Vasil'eva E A, Melino D, Barlev N A
Tsitologiia. 2015;57(1):19-30.
Genome editing systems based on site-specific nucleases became very popular for genome editing in modern bioengineering. Human pluripotent stem cells provide a unique platform for genes function study, disease modeling, and drugs testing. Consequently, technology for fast, accurate and well controlled genome manipulation is required. CRISPR/Cas (clustered regularly interspaced short palindromic repeat/CRISPR-associated) system could be employed for these purposes. This system is based on site-specific programmable nuclease Cas9. Numerous advantages of the CRISPR/Cas system and its successful application to human stem cells provide wide opportunities for genome therapy and regeneration medicine. In this publication, we describe and compare the main genome editing systems based on site-specific programmable nucleases and discuss opportunities and perspectives of the CRISPR/Cas system for application to pluripotent stem cells.
基于位点特异性核酸酶的基因组编辑系统在现代生物工程的基因组编辑中变得非常流行。人类多能干细胞为基因功能研究、疾病建模和药物测试提供了一个独特的平台。因此，需要快速、准确且可控性良好的基因组操作技术。CRISPR/Cas（成簇规律间隔短回文重复序列/CRISPR相关蛋白）系统可用于这些目的。该系统基于位点特异性可编程核酸酶Cas9。CRISPR/Cas系统的众多优势及其在人类干细胞中的成功应用为基因组治疗和再生医学提供了广泛的机会。在本出版物中，我们描述并比较了基于位点特异性可编程核酸酶的主要基因组编辑系统，并讨论了CRISPR/Cas系统应用于多能干细胞的机会和前景。
