中文摘要：

类转录激活因子效应物核酸酶（transcription activator-like effector nucleases,TALENs）是近年兴起的一种基因定点编辑技术,由特异性DNA结合结构域TALE和DNA切割结构域Fok I核酸内切酶组成。由于TALEN能对基因组中特异性基因进行识别和定点剪切,导致基因DNA双链断裂,进一步诱导DNA损伤后修复,可对基因组中的特定位点进行高效遗传操作,如基因的敲入、敲除和修复等。TALEN技术以其设计简单、特异性高、毒性低及靶点选择灵活等特点成为目前应用最为广泛的基因定点编辑技术。本文将就TALEN的原理、研究进展以及临床应用展望作一综述。

英文摘要：

Transcription activator-like effectors nucleases（ TALENs） has been a novel landmark genetic engineering tool for targeted genome site-specific editing in recent years,which consists of sequence-specific DNA-binding domain TALE and non-specific DNA cleavage domain FokI endonuclease. TALEs recognize and bind to specific DNA sequences and FokI generate a double-strand break（ DSB） by its nuclease activity,and then DNA damage repair system induced. TALENs are constructed to mediate high efficient multiple genetic manipulation,through forming DSB,including target genes specific sites of gene segment knock-in,knock-out or correction,etc. TALEN technology with its simple design,high specificity,low toxicity and target selection flexible becomes the most widely used genome site-specific editing technology. Here,we will review the recent progress,clinical application and prospects of TALEN technology.