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在生物中通过CRISPR/Cas9介导的跨表观基因调节激活目标基因

Hsin-Kai Liao¹, Fumiyuki Hatanaka¹, Toshikazu Araoka²

¹Gene Expression Laboratory, Salk Institute for Biological Studies, La Jolla, CA 92037, USA.

|December 12, 2017

概括

这项研究引入了一种新的CRISPR/Cas9系统,通过表观遗传改造激活基因,绕过DNA双链断裂 (DSB). 通过调节基因表达而没有有害突变, 这一突破为治疗遗传疾病提供了更安全的方法.

科学领域:

分子生物学
表观遗传学
基因治疗

背景情况:

目前的基因组编辑技术通常依赖于DNA双链断裂 (DSB),这可能导致不必要的突变并限制临床应用.
CRISPR/Cas9已经适应了没有DSB的基因激活,但在体内实施仍然具有挑战性.

研究的目的:

通过跨表观遗传改造开发一种强大的体内目标基因激活系统.
在临床前疾病模型中证明该系统的有效性.

主要方法:

通过修改的单导 RNA 来招募 Cas9 和转录激活复合体来准 DNA 位置.
在糖尿病,肌肉衰竭和急性病的小鼠模型中应用该系统.

主要成果:

使用开发的CRISPR/ Cas9系统成功激活了内源性基因.
在治疗小鼠模型中观察到可测量的表型改善和疾病症状的改善.

结论:

这种新型的CRISPR/Cas9基因激活方法为体内基因调节的DSB诱导方法提供了有希望的替代方案.
这项技术为开发针对各种人类疾病的表观遗传疗法创造了新的可能性.

关键词:

在CRISPR/Cas9中染色体重塑疾病模型表观遗传修饰表观遗传疗法基因编辑肌肉发育不良复原医学干细胞转变差异化

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