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Updated: Jul 4, 2026

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在使用DB-OTO的Otoferlin缺陷小鼠中,功能性,持续的听力恢复,DB-OTO是一种基于毛细胞特异性AAV的基因疗法

Yoojin Chung1,2, Seth D Koehler1,2, Sarah Cancelarich1

  • 1Regeneron Pharmaceuticals, Inc., Tarrytown, NY 10591, USA.

Molecular therapy. Methods & clinical development
|October 2, 2025
PubMed
概括

使用DB-OTO的基因疗法,一种基于腺相关病毒 (AAV) 的双向量系统,通过启用毛细胞特异性OTOF基因表达来恢复与Otoferlin (OTOF) 相关的先天性听力损失的个体的听力.

关键词:
在 AAV AAV AAV 中.在DB-OTOTO中,我们使用了DB-OTO.DFNB9 DFNB9 在线观看听觉神经病变是一种听觉神经病变.听力障碍 听力障碍 听力障碍这是一个双向量向量.基因治疗的基因疗法听力损失 听力损失是什么一个人的心灵,一个人的心灵这就是Otoferlin.

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相关概念视频

In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.