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.
Publication types
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Research Support, N.I.H., Extramural
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Research Support, Non-U.S. Gov't
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Research Support, U.S. Gov't, Non-P.H.S.
MeSH terms
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Bacterial Proteins / genetics
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Clustered Regularly Interspaced Short Palindromic Repeats*
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HEK293 Cells
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Humans
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RNA Editing*
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RNA, Small Untranslated
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Recombinant Fusion Proteins / genetics*
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Ribonucleases / genetics
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Streptococcus pyogenes / genetics
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Transcriptional Activation*
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Vascular Endothelial Growth Factor A / genetics*
Substances
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Bacterial Proteins
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Recombinant Fusion Proteins
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VEGFA protein, human
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Vascular Endothelial Growth Factor A
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Ribonucleases
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RNA, Small Untranslated