Improving Precise CRISPR Genome Editing by Small Molecules: Is there a Magic Potion?

Cells. 2020 May 25;9(5):1318. doi: 10.3390/cells9051318.

Abstract

Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) genome editing has become a standard method in molecular biology, for the establishment of genetically modified cellular and animal models, for the identification and validation of drug targets in animals, and is heavily tested for use in gene therapy of humans. While the efficiency of CRISPR mediated gene targeting is much higher than of classical targeted mutagenesis, the efficiency of CRISPR genome editing to introduce defined changes into the genome is still low. Overcoming this problem will have a great impact on the use of CRISPR genome editing in academic and industrial research and the clinic. This review will present efforts to achieve this goal by small molecules, which modify the DNA repair mechanisms to facilitate the precise alteration of the genome.

Keywords: CRISPR efficiency; homology directed repair; low molecular weight compounds.

Publication types

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

MeSH terms

  • Animals
  • CRISPR-Cas Systems / genetics*
  • Cell Cycle / genetics
  • DNA Damage / genetics
  • DNA Repair / genetics
  • Gene Editing*
  • Humans
  • Small Molecule Libraries / metabolism*

Substances

  • Small Molecule Libraries