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来自干细胞的细胞外囊泡在SHANK3相关的ASD神经和小鼠模型中拯救细胞表型和行为缺陷

Ashwani Choudhary¹, Idan Rosh¹, Yara Hussein¹

¹Sagol Department of Neurobiology, Faculty of Natural Sciences, University of Haifa, Haifa, Israel.

Cell death & disease

|February 21, 2026

概括

来自SHANK3缺陷神经元的细胞外囊泡 (EVs) 传递神经元过激动性. 介酶干细胞衍生的EVs通过在小鼠中拯救自闭症谱系障碍 (ASD) 末型来显示治疗潜力.

科学领域:

神经科学是一个神经科学.
细胞生物学细胞生物学
遗传学是一个遗传学.

背景情况:

SHANK3 缺乏与自闭症谱系障碍 (ASD) 和菲兰-麦克德米德综合征 (PMS) 有关.
皮层神经元过度兴奋性是ASD的早期内类型.
通过细胞外囊泡 (EVs) 的细胞间通信在神经元功能中起作用.

研究的目的:

研究EVs在SHANK3缺陷相关神经元功能障碍中的作用.
探索EVs对ASD和相关神经发育障碍的治疗潜力.

主要方法:

利用人类诱导多能干细胞 (iPSC) 衍生的皮质神经元和Shank3B-/-小鼠.
在突变神经元和控制神经元之间进行了EV传输实验.
进行了EVs的蛋白质组分析.
在Shank3B-/-小鼠中用鼻腔注射IPSC衍生的EV.

主要成果:

来自SHANK3突变神经元的EV转移了过敏性和加速成熟以控制神经元.
蛋白质组学揭示了突变EVs丰富的突触结构调节器,与细胞骨失调一致.
来自介酶干细胞 (MSC) 和健康的iPSC的EVs在突变神经元中恢复了过度刺激和正常成熟.
在Shank3B-/-小鼠中,鼻内给药iPSC-EV显著挽救了类似ASD的行为.

结论:

在SHANK3缺乏症中,EVs介导神经元表型的细胞间转移.
来自MSC和iPSC的EV具有通过调节神经元刺激和成熟来治疗ASD的治疗潜力.
基于EV的疗法为与SHANK3突变相关的神经发育障碍提供了一个有希望的策略.

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