articleCell ResearchAug 27, 2013HYBRID OA

Multiplexed activation of endogenous genes by CRISPR-on, an RNA-guided transcriptional activator system

Whitehead Institute for Biomedical Research · Massachusetts Institute of Technology · +1 more institution

PubMed
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Abstract

Technologies allowing for specific regulation of endogenous genes are valuable for the study of gene functions and have great potential in therapeutics. We created the CRISPR-on system, a two-component transcriptional activator consisting of a nuclease-dead Cas9 (dCas9) protein fused with a transcriptional activation domain and single guide RNAs (sgRNAs) with complementary sequence to gene promoters. We demonstrate that CRISPR-on can efficiently activate exogenous reporter genes in both human and mouse cells in a tunable manner. In addition, we show that robust reporter gene activation in vivo can be achieved by injecting the system components into mouse zygotes. Furthermore, we show that CRISPR-on can…

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