A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.
A Fluorescent Reporter Mouse for In Vivo Assessment of Genome Editing with Diverse Cas Nucleases and Prime Editors.
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DOI：
10.1089/crispr.2023.0048
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发表时间：
2023-12
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通讯作者：
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;E. Sontheimer;Jonathan K. Watts
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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;E. Sontheimer;Jonathan K. Watts
基于CRISPR的基因组编辑技术，包括核酸酶编辑、碱基编辑和引物编辑，最近彻底改变了靶向致病突变的治疗方法的发展。为了推进基因组编辑工具的评估和开发，稳健的小鼠模型是有价值的，特别是对于评估体内活性和递送策略。在这项研究中，我们成功地产生了一个敲入小鼠线携带交通灯报告设计称为TLR-multi-Cas变体1（TLR-MCV 1）。我们全面验证了该小鼠模型在体外和体内核酸酶和引物编辑方面的功能。TLR-MCV 1报告小鼠代表了一种多功能和强大的工具，用于加快编辑技术及其治疗应用的开发。
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.