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TIGER:用于研究精确编辑器交付方法的体内基因组编辑报告小鼠

Samuel W Du^1,2, Grazyna Palczewska¹, Zhiqian Dong¹

¹Gavin Herbert Eye Institute-Robert M. Brunson Center for Translational Vision Research, Department of Ophthalmology and Visual Sciences, University of California Irvine, Irvine, CA 92697.

Proceedings of the National Academy of Sciences of the United States of America

|August 29, 2025

概括

一个新的TIGER小鼠模型可以在体内精确跟踪基因组编辑工具,如腺基编辑 (ABE) 和原始编辑 (PE). 这种报告系统加速了对各种组织的安全高效基因编辑传递技术的开发和优化.

科学领域:

分子生物学
遗传学
生物技术

背景情况:

在体内基因组编辑有望治疗遗传和环境疾病.
一个重要的挑战是安全有效地将编辑工具传递给目标组织.
需要一个多功能报告系统来评估交付技术.

研究的目的:

为评估体内基因组编辑交付开发一种模式不可知报告者小鼠模型.
能够快速,准确和可量化的评估基编辑 (ABE) 和原始编辑 (PE) 技术.

主要方法:

在生物基因组编辑报告者 (TIGER) 鼠标中,TdTomato在Polr2a位点中的突变.
使用ABE和PE恢复茄光, 表明编辑成功.
使用病毒和非病毒载体传递到眼组织 (RPE,光受体,Müller glia,状网) 和眼外组织 (肝细胞,肌肉,神经元).
使用体内/体外双光子共聚焦显微镜和光分析评估编辑效率.

主要成果:

在TIGER小鼠模型中,成功地展示了组织和细胞类型特定的编辑评估.
在多种眼细胞类型中使用各种传递载体实现了有效的ABE和PE.
通过静脉注射,由腺相关病毒 (AAV) 介导的PE在眼外组织 (包括肝脏,肌肉和大脑神经元) 中是成功的.
报告系统准确地量化了各种组织的编辑效率和传递.

结论:

TIGER小鼠是开发和优化体内基因组编辑交付平台的宝贵工具.
这种报告系统为广泛的治疗应用促进了ABE和PE的发展.
提格模型加速基因组编辑技术向临床应用转化.

关键词:

关于CRISPR的研究基础编辑基因组编辑主要编辑记者

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