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Updated: Dec 30, 2025

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
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Efficient base editing with high precision in rabbits using YFE-BE4max.

Zhiquan Liu1, Siyu Chen1, Huanhuan Shan1

  • 1Key Laboratory of Zoonosis Research, Ministry of Education, College of Animal Science, Jilin University, Changchun, 130062, China.

Cell Death & Disease
|January 22, 2020
PubMed
Summary
This summary is machine-generated.

A new base editor, YFE-BE4max, offers high efficiency and precision for C-to-T conversion in rabbits. This tool precisely models human diseases by enabling targeted gene mutations.

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Area of Science:

  • Molecular Biology
  • Gene Editing
  • Biotechnology

Background:

  • Cytidine base editors facilitate C-to-T conversion but face efficiency-precision trade-offs.
  • Existing base editors require improvement for precise genome editing applications.

Purpose of the Study:

  • To develop a novel base editor with enhanced precision and maintained efficiency.
  • To create a tool for precise modeling of human genetic diseases in rabbits.

Main Methods:

  • Rational mutation of the cytidine deaminase domain.
  • Development and application of the YFE-BE4max base editor in rabbits.
  • Generation of specific mutations (Tyr p. Q68Stop, Lmna p. G607G) to model human diseases.

Main Results:

  • YFE-BE4max narrowed the editing width to approximately three nucleotides with high efficiency.
  • Successfully mediated Tyr p. Q68Stop and Lmna p. G607G mutations in F0 rabbits.
  • Precisely recapitulated human diseases Oculocutaneous Albinism type 1 (OCA1) and Hutchinson-Gilford Progeria Syndrome (HGPS).

Conclusions:

  • YFE-BE4max provides a precise and efficient base editing tool for rabbit models.
  • Enhances the capacity for accurate modeling of human genetic diseases.
  • Offers a promising system for advanced gene editing applications.