A Fluorescent Reporter Mouse for <i>In Vivo</i> Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editorsopen access
- Authors
- Chen, Zexiang; Kwan, Suet-Yan; Mir, Aamir; Hazeltine, Max; Shin, Minwook; Liang, Shun-Qing; Chan, Io Long; Kelly, Karen; Ghanta, Krishna S.; Gaston, Nicholas; Cao, Yueying; Xie, Jun; Gao, Guangping; Xue, Wen; Sontheimer, Erik J.; Watts, Jonathan K.
- Issue Date
- Dec-2023
- Publisher
- MARY ANN LIEBERT, INC
- Citation
- CRISPR JOURNAL, v.6, no.6, pp 570 - 582
- Pages
- 13
- Journal Title
- CRISPR JOURNAL
- Volume
- 6
- Number
- 6
- Start Page
- 570
- End Page
- 582
- URI
- https://scholarworks.sookmyung.ac.kr/handle/2020.sw.sookmyung/159608
- DOI
- 10.1089/crispr.2023.0048
- ISSN
- 2573-1599
2573-1602
- 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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