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通过RNA向Cas9消除有毒的微卫星重复扩张RNA

Ranjan Batra¹, David A Nelles¹, Elaine Pirie¹

¹Department of Cellular and Molecular Medicine, University of California at San Diego, La Jolla, CA, USA; Stem Cell Program, University of California at San Diego, La Jolla, CA, USA; Institute for Genomic Medicine, University of California at San Diego, La Jolla, CA, USA.

|August 15, 2017

概括

科学家开发了一种CRISPR系统来准并消除重复扩散疾病中的毒性RNA. 这种向RNA的Cas9 (RCas9) 系统显示出诊断和治疗肌性缩和ALS等疾病的潜力.

科学领域:

遗传学
分子生物学
生物技术

背景情况:

在DNA中的微卫星重复扩张导致病原性RNA物种.
这些有毒RNA会导致严重的主导性遗传神经疾病,包括肌性缩 (DM1/2),亨廷顿病和C9orf72相关的肌缩侧面硬化症 (C9-ALS).
迫切需要针对这些重复RNA的有效诊断和治疗策略.

研究的目的:

开发一种可编程的CRISPR系统,专门用于可视化和消除有毒的重复扩张RNA.
评估这种针对RNA的CRISPR系统在疾病模型和患者细胞中的有效性.

主要方法:

开发一种可编程的CRISPR系统,称为RNA向Cas9 (RCas9).
在外源表达系统和患者衍生细胞中测试RCas9的特定向和消除微卫星重复扩张RNA.
对RCas9逆转疾病特异性的分子和细胞表型的能力的评估.

主要成果:

RCas9专门向并有效消除有毒的重复扩张RNA.
在多种疾病模型中观察到RNA焦点的消除 (DM1,DM2,C9-ALS,多重质胺疾病).
已证明可减少有毒蛋白质产物,恢复蛋白质局部化,并扭转患者细胞的拼接缺陷.

结论:

开发的RCas9系统有效地准并消除导致重复扩张疾病的致病RNA.
在治疗一系列衰弱的遗传疾病方面,RCas9具有显著的治疗潜力.
成功开发了一种适用于腺相关病毒载体的截断RCas9变体,从而提高了治疗前景.

关键词:

美国关于CRISPR的研究亨廷顿病针对RNA的Cas9 腺相关病毒基因疗法微卫星的重复扩张最低的Cas9 肌性变质症

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