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.National Institutes of Health (U.S.) (United States Public Health Service Grant RO1-GM34277)National Institutes of Health (U.S.) (United States Public Health Service Grant R01-CA133404)National Cancer Institute (U.S.) (Grant PO1-CA42063)National Cancer Institute (U.S.) (Koch Institute Support (Core) Grant P30-CA14051