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针对低水平的MIF表达作为ALS潜在的治疗策略

Leenor Alfahel¹, Thomas Gschwendtberger², Velina Kozareva³

¹Department of Physiology and Cell Biology, Faculty of Health Sciences, Ben-Gurion University of the Negev, P.O.B. 653, Beer Sheva 84105, Israel; The School of Brain Sciences and Cognition, Ben-Gurion University of the Negev, P.O.B. 653, Beer Sheva 84105, Israel.

Cell reports. Medicine

|May 4, 2024

概括

巨细胞迁移抑制因子 (MIF) 的外周给药改善了肌缩性侧面硬化症 (ALS) 的小鼠模型中的运动功能和生存率. 这种神经保护因子还可以减少炎症和错误折叠的SOD1蛋白,提供潜在的ALS疗法.

科学领域:

神经科学是一个神经科学.
遗传学是一个遗传学.
免疫学免疫学免疫学

背景情况:

肌缩侧面硬化症 (ALS) 是一种神经退行性疾病,与SOD1突变和运动神经元 (MN) 损失有关.
巨细胞迁移抑制因子 (MIF) 在脊柱MNs中发现的水平较低,但抑制了突变SOD1的错误折叠和毒性.

研究的目的:

在SOD1相关的ALS小鼠模型中研究外围巨细胞迁移抑制因子 (MIF) 的治疗潜力.
评估MIF对ALS运动功能,疾病进展,神经炎症和分子通路的影响.

主要方法:

基相关病毒 (AAV) 介导的MIF传递给成年SOD1G37R小鼠.
评估运动功能,生存,神经炎症和错误折叠的SOD1.
蛋白质组和转录组分析以确定失调的途径.
对人类ALS患者衍生的细胞和组织中MIF水平的分析.

主要成果:

在SOD1G37R小鼠中,外周MIF的使用显著改善了运动功能,延缓了疾病的进展,并延长了生存时间.
MIF治疗减少了神经炎症,减少了错误折叠的SOD1积累,并挽救了运动神经元.
蛋白质组学和转录组学揭示了MIF治疗后失调路径的纠正.
在人类家族性和偶发性ALS患者衍生的细胞和组织中观察到低MIF水平.

结论:

外围MIF输送是ALS的一个有前途的治疗策略,在体内有效调节错误折叠的SOD1.
在临床前的ALS模型中,MIF的使用显示了神经保护作用,并改善了疾病的结果.
这些发现表明,恢复MIF水平可能是ALS患者的可行的治疗方法.

关键词:

在 AAV AAV AAV 中.这就是ALS.多元期权基金和多元期权基金.这是家族性ALS.这些是iPSCs.一个错误折叠的SOD1.运动神经元运动神经元突变的SOD1病毒.突变的SOD1小鼠.偶发性ALS是一种偶发性ALS.

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