Use of Customizable Nucleases for Gene Editing and Other Novel Applications

Genes (Basel). 2020 Aug 22;11(9):976. doi: 10.3390/genes11090976.


The development of novel genome editing tools has unlocked new opportunities that were not previously possible in basic and biomedical research. During the last two decades, several new genome editing methods have been developed that can be customized to modify specific regions of the genome. However, in the past couple of years, many newer and more exciting genome editing techniques have been developed that are more efficient, precise, and easier to use. These genome editing tools have helped to improve our understanding of genetic disorders by modeling them in cells and animal models, in addition to correcting the disease-causing mutations. Among the genome editing tools, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system has proven to be the most popular one due to its versatility and has been successfully used in a wide variety of laboratory animal models and plants. In this review, we summarize the customizable nucleases currently used for genome editing and their uses beyond the modification of genome. We also discuss the potential future applications of gene editing tools for both basic research and clinical purposes.

Keywords: embryos; genetic diseases; genome editing.

Publication types

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

MeSH terms

  • Animals
  • CRISPR-Cas Systems*
  • Endonucleases / genetics
  • Endonucleases / metabolism*
  • Gene Editing / methods*
  • Genetic Engineering*
  • Genome*
  • Humans


  • Endonucleases