Exploring the potential of genome editing CRISPR-Cas9 technology

Gene. 2017 Jan 30;599:1-18. doi: 10.1016/j.gene.2016.11.008. Epub 2016 Nov 9.


CRISPR-Cas9 is an RNA-mediated adaptive immune system that protects bacteria and archaea from viruses or plasmids. Herein we discuss the recent development of CRISPR-Cas9 into a key technology for genome editing, targeting, and regulation in a wide range of organisms and cell types. It requires a custom designed single guide-RNA (sgRNA), a Cas9 endonuclease, and PAM sequences in the target region. The sgRNA-Cas9 complex binds to its target and creates a double-strand break (DSB) that can be repaired by non-homologous end joining (NHEJ) or by the homology-directed repair (HDR) pathway, modifying or permanently replacing the genomic target sequence. Additionally, we highlight recent advances in the repurposing of CRISPR-Cas9 for repression, activation, and loci imaging. In this review, we underline the current progress and the future potential of the CRISPR-Cas9 system towards biomedical, therapeutic, industrial, and biotechnological applications.

Keywords: Activation; CRISPR-Cas9; CRISPRi; Gene therapy; Genome editing; Indel; Repression; sgRNA.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Chromosome Mapping
  • Gene Editing / ethics
  • Gene Editing / methods*
  • Gene Editing / trends
  • Gene Targeting
  • Humans
  • Transcription, Genetic