Single-nucleotide editing: From principle, optimization to application

Hum Mutat. 2019 Dec;40(12):2171-2183. doi: 10.1002/humu.23819. Epub 2019 Sep 15.

Abstract

Cytosine base editors (CBEs) and adenine base editors (ABEs), which are generally composed of an engineered deaminase and a catalytically impaired CRISPR-Cas9 variant, are new favorite tools for single base substitution in cells and organisms. In this review, we summarize the principle of base-editing systems and elaborate on the evolution of different platforms of CBEs and ABEs, including their deaminase, Cas9 variants, and editing outcomes. Moreover, we highlight their applications in mouse and human cells and discuss the challenges and prospects of base editors. The ABE- and CBE systems have been used in gene silencing, pathogenic gene correction, and functional genetic screening. Single base editing is becoming a new promising genetic tool in biomedical research and gene therapy.

Keywords: CRISPR-Cas9; adenine base editors; base editing; cytosine base editors; genetic engineering.

Publication types

  • Research Support, N.I.H., Extramural
  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Aminohydrolases / metabolism*
  • Animals
  • CRISPR-Cas Systems
  • Cytosine Deaminase / metabolism*
  • Gene Editing / methods*
  • Gene Silencing
  • Humans
  • Mice
  • Polymorphism, Single Nucleotide

Substances

  • Aminohydrolases
  • Cytosine Deaminase
  • adenine deaminase