(上海市儿童医院,上海交通大学附属儿童医院医学遗传研究所,上海 200040)

摘 要:摘 要: 转录激活因子样效应蛋白核酸酶(transcription activator-like effector nuclease, TALEN),由TALE (transcription activator-like effector)结构域和Fok I核酸内切酶结构域人工融合而成,它是近几年发展起来的一种可对基因组定点改造的新技术。TALEN能够特异识别一段DNA序列,并能够对双链DNA进行切割,TALEN造成DNA断裂后可以启动细胞对DNA的修复,从而实现特定位点的基因操作如基因敲除、基因敲进、基因修复等。相比ZFNs,TALENs的获得更为容易、效果更为明显,它在理论上真正实现了对任意序列进行基因操作的可能。综述了TALEN技术的研究发展过程、结构与作用机制、构建TALEN的方法,以及目前这一技术的应用等。
关键词:TALEN ;基因定点修饰技术;作用机制;构建方法

TALENs: A new genome site-specific modification technology
ZHANG Jin-Mai, REN Zhao-Rui*
(Shanghai Institute of Medical Genetics, Children′s Hospital of Shanghai, Shanghai Jiao Tong University, Shanghai 200040, China)

Abstract: Abstract: Transcription activator-like effector nuclease (TALEN) is an artificially engineered hybrid protein that contains a transcription activator-like effector (TALE) domain and a Fok I endonuclease cleavage domain. It has recently emerged as a powerful molecular tool for targeted genome modifications. TALENs recognize and bind to specific DNA sequences to generate a double-strand break (DSB) by its nuclease activity. Based on this finding, various genetic methods, including gene targeting (gene disruption), gene addition, gene editing are being designed to manipulate the genomes at specific loci. Compared with ZFNs, TALENs are obtained more easily and much more effective. It is TALEN that really achieving gene modification at any site of genome. Here, we reviewed the recent progress and prospects of TALEN technology, the mechanism of how it works, construction methods, as well as a collection of species and genes that have been successfully modified by TALEN.
Key words: TALEN; genome site-specific modification technology; mechanism; construction

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