title: 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.
authors.affiliation: 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.
authors.forename: Morgan L
authors.initials: ML
authors.lastname: Maeder
authors.forename: Samantha J
authors.initials: SJ
authors.lastname: Linder
authors.forename: Vincent M
authors.initials: VM
authors.lastname: Cascio
authors.forename: Yanfang
authors.initials: Y
authors.lastname: Fu
authors.forename: Quan H
authors.initials: QH
authors.lastname: Ho
authors.forename: J Keith
authors.initials: JK
authors.lastname: Joung
mesh_terms: D001426:Bacterial Proteins / Q000235:genetics; D064112:Clustered Regularly Interspaced Short Palindromic Repeats; D057809:HEK293 Cells; D006801:Humans; D017393:RNA Editing; D011993:Recombinant Fusion Proteins / Q000235:genetics*; D012260:Ribonucleases / Q000235:genetics; D013297:Streptococcus pyogenes / Q000235:genetics; D015533:Transcriptional Activation; D042461:Vascular Endothelial Growth Factor A / Q000235:genetics*; D058727:RNA, Small Untranslated
chemical_list: D001426:Bacterial Proteins; D011993:Recombinant Fusion Proteins; C467484:VEGFA protein, human; D042461:Vascular Endothelial Growth Factor A; D012260:Ribonucleases; D058727:RNA, Small Untranslated
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