Center for Precision Genome Editing and Genetic Technologies for Biomedicine, Institute of Gene Biology, Russian Academy of Sciences, Moscow, Russia, 119334.
Federal State Budgetary Institution "National Medical Research Center of Oncology Named After N.N. Blokhin" of the Ministry of Health of the Russian Federation, Research Institute of Carcinogenesis, Moscow, Russia, 115478.
Mol Biol Rep. 2024 Jan 18;51(1):135. doi: 10.1007/s11033-023-09066-0.
Modeling a human disease is an essential part of biomedical research. The recent advances in the field of molecular genetics made it possible to obtain genetically modified animals for the study of various diseases. Not only monogenic disorders but also chromosomal and multifactorial disorders can be mimicked in lab animals due to genetic modification. Even human infectious diseases can be studied in genetically modified animals. An animal model of a disease enables the tracking of its pathogenesis and, more importantly, to test new therapies. In the first part of this paper, we review the most common DNA modification technologies and provide key ideas on specific technology choices according to the task at hand. In the second part, we focus on the application of genetically modified mice in studying human diseases.
建立人类疾病模型是生物医学研究的重要组成部分。分子遗传学领域的最新进展使得研究各种疾病的基因修饰动物成为可能。不仅单基因疾病，而且染色体和多因素疾病也可以通过遗传修饰在实验动物中模拟。即使是人类传染病也可以在基因修饰动物中进行研究。疾病的动物模型能够追踪其发病机制，更重要的是，可以测试新的治疗方法。本文第一部分回顾了最常见的 DNA 修饰技术，并根据手头的任务提供了特定技术选择的关键思路。第二部分重点介绍了基因修饰小鼠在研究人类疾病中的应用。
