Target specificity of the CRISPR-Cas9 system.

ABSTRACT: 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.

SUBMITTER: Wu X 

PROVIDER: S-EPMC4338555 | biostudies-literature | 2014 Jun

REPOSITORIES: biostudies-literature