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重新编写和定制三种重要的疾病基因组

Weimin Zhang¹, Ilona Golynker², Ran Brosh¹

¹Institute for Systems Genetics and Department of Biochemistry and Molecular Pharmacology, NYU Langone Health, New York, NY, USA.

|November 2, 2023

概括

一种名为mSwAP-In的新方法可以在小鼠中高效地重写基因组以获得更好的疾病模型. 这项技术创建了基因组重写和定制的GEMM (GREAT-GEMM),改善了人类疾病复制和治疗发展.

科学领域:

基因组学
分子生物学
基因工程

背景情况:

基因工程小鼠模型 (GEMMs) 对于研究人类疾病和开发疗法至关重要.
在小鼠中忠实地复制人类疾病仍然是一个重大挑战.
基因组学的进步强调了疾病中非编码的调节序列的重要性.

研究的目的:

在小鼠中开发高效的大规模基因组工程的方法.
创建高度信息化的,基因组重写和定制的GEMM (GREAT-GEMMs).
提高人类病理的小鼠模型的准确性.

主要方法:

在小鼠胚胎干细胞中进行基因组重写的"哺乳动物逐步切换抗生素耐药性标志物" (mSwAP-In) 方法.
大型DNA片段的代基因组重写 (高达115kb).
用大型人类基因组区域 (例如,ACE2,TMPRSS2) 人化小鼠位置.

主要成果:

证明了针对Trp53位点 (115 kb) 的高效基因组重写.
成功使小鼠ACE2位点与大型人类碎片 (116kB和180kB).
与现有模型相比,开发了一种更像人类的ACE2小鼠模型来治疗SARS-CoV-2感染.

结论:

mSwAP-In促进了高效的基因组重写,使得创建先进的GEMM成为可能.
开发的GREAT-GEMMs,特别是人性化的ACE2模型,可以改善人类疾病的复述.
mSwAP-In的多功能性支持复杂模型生成的序列基因组编写.

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