Targeted genome engineering in human cells with the Cas9 RNA-guided endonuclease

Abstract

The genome of human cells is edited using the bacterial RNA-guided Cas9 endonuclease. We employ the CRISPR-Cas system of Streptococcus pyogenes as programmable RNA-guided endonucleases (RGENs) to cleave DNA in a targeted manner for genome editing in human cells. We show that complexes of the Cas9 protein and artificial chimeric RNAs efficiently cleave two genomic sites and induce indels with frequencies of up to 33%.