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Updated: Jul 12, 2025

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基RNA编辑疗法可以治疗由OTOF基因突变引起的听力损失

Yuanyuan Xue1, Yong Tao2, Xing Wang3

  • 1Department of Otolaryngology-Head and Neck Surgery, Shanghai Ninth People's Hospital, Shanghai Jiaotong University School of Medicine, Shanghai 200011, China; HuidaGene Therapeutics Co., Ltd., Shanghai 200131, China.

Molecular therapy : the journal of the American Society of Gene Therapy
|November 2, 2023
PubMed
概括

一种新的基因编辑工具有效地恢复了Otoferlin (OTOF) 基因突变的小鼠的听力. 这种增强的迷你dCas13XRNA基编辑器 (emxABE) 为与OTOF相关的耳聋提供了一个有前途的治疗策略.

关键词:
在外面,在外面.编辑RNA基的编辑在 emxABEE 中使用.基因治疗的基因疗法听力损失 听力损失是什么这就是Otoferlin.

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