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

一种用于体内评估多种Cas核酸酶和Prime编辑器基因组编辑的荧光报告小鼠

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作者: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

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.

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