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临床基因疗法恢复听力:一个模式转变

Shuang Han¹, Ziting Chen¹, Daqi Wang¹

¹ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Fudan University, Shanghai, China; Institutes of Biomedical Science, Fudan University, Shanghai, China; State Key Laboratory of Brain Function and Disorders and MOE Frontiers Center for Brain Science, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China.

Trends in molecular medicine

|September 4, 2025

概括

自体衰退性耳聋9 (DFNB9) 的基因疗法进展迅速. 对OTOF突变的临床试验正在改变遗传性听力损失 (HHL) 的治疗方法,为未来的疗法奠定了基础.

科学领域:

耳鼻喉科
遗传学
复原医学

背景情况:

自体衰退性聋9 (DFNB9) 是由OTOF基因突变引起的,这是遗传性听力损失 (HHL) 的重要原因.
最近的基因疗法有望治愈以前无法治愈的聋症.
OTOF基因在听觉毛细胞功能中起着至关重要的作用.

研究的目的:

对治疗DFNB9的OTOF基因疗法的临床试验进展进行审查.
突出关键的转化方面,包括药物输送,试验设计和安全性.
检查在听觉医学中优化基因治疗的挑战和未来方向.

主要方法:

对OTOF相关的DFNB9进行注册临床试验的系统审查.
转化焦点的分析:内耳药物输送,试验设计,安全性和听觉结果.
挑战的批判性检查:解剖学约束,患者选择和结果标准化.

主要成果:

目前在8个国家进行8项DFNB9的临床试验,这表明该药物正在迅速发展.
开创性的研究正在为基因疗法在听觉医学中的转化奠定基础.
在内耳药物输送,试验设计,安全性和听力恢复方面取得了进展.

结论:

对OTOF突变的基因疗法代表了治疗遗传性听力损失的模式转变.
解决解剖学,患者选择和结果测量挑战对于未来的治疗优化至关重要.
持续的研究和标准化的方法将加速基因疗法的临床转化.

关键词:

其他国家临床试验基因疗法遗传性听力损失

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