CRISPR-Cas9 therapeutics in cancer: promising strategies and present challenges

Biochim Biophys Acta. 2016 Dec;1866(2):197-207. doi: 10.1016/j.bbcan.2016.09.002. Epub 2016 Sep 15.


Cancer is characterized by multiple genetic and epigenetic alterations that drive malignant cell proliferation and confer chemoresistance. The ability to correct or ablate such mutations holds immense promise for combating cancer. Recently, because of its high efficiency and accuracy, the CRISPR-Cas9 genome editing technique has been widely used in cancer therapeutic explorations. Several studies used CRISPR-Cas9 to directly target cancer cell genomic DNA in cellular and animal cancer models which have shown therapeutic potential in expanding our anticancer protocols. Moreover, CRISPR-Cas9 can also be employed to fight oncogenic infections, explore anticancer drugs, and engineer immune cells and oncolytic viruses for cancer immunotherapeutic applications. Here, we summarize these preclinical CRISPR-Cas9-based therapeutic strategies against cancer, and discuss the challenges and improvements in translating therapeutic CRISPR-Cas9 into clinical use, which will facilitate better application of this technique in cancer research. Further, we propose potential directions of the CRISPR-Cas9 system in cancer therapy.

Trial registration: NCT02793856 NCT02867345 NCT02863913 NCT02867332.

Keywords: CRISPR-Cas9; Cancer genome manipulation; Cancer therapy; Gene therapy; Genome editing.

Publication types

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

MeSH terms

  • Animals
  • CRISPR-Cas Systems* / physiology
  • Epigenesis, Genetic
  • Gene Editing
  • Humans
  • Immunotherapy
  • Molecular Targeted Therapy
  • Neoplasms / genetics
  • Neoplasms / therapy*
  • Oncolytic Virotherapy

Associated data