CRISPR RNA-guided activation of endogenous human genes.
basic_science · Level V
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- Record sourced from PubMed, PMID 23892898.
- Also identified by DOI 10.1038/nmeth.2598 and PMC identifier 3794058.
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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.
Medical subject headings
- Clustered Regularly Interspaced Short Palindromic Repeats
- RNA Editing
- Recombinant Fusion Proteins
- Transcriptional Activation
- Vascular Endothelial Growth Factor A