一种荧光报告小鼠，用于在体内评估使用多种 Cas 核酸酶和主编辑器进行基因组编辑的效果。

IF 3.7 4区生物学 Q2 GENETICS & HEREDITY

CRISPR Journal Pub Date : 2023-12-01 DOI:10.1089/crispr.2023.0048

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

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引用次数: 0

摘要

基于 CRISPR 的基因组编辑技术（包括核酸酶编辑、碱基编辑和质粒编辑）最近彻底改变了针对致病突变的疗法的开发。为了推动基因组编辑工具的评估和开发，一个强大的小鼠模型是非常有价值的，尤其是在评估体内活性和递送策略方面。在这项研究中，我们成功培育出了一种携带交通灯报告设计的基因敲入小鼠品系，即 TLR-multi-Cas变体 1（TLR-MCV1）。我们全面验证了这种小鼠模型在体外和体内核酸酶和质粒编辑方面的功能。TLR-MCV1 报告小鼠是一种多功能的强大工具，可加快编辑技术的开发及其治疗应用。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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

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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来源期刊

CRISPR Journal Biochemistry, Genetics and Molecular Biology-Biotechnology

CiteScore

6.30

自引率

2.70%

发文量

期刊介绍： In recognition of this extraordinary scientific and technological era, Mary Ann Liebert, Inc., publishers recently announced the creation of The CRISPR Journal -- an international, multidisciplinary peer-reviewed journal publishing outstanding research on the myriad applications and underlying technology of CRISPR. Debuting in 2018, The CRISPR Journal will be published online and in print with flexible open access options, providing a high-profile venue for groundbreaking research, as well as lively and provocative commentary, analysis, and debate. The CRISPR Journal adds an exciting and dynamic component to the Mary Ann Liebert, Inc. portfolio, which includes GEN (Genetic Engineering & Biotechnology News) and more than 80 leading peer-reviewed journals.