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Updated: Jul 30, 2026

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane
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在Usher1G临床前模型中,通过基因疗法恢复内耳功能的延长时间框架

Ghizlene Lahlou1,2, Charlotte Calvet1, François Simon3,4,5

  • 1Institut Pasteur, Université Paris Cité, Inserm, Institut de l'Audition, Paris, France.

JCI insight
|January 9, 2024
PubMed
概括

在典型的新生儿期之外,基因疗法可以恢复阿舍尔综合征1G型 (USH1G) 小鼠的听力和平衡. 这一发现表明,治疗新生儿遗传性聋盲的常见原因的治疗窗口更广.

关键词:
基因治疗是一种基因疗法.鼠标模型 鼠标模型治疗方法 治疗方法

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相关概念视频

Unrenewable Cells00:50

Unrenewable Cells

In humans, the photoreceptor cells of the eye and sensory hair cells of the ear lack stem cells. These cells are thus unrenewable and cannot be replaced when they are damaged or destroyed.
Photoreceptors
The retina is composed of several layers and contains specialized cells called photoreceptors. The photoreceptors (rods and cones) change their membrane potential when stimulated by light energy. There are two types of photoreceptors—rods and cones—which differ in the shape of their outer...