相关实验视频

Updated: Mar 2, 2026

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

Published on: February 17, 2023

精确而高效的DNA基编辑恢复了成年DFNB9小鼠模型中的正常听力

Ziyu Zhang¹, Man Wang¹, Fangzhi Tan¹

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 210096, China.

Med (New York, N.Y.)

|February 28, 2026

概括

基因编辑成功地恢复了OTOF缺陷小鼠的听力,证明了治疗DFNB9听力损失的广泛治疗窗口. 这种腺基编辑方法对未来的基因疗法有很大的前景.

科学领域:

遗传学是一个遗传学.
眼科医生眼科眼科
神经科学是一个神经科学.

背景情况:

在OTOF基因的突变导致自体逆性非综合征性听力损失 (DFNB9),听力神经病变的主要原因.
虽然基因增强已在DFNB9患者中使用,但最佳治疗时间尚不清楚.

研究的目的:

为了确定一个最佳的腺基编辑器来纠正OTOFc.2815C>T突变.
在小鼠模型中评估基编辑器介导的DFNB9基因疗法的疗效和安全性,并确定治疗时间窗口.

主要方法:

选了腺基编辑器,以找到一个有效的OTOF突变.
通过腺相关病毒将所选编辑器传递给不同年龄段的OTOF缺乏小鼠的耳.
在接受治疗的小鼠和野生类型对照中评估听觉功能和安全性.

主要成果:

Nme2ABE8e显示出优越的目标编辑效率.
在成年OTOF缺陷小鼠中,由腺相关病毒介导的Nme2ABE8e的输送恢复了听力,至少在6个月内将听力恢复到接近正常水平.
晚年 (2-4个月) 的治疗也改善了听力,这表明治疗窗口延长了,没有观察到耳毒性或神经缺陷.

结论:

Nme2ABE8e是一种安全有效的基因治疗工具,用于DFNB9.
患有DFNB9的患者可能会从相对较长的基因治疗干预治疗窗口中受益.

关键词:

在AAV基因疗法中,AAV基因疗法 DFNB9 DFNB9 在线观看编辑DNA基的编辑这是突变的突变.甲状腺甲状腺是什么双AAV-双AAV可以使用.听力恢复听力恢复遗传性听力损失是一种遗传性听力损失.内耳的内耳,就是内耳的内耳.临床前研究研究.治疗时间窗口.

更多相关视频

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

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

Published on: March 16, 2015

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Published on: March 2, 2018

相关概念视频

In-vitro Mutagenesis

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.