CRISPR RNA–guided activation of endogenous human genes

ML Maeder, SJ Linder, VM Cascio, Y Fu, QH Ho… - Nature …, 2013 - nature.com
ML Maeder, SJ Linder, VM Cascio, Y Fu, QH Ho, JK Joung
Nature methods, 2013nature.com
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.
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.
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