CRISPR RNA 引导的内源性人类基因激活。
CRISPR RNA-guided activation of endogenous human genes.
机构信息
1] Molecular Pathology Unit, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [2] Center for Computational and Integrative Biology, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [3] Center for Cancer Research, Massachusetts General Hospital, Charlestown, Massachusetts, USA. [4] Program in Biological and Biomedical Sciences, Harvard Medical School, Boston, Massachusetts, USA.
出版信息
Nat Methods. 2013 Oct;10(10):977-9. doi: 10.1038/nmeth.2598. Epub 2013 Jul 25.
Short guide RNAs (gRNAs) can direct catalytically inactive CRISPR-associated 9 nuclease (dCas9) to repress endogenous genes in bacteria and human cells. Here we show that single or multiple gRNAs can direct dCas9 fused to a VP64 transcriptional activation domain to increase expression of endogenous human genes. This proof-of-principle work shows that clustered regularly interspaced short palindromic repeat (CRISPR)-Cas systems can target heterologous effector domains to endogenous sites in human cells.
短向导 RNA（gRNAs）可将无催化活性的 CRISPR 相关 9 核酸酶（dCas9）引导至细菌和人类细胞中抑制内源性基因。本文作者展示了单个或多个 gRNAs 可引导融合 VP64 转录激活结构域的 dCas9 增加内源性人类基因的表达。该原理验证性研究表明，成簇规律间隔短回文重复序列（CRISPR）-Cas 系统可将异源效应结构域靶向人类细胞中的内源性靶位。
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