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Related Experiment Video

Updated: Feb 8, 2026

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes

Efficient Genome Editing of Mice by CRISPR Electroporation of Zygotes

Published on: December 16, 2022

Optimized base editors enable efficient editing in cells, organoids and mice.

Maria Paz Zafra¹, Emma M Schatoff^1,2, Alyna Katti^1,3

¹Sandra and Edward Meyer Cancer Center, Department of Medicine, Weill Cornell Medicine, New York, New York, USA.

Nature Biotechnology

|July 4, 2018

Summary

This study optimized CRISPR base editors (BEs) for higher efficiency in gene editing. The enhanced base editors successfully created single-nucleotide variants in various cell types and in vivo.

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Area of Science:

Molecular Biology
Gene Editing Technologies
Biotechnology

Background:

CRISPR base editing allows precise DNA modifications without double-strand breaks.
Current base editors exhibit low efficiency in many cell types, limiting their application.
Efficient gene editing tools are crucial for genetic research and therapeutic development.

Purpose of the Study:

To reengineer existing base editors (BE3, BE4Gam, xBE3) to enhance their efficiency.
To develop optimized constitutive and inducible base-editing vector systems.
To demonstrate the broad applicability of these improved editors in various cell types and in vivo.

Main Methods:

Codon optimization of base editor sequences.
Incorporation of additional nuclear-localization sequences.
Testing of engineered base editors in diverse mouse and human cell lines, intestinal organoids, and in vivo liver models.

Main Results:

The reengineered base editors significantly improved the efficiency of single-nucleotide variant creation.
Successful target modification was achieved across a wide range of cell lines and organoids.
Efficient in vivo somatic editing was demonstrated in the livers of adult mice.

Conclusions:

Optimized base editors offer a substantial improvement in gene editing efficiency.
These enhanced systems are effective in various cellular contexts and in vivo.
The developed vector systems provide a powerful tool for creating single-nucleotide variants in research and potentially therapeutic applications.