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Base editing and prime editing in laboratory animals.

Federico Caso1, Benjamin Davies1

  • 1Wellcome Centre for Human Genetics, 6396University of Oxford, UK.

Laboratory Animals
|February 18, 2021
PubMed
Summary

Genome editing technologies like CRISPR have advanced laboratory animal research. Newer methods, base editing and prime editing, offer improved precision and broader applications for genetic engineering in animals.

Keywords:
Animal modelsCRISPRadenine base editingbase editingcytosine base editinggene therapyprime editingtransgenesis

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Area of Science:

  • Genetics
  • Molecular Biology
  • Bioengineering

Background:

  • Programmable RNA-dependent Cas endonucleases revolutionized genome engineering.
  • Conventional CRISPR editing induces DNA double-strand breaks (DSBs), raising precision concerns.
  • Iterations like base editing were developed to improve editing precision without DSBs.

Purpose of the Study:

  • To review advancements in genome editing technologies for laboratory animal research.
  • To highlight the precision and applications of base editing and prime editing.
  • To discuss the potential of new genome editing tools in animal models and gene therapy.

Main Methods:

  • Review of base editing (cytosine and adenine) applications in animal models.
  • Analysis of prime editing as a 'search and replace' genome manipulation tool.
  • Examination of genome editing precision and limitations in laboratory animal research.

Main Results:

  • Base editing enables precise single base conversions without DSBs, successfully applied in zygotes and somatic cells.
  • Cytosine and adenine base editors facilitate animal model generation and gene therapy development.
  • Prime editing offers potential for wider genomic manipulations beyond base editing's scope.

Conclusions:

  • Base editing provides precise genome editing for animal models and gene therapy.
  • Prime editing represents a significant advancement, potentially overcoming limitations of previous methods.
  • These genome editing tools are crucial for future laboratory animal research and therapeutic development.