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Updated: May 23, 2025

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane
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Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Published on: March 16, 2015

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在GJB2主导负突变相关综合征性听力损失模型中,以AAV为媒介的基础编辑恢复了耳间隙结

Takao Ukaji1, Daisuke Arai1, Harumi Tsutsumi1

  • 1Department of Otorhinolaryngology, Juntendo University Faculty of Medicine, Tokyo, Japan.

JCI insight
|March 10, 2025
PubMed
概括

腺基编辑纠正了GJB2 R75W突变,恢复了间隙连接功能. 这种基因治疗方法为遗传性聋和由GJB2突变引起的相关皮肤疾病提供了潜在的治疗方法.

关键词:
基因治疗是一种基因疗法.遗传学 遗传学 是一个治疗方法 治疗方法

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