicine, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Southeast University, Nanjing, China.

Nature communications
|September 18, 2025
PubMed
概括

基因编辑成功地通过精确纠正POU4F3基因突变,在遗传性聋 (DFNA15) 的小鼠模型中恢复了听力. 这种腺基编辑器疗法为遗传性听力损失提供了一个有前途的新疗法.