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Updated: Jan 9, 2026

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse
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在CRISPR无RNA基编辑中介的PTC阅读恢复了Otof无意义突变的小鼠的听力

Hanxiao Sun1, Qi Teng2, Wenqing Liu3,4

  • 1The National Key Laboratory of Gene Function Studies and Manipulation, School of Life Sciences, Peking University, Beijing, China.

Nature communications
|December 6, 2025
PubMed
概括

使用AAV介导的奥托费林过度表达的基因治疗显示出对先天性聋的有前途. 一种新的RNA基编辑器成功地恢复了特定的奥托费林无意义突变的小鼠模型中的听力,为临床翻译提供了希望.

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