转录激活因子样效应物核酸酶

Transcription activator-like effector nucleases

人工设计的核酸酶是目前进行精确基因组修饰的有效工具之一,在生命科学基础研究和人类遗传疾病的治疗研究领域有着广泛的应用价值。有必要综述转录激活因子样效应物核酸酶(transcription activator-like effector nucleases,TALEN)的技术背景,以及其应用研究基础上的核酸酶活性和特异性,并探讨TALEN用于靶向基因组修饰过程中所存在的一些问题。

Custom-designed nucleases have become an effective tool for precise modification of complex genomes which have been widely used for basic research in life science and gene therapy for human genetic disease. It is necessary to summarize the technological background, the activity and specificity of transcription activator-like effector nucleases （TALENs） for applied research, and further discuss the existing problems during the process used for targeted genome modification.