副突触中间神经元受损会导致 SCN8A 发育不良和癫痫性脑病的突触传递障碍和癫痫发作。

IF 6.3 1区 医学 Q1 MEDICINE, RESEARCH & EXPERIMENTAL
Raquel M Miralles, Alexis R Boscia, Shrinidhi Kittur, Jessica C Hanflink, Payal S Panchal, Matthew S Yorek, Tyler C J Deutsch, Caeley M Reever, Shreya R Vundela, Eric R Wengert, Manoj K Patel
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引用次数: 0

摘要

SCN8A 发育性癫痫脑病(DEE)是一种严重的癫痫综合征,是由 SCN8A 基因编码的电压门控钠通道 Nav1.6 突变引起的。Nav1.6 在兴奋性和抑制性神经元中均有表达,但以往的研究主要集中于 SCN8A 突变如何影响兴奋性神经元,对抑制性中间神经元的重要性研究有限。为了在 SCN8A DEE 中评估 PV 中间神经元的功能,我们使用了 2 种携带源自患者的 SCN8A 功能增益变体的小鼠模型:Scn8aD/+ 和 Scn8aW/+-PV,前者的 SCN8A 变体 N1768D 全局表达,后者的 SCN8A 变体 R1872W 选择性地在 PV 中间神经元中表达。R1872W SCN8A变体选择性地在PV中间神经元中表达会导致自发性癫痫发作和癫痫发作诱导的死亡。电生理学研究表明,Scn8aD/+和Scn8aW/+-PV中间神经元易受去极化阻滞的影响,并表现出持续性钠电流增加。对PV中间神经元和锥体细胞之间的突触连接进行的评估显示,Scn8aD/+和Scn8aW/+-PV中间神经元存在突触传递缺陷。总之,我们的研究结果表明,通过去极化阻滞导致的 PV 中间神经元衰竭以及抑制性突触损伤可能会引起 SCN8A DEE 的整体抑制性降低,从而导致无节制的兴奋,并最终导致癫痫发作和癫痫发作诱导的死亡。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
Parvalbumin interneuron impairment causes synaptic transmission deficits and seizures in SCN8A developmental and epileptic encephalopathy.

SCN8A developmental and epileptic encephalopathy (DEE) is a severe epilepsy syndrome resulting from mutations in the voltage-gated sodium channel Nav1.6, encoded by the gene SCN8A. Nav1.6 is expressed in excitatory and inhibitory neurons, yet previous studies primarily focus on how SCN8A mutations affect excitatory neurons, with limited studies on the importance of inhibitory interneurons. Parvalbumin (PV) interneurons are a prominent inhibitory interneuron subtype that expresses Nav1.6. To assess PV interneuron function within SCN8A DEE, we used 2 mouse models harboring patient-derived SCN8A gain-of-function variants, Scn8aD/+, where the SCN8A variant N1768D is expressed globally, and Scn8aW/+-PV, where the SCN8A variant R1872W is selectively expressed in PV interneurons. Expression of the R1872W SCN8A variant selectively in PV interneurons led to development of spontaneous seizures and seizure-induced death. Electrophysiology studies showed that Scn8aD/+ and Scn8aW/+-PV interneurons were susceptible to depolarization block and exhibited increased persistent sodium current. Evaluation of synaptic connections between PV interneurons and pyramidal cells showed synaptic transmission deficits in Scn8aD/+ and Scn8aW/+-PV interneurons. Together, our findings indicate that PV interneuron failure via depolarization block along with inhibitory synaptic impairment likely elicits an overall inhibitory reduction in SCN8A DEE, leading to unchecked excitation and ultimately resulting in seizures and seizure-induced death.

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来源期刊
JCI insight
JCI insight Medicine-General Medicine
CiteScore
13.70
自引率
1.20%
发文量
543
审稿时长
6 weeks
期刊介绍: JCI Insight is a Gold Open Access journal with a 2022 Impact Factor of 8.0. It publishes high-quality studies in various biomedical specialties, such as autoimmunity, gastroenterology, immunology, metabolism, nephrology, neuroscience, oncology, pulmonology, and vascular biology. The journal focuses on clinically relevant basic and translational research that contributes to the understanding of disease biology and treatment. JCI Insight is self-published by the American Society for Clinical Investigation (ASCI), a nonprofit honor organization of physician-scientists founded in 1908, and it helps fulfill the ASCI's mission to advance medical science through the publication of clinically relevant research reports.
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