相关实验视频

Updated: Feb 4, 2026

Electrophoretic Delivery of γ-aminobutyric Acid GABA into Epileptic Focus Prevents Seizures in Mice
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Electrophoretic Delivery of γ-aminobutyric Acid GABA into Epileptic Focus Prevents Seizures in Mice

Published on: May 16, 2019

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基础编辑在SCN8A突变相关的发育性脑病变模型中拯救了发作和突然死亡

Caeley M Reever1,2, Alexis R Boscia1, Tyler Cj Deutsch1

  • 1Department of Anesthesiology, University of Virginia Health System, Charlottesville, Virginia, USA.

The Journal of clinical investigation
|February 2, 2026
PubMed
概括

基编辑成功地纠正了小鼠中的SCN8A R1872W突变,显著改善了SCN8A发育性和性脑病变 (DEE) 的生存率和减少了发作. 这种基因治疗方法为这种严重综合征提供了有希望的向治疗方法.

关键词:
是一种病.基因治疗是一种基因疗法.遗传学 是一个遗传学.神经科学是一个神经科学.通道中的.

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