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Single-nucleotide editing: From principle, optimization to application.

Jinling Tang1, Trevor Lee2, Tao Sun1

  • 1Center for Precision Medicine, School of Medicine and School of Biomedical Sciences, Huaqiao University, Xiamen, Fujian, China.

Human Mutation
|May 28, 2019
PubMed
Summary
This summary is machine-generated.

Base editors, including cytosine (CBEs) and adenine (ABEs) variants, enable precise single base substitutions. These powerful tools are revolutionizing gene editing for research and therapeutic applications.

Keywords:
CRISPR-Cas9adenine base editorsbase editingcytosine base editorsgenetic engineering

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Area of Science:

  • Molecular Biology
  • Gene Editing Technologies

Background:

  • Cytosine base editors (CBEs) and adenine base editors (ABEs) are advanced gene editing tools.
  • They utilize engineered deaminases fused to catalytically impaired CRISPR-Cas9 variants.
  • These systems facilitate precise single base substitutions in genomic DNA.

Purpose of the Study:

  • To review the principles and evolution of CBE and ABE platforms.
  • To summarize their deaminase components, Cas9 variants, and editing outcomes.
  • To highlight applications and discuss future prospects of base editors.

Main Methods:

  • Review of existing literature on base editing systems.
  • Analysis of different CBE and ABE platforms, including their components and performance.
  • Compilation of reported applications in various cell types and organisms.

Main Results:

  • Detailed overview of CBE and ABE system evolution and mechanisms.
  • Summary of diverse applications, including gene silencing, pathogenic gene correction, and genetic screening.
  • Identification of key challenges and future directions for base editing technology.

Conclusions:

  • Base editing systems offer a promising approach for precise genetic modifications.
  • Their applications are expanding in biomedical research and hold potential for gene therapy.
  • Continued development is expected to enhance their efficiency and broaden their utility.