CRISPR/Cas9 for cancer treatment: technology, clinical applications and challenges

Brief Funct Genomics. 2020 May 20;19(3):209-214. doi: 10.1093/bfgp/elaa001.

Abstract

Clustered regularly interspaced short palindromic repeats (CRISPR) is described as RNA mediated adaptive immune system defense, which is naturally found in bacteria and archaea. CRISPR-Cas9 has shown great promise for cancer treatment in cancer immunotherapy, manipulation of cancer genome and epigenome and elimination or inactivation of carcinogenic viral infections. However, many challenges remain to be addressed to increase its efficacy, including off-target effects, editing efficiency, fitness of edited cells, immune response and delivery methods. Here, we explain CRISPR-Cas classification and its general function mechanism for gene editing. Then, we summarize these preclinical CRISPR-Cas9-based therapeutic strategies against cancer. Moreover, the challenges and improvements of CRISPR-Cas9 clinical applications will be discussed.

Keywords: CRISPR/Cas9; application; cancer treatment; challenge; technology.

Publication types

  • Review

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics*
  • Clustered Regularly Interspaced Short Palindromic Repeats / genetics*
  • Genetic Therapy / methods
  • Humans
  • Neoplasms / genetics*