CRISPR/Cas9 Gene Editing: From Basic Mechanisms to Improved Strategies for Enhanced Genome Engineering In Vivo

Curr Gene Ther. 2017;17(4):263-274. doi: 10.2174/1566523217666171122094629.


Introduction: Targeted genome editing using the CRISPR/Cas9 technology is becoming a major area of research due to its high potential for the treatment of genetic diseases. Our understanding of this approach has expanded in recent years yet several new challenges have presented themselves as we explore the boundaries of this exciting new technology. Chief among these is improving the efficiency but also the preciseness of genome editing. The efficacy of CRISPR/Cas9 technology relies in part on the use of one of the major DNA repair pathways, Homologous recombination (HR), which is primarily active in S and G2 phases of the cell cycle. Problematically, the HR potential is highly variable from cell type to cell type and most of the cells of interest to be targeted in vivo for precise genome editing are in a quiescent state.

Conclusion: In this review, we discuss the recent advancements in improving targeted CRISPR/Cas9 based genome editing and the promising ways of delivering this technology in vivo to the cells of interest.

Keywords: CRISPR-Cas9; DNA repair; Gene editing; Homologous recombination; Non-homologous end-joining.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Gene Editing / methods*
  • Gene Editing / trends
  • Genetic Engineering / methods*
  • Genetic Engineering / trends
  • Genetic Therapy / methods
  • Genetic Therapy / trends
  • Genome / genetics*
  • Humans
  • Translational Research, Biomedical / methods*
  • Translational Research, Biomedical / trends