A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors

  • Zexiang Chen
  • , Suet Yan Kwan
  • , Aamir Mir
  • , Max Hazeltine
  • , Minwook Shin
  • , Shun Qing Liang
  • , Io Long Chan
  • , Karen Kelly
  • , Krishna S. Ghanta
  • , Nicholas Gaston
  • , Yueying Cao
  • , Jun Xie
  • , Guangping Gao
  • , Wen Xue
  • , Erik J. Sontheimer
  • , Jonathan K. Watts

Research output: Contribution to journalArticlepeer-review

5 Scopus citations

Abstract

CRISPR-based genome-editing technologies, including nuclease editing, base editing, and prime editing, have recently revolutionized the development of therapeutics targeting disease-causing mutations. To advance the assessment and development of genome editing tools, a robust mouse model is valuable, particularly for evaluating in vivo activity and delivery strategies. In this study, we successfully generated a knock-in mouse line carrying the Traffic Light Reporter design known as TLR-multi-Cas variant 1 (TLR-MCV1). We comprehensively validated the functionality of this mouse model for both in vitro and in vivo nuclease and prime editing. The TLR-MCV1 reporter mouse represents a versatile and powerful tool for expediting the development of editing technologies and their therapeutic applications.

Original languageEnglish (US)
Pages (from-to)570-582
Number of pages13
JournalCRISPR Journal
Volume6
Issue number6
DOIs
StatePublished - Dec 1 2023
Externally publishedYes

Bibliographical note

Publisher Copyright:
© Mary Ann Liebert, Inc.

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