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探索内耳疾病的AAV介导基因疗法:从临床前成功到临床潜力

Fan Wu^1,2, Wuhui He², Yun Xiao³

¹Department of Pathology and Laboratory Medicine, The Medical University of South Carolina, Walton Research Building, Room 403-E, Charleston, SC, 29425, USA.

Advanced science (Weinheim, Baden-Wurttemberg, Germany)

|June 20, 2025

概括

腺关联病毒 (AAV) 介导的基因疗法在治疗听力损失方面表现有前途. 本综述涵盖了针对遗传性和获得性听力损失的AAV策略,包括基因替代和毛细胞再生.

科学领域:

耳鼻喉科耳鼻喉科耳鼻喉科
遗传学遗传学是一个
分子疗法分子疗法

背景情况:

听力损失是一个主要的全球健康问题,具有遗传和环境原因.
腺相关病毒 (AAV) 介导的基因疗法正在成为内耳疾病的潜在治疗方法.
目前的临床试验显示了AAV基因疗法的有希望的初步结果.

研究的目的:

审查用于内耳疾病的AAV介导基因疗法的发展.
为了对遗传性听力损失的AAV策略进行分类 (基因替代,基于CRISPR的方法).
对获得性听力损失 (毛细胞再生,预防) 的AAV进行审查.

主要方法:

对听力损失的AAV介导基因疗法进行的临床前和临床研究的审查.
基于听力损失的类型 (遗传与获得) 的治疗策略的分类.
对AAV传导效率和安全配置文件的分析.

主要成果:

在临床前研究中,以AAV为媒介的基因疗法显示出高转导效率和安全性.
基因替代和基于CRISPR的方法是遗传性听力损失的关键策略.
AAV显示了头发细胞再生和预防获得性听力损失的潜力.

结论:

通过AAV介导的基因疗法为各种形式的听力损失提供了有前途的治疗方法.
进一步的研究是必要的,以确认这些治疗的长期安全性和有效性.
这篇评论强调了AAV基因治疗对研究人员和临床医生在听力学和遗传学的重要性.

关键词:

在AAV中,AAV是AAV.获得的听力损失.声器的声器是什么基因疗法基因疗法遗传性听力损失是一种遗传性听力损失.内部耳朵内部耳朵感官毛发细胞感官毛发细胞

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