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首页> 外文期刊>Journal of Cellular Physiology >Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes
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Programmable Site-Specific Nucleases for Targeted Genome Engineering in Higher Eukaryotes

机译：可编程的特有的核酸酶来的目标高等真核生物基因组工程

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摘要

Recent advances in the targeted genome engineering enable molecular biologists to generate sequence specific modifications with greater efficiency and higher specificity in complex eukaryotic genomes. Programmable site-specific DNA cleavage reagents and cellular DNA repair mechanisms have made this possible. These reagents have become powerful tools for delivering a site-specific genomic double-strand break (DSB) at the desired chromosomal locus, which produces sequence alterations through error-prone non-homologous end joining (NHEJ) resulting in gene inactivations/knockouts. Alternatively, the DSB can be repaired through homology-directed repair (HDR) using a donor DNA template, which leads to the introduction of desired sequence modifications at the predetermined site. Here, we summarize the role of three classes of nucleases; zinc finger nucleases (ZFNs), transcription activator like effector nucleases (TALENs), and clustered regularly interspaced palindromic repeats (CRISPR)/CRISPR associated protein 9 (Cas9) system in achieving targeted genome modifications. Further, we discuss the progress towards the applications of programmable site-specific nucleases (SSNs) in treating human diseases and other biological applications in economically important higher eukaryotic organisms such as plants and livestock. (C) 2016 Wiley Periodicals, Inc.
机译：有针对性的基因组工程的最新进展使分子生物学家来生成序列具体的修改以更大的效率和更高的特异性在复杂的真核生物基因组。试剂和细胞DNA修复机制让这成为可能。交付一个特定站点的有力工具基因组双链断裂(双边带)染色体位点,生产序列通过改变容易出错的异源最终加入(NHEJ)导致的基因失活/淘汰赛。可以通过homology-directed修理修理吗使用供体DNA模板(HDR),导致引入所需的序列修改在预定的网站。总结三个类的核酸酶的作用;锌指核酸酶(ZFNs)转录激活效应核酸酶(取得),集群定期空隙回文重复(CRISPR) / CRISPR相关蛋白9(Cas9)系统在实现有针对性的基因组修改。可编程的应用在治疗人类特有的核酸酶(ssn)疾病和其他生物应用经济上重要的高等真核生物如植物和家畜。威利期刊公司。

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