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Nat Methods. 2013 Oct;10(10):977-9. doi: 10.1038/nmeth.2598. Epub 2013 Jul 25.

CRISPR RNA-guided activation of endogenous human genes.

Author information

1
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.

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.

PMID:
23892898
PMCID:
PMC3794058
DOI:
10.1038/nmeth.2598
[Indexed for MEDLINE]
Free PMC Article

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