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在GFP上的小鼠模型用于对体内基因编辑的询问

Carla Dib^1,2, Jack A Queenan^3,4,5, Leah Swartzrock^1,2

¹Department of Pediatrics, Division of Hematology, Oncology, Stem Cell Transplantation and Regenerative Medicine, Stanford University School of Medicine, Stanford, CA, USA.

Nature communications

|July 31, 2025

概括

一个新的GFP-on记者小鼠模型可以快速评估体内基因编辑工具. 该系统为基因编辑技术和遗传疾病治疗的传递方法提供了高通量选的便利.

科学领域:

分子生物学分子生物学
遗传学是一个遗传学.
生物技术是生物技术.

背景情况:

在体内基因编辑对遗传疾病具有治疗的前景.
为目标细胞编辑提供高效的交付工具仍然是一个重大挑战.
需要一个系统基因编辑的快速评估系统.

研究的目的:

开发和验证一种新的报告者小鼠模型,用于评估体内基因编辑系统.
在报告者模型中评估通过腺相关病毒 (AAV9) 传递的腺基编辑器 (ABE) 的疗效.

主要方法:

开发了GFP上的记者小鼠,在EGFP中具有可纠正的无意义突变.
使用编码ABE8e和sgRNA的腺相关病毒血清型9 (AAV9) 进行验证.
在成年和胎儿GFP-on小鼠中,AAV9-ABE8e-sgRNA的全身和肝内输送.

主要成果:

腺基编辑器 (ABE) 成功纠正了GFP在GFP上的小鼠中的EGFP突变.
在静脉注射后,EGFP表达与AAV9热带性相关.
在胎儿小鼠的肝内分娩恢复了EGFP在目标器官中的表达至少六个月.

结论:

在GFP上的记者小鼠模型对于评估体内基因编辑是有效的.
该模型支持对新型基因编辑工具和传递策略的高通量评估.
该系统推动了基因编辑疗法用于遗传疾病的开发.

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