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通过使用向RNA的CRISPR效应蛋白来向ATAXIN-2来缓解TDP-43蛋白质病变

M Alejandra Zeballos C¹, Hayden J Moore¹, Tyler J Smith¹

¹Department of Bioengineering, University of Illinois Urbana-Champaign, Urbana, IL, 61801, USA.

Nature communications

|October 14, 2023

概括

向RNA的CRISPR系统,如Cas13,可以减少神经退行性疾病中TDP-43蛋白质的积累. 这种方法改善了TDP-43蛋白质病变的小鼠模型中的症状和生存率.

科学领域:

神经科学是一个神经科学.
遗传学是一个遗传学.
分子生物学分子生物学

背景情况:

包括ALS和FTD在内的TDP-43蛋白病变涉及TDP-43的错位化和聚合.
ATAXIN-2修改了TDP-43的毒性,使其成为一个潜在的治疗目标.

研究的目的:

研究向RNA的CRISPR效应器在缓解TDP-43病理方面的有效性.
评估在小鼠模型中使用CRISPR-Cas13系统准ATAXIN-2的治疗潜力.

主要方法:

使用了向RNA的CRISPR效应器 (Cas13,Cas7-11),这些效应器被编程为向ATAXIN-2.
在体内给TDP-43蛋白质病变的小鼠模型注射了一种向素-2-向的Cas13系统.
基准测试了不同的RNA向CRISPR平台,以获得全转录组特异性.

主要成果:

克里斯普尔向阿塔克辛-2抑制了TDP-43聚合和压力颗粒过境.
在体内输送改善了功能缺陷,并在小鼠模型中延长了生存期.
与其他平台相比,高保真性Cas13显示出优越的转录组范围的特异性.

结论:

向RNA的CRISPR技术,特别是向ATAXIN-2的Cas13,显示出治疗TDP-43蛋白质病变的前景.
这种方法为特征为TDP-43病理的神经退行性疾病提供了潜在的治疗策略.

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