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CRISPR RNA-guided activation of endogenous human genes

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2013

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Maeder, Morgan L, Samantha J Linder, Vincent M Cascio, Yanfang Fu, Quan H Ho, and J Keith Joung. 2013. “CRISPR RNA-guided activation of endogenous human genes.” Nature methods 10 (10): 977-979. doi:10.1038/nmeth.2598. http://dx.doi.org/10.1038/nmeth.2598.

Abstract

Catalytically inactive CRISPR-associated 9 nuclease (dCas9) can be directed by short guide RNAs (gRNAs) to repress endogenous genes in bacteria and human cells. Here we show that a dCas9-VP64 transcriptional activation domain fusion protein can be directed by single or multiple gRNAs to increase expression of specific endogenous human genes. These results provide an important proof-of-principle that CRISPR-Cas systems can be used to target heterologous effector domains in human cells.

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