Molecular Cancer Research, Center for Molecular Medicine, University Medical Center Utrecht, Utrecht University, The Netherlands.
Oncode Institute, The Netherlands.
Nucleic Acids Res. 2018 Jul 27;46(13):6435-6454. doi: 10.1093/nar/gky571.
Model systems with defined genetic modifications are powerful tools for basic research and translational disease modelling. Fortunately, generating state-of-the-art genetic model systems is becoming more accessible to non-geneticists due to advances in genome editing technologies. As a consequence, solely relying on (transient) overexpression of (mutant) effector proteins is no longer recommended since scientific standards increasingly demand genetic modification of endogenous loci. In this review, we provide up-to-date guidelines with respect to homology-directed repair (HDR)-mediated editing of mammalian model systems, aimed at assisting researchers in designing an efficient genome editing strategy.
具有明确遗传修饰的模型系统是基础研究和转化疾病建模的有力工具。幸运的是，由于基因组编辑技术的进步，非遗传学家也越来越容易生成最先进的遗传模型系统。因此，仅仅依靠（瞬时）过表达（突变）效应蛋白不再被推荐，因为科学标准越来越要求对内源性基因座进行遗传修饰。在这篇综述中，我们提供了关于哺乳动物模型系统同源定向修复（HDR）介导编辑的最新指南，旨在帮助研究人员设计有效的基因组编辑策略。
