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CRISPR RNA-guided activation of endogenous human genes.

ABSTRACT: 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.

SUBMITTER: Maeder ML

PROVIDER: S-EPMC3794058 | biostudies-literature | 2013 Oct

REPOSITORIES: biostudies-literature

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CRISPR RNA-guided activation of endogenous human genes.

Maeder Morgan L ML Linder Samantha J SJ Cascio Vincent M VM Fu Yanfang Y Ho Quan H QH Joung J Keith JK

Nature methods 20130725 10

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 ...[more]

PMID: 23892898

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