CRISPR/Cas9 technology as a potent molecular tool for gene therapy

J Cell Physiol. 2019 Aug;234(8):12267-12277. doi: 10.1002/jcp.27972. Epub 2019 Jan 30.

Abstract

Clustered regularly interspaced short palindromic repeats/CRISPR-associated nuclease 9 (CRISPR-Cas9) is an RNA-guided gene editing tool which offers several advantageous characteristics in comparison with the conventional methods (e.g., zinc finger nucleases and transcription activator-like effector nucleases) such as cost-effectiveness, flexibility, and being easy-to-use. Despite some limitations such as efficient delivery and safety, CRISPR-Cas9 is still the most convenient tool for gene editing purposes. Due to the potential capability of the CRISPR-Cas9 system in genome editing and correction of casual mutations, it can be considered as a possible therapeutic system in the treatment of disorders associated with the genome mutations and in particular cancer treatment. In this review, we will discuss CRISPR-Cas-based gene editing along with its classifications and mechanism of action. Furthermore, the therapeutic application of the CRISPR-Cas9 system in mutational disorders, delivery systems, as well as its advantages and limitations with a special emphasis on cancer treatment will be discussed.

Keywords: CRISPR-Cas9; cancer treatment; gene therapy; genome editing.

Publication types

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

MeSH terms

  • CRISPR-Cas Systems / genetics*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Gene Editing / methods*
  • Gene Targeting / methods
  • Genetic Therapy / methods*
  • Humans
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • RNA Interference