Target specificity of the CRISPR-Cas9 system.
The CRISPR-Cas9 system, naturally a defense mechanism in prokaryotes, has been repurposed as an RNA-guided DNA targeting platform. It has been widely used for genome editing and transcriptome modulation, and has shown great promise in correcting mutations in human genetic diseases. Off-target effects are a critical issue for all of these applications. Here we review the current status on the target specificity of the CRISPR-Cas9 system.
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA ; Computational and Systems Biology Graduate Program, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Xuebing
X
Wu
Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Andrea J
AJ
Kriz
David H. Koch Institute for Integrative Cancer Research, Massachusetts Institute of Technology, Cambridge, MA 02139, USA ; Department of Biology, Massachusetts Institute of Technology, Cambridge, MA 02139, USA.
Phillip A
PA
Sharp
CRISPR; Cas9; genome engineering; off-targets; target specificity