Ousterout David G, Gersbach Charles A
Department of Biomedical Engineering, Duke University, Durham, NC, 27708, USA.
Department of Biomedical Engineering, Duke University, Room 136 Hudson Hall, Box 90281, Durham, NC, 27708-0281, USA.
Methods Mol Biol. 2016;1338:27-42. doi: 10.1007/978-1-4939-2932-0_3.
The development of a facile genome engineering technology based on transcription activator-like effector nucleases (TALENs) has led to significant advances in diverse areas of science and medicine. In this review, we provide a broad overview of the development of TALENs and the use of this technology in basic science, biotechnology, and biomedical applications. This includes the discovery of DNA recognition by TALEs, engineering new TALE proteins to diverse targets, general advances in nuclease-based editing strategies, and challenges that are specific to various applications of the TALEN technology. We review examples of applying TALENs for studying gene function and regulation, generating disease models, and developing gene therapies. The current status of genome editing and future directions for other uses of these technologies are also discussed.
基于转录激活样效应物核酸酶(TALENs)的简便基因组工程技术的发展,已在科学和医学的各个领域取得了重大进展。在本综述中,我们广泛概述了TALENs的发展及其在基础科学、生物技术和生物医学应用中的技术应用。这包括TALE对DNA识别的发现、针对不同靶点设计新的TALE蛋白、基于核酸酶的编辑策略的一般进展,以及TALEN技术各种应用所特有的挑战。我们回顾了应用TALENs研究基因功能和调控、生成疾病模型以及开发基因疗法的实例。还讨论了基因组编辑的现状以及这些技术其他用途的未来方向。