Journal
NATURE METHODS
Volume 10, Issue 10, Pages 977-+Publisher
NATURE PUBLISHING GROUP
DOI: 10.1038/NMETH.2598
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Funding
- US National Institutes of Health (NIH) [DP1 GM105378, NIH R01 NS073124, NIH P50 HG005550]
- Defense Advanced Research Projects Agency (DARPA) [W911NF-11-2-0056]
- Jim and Ann Orr Massachusetts General Hospital Research Scholar Award
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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.
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