基因组编辑脱靶研究进展

Genome-editing: focus on the off-target effects

摘要:

近年各种基因组编辑技术的成功研发为人类疾病的治疗与预防谱写了新的篇章，这些技术对应的基因组编辑工具主要包括锌指核酸酶 (ZFNs)、转录激活子样效应因子核酸酶 (TALENs) 和最近发现的规律成簇间隔短回文重复 (CRISPR)/Cas系统。这些工具相应的脱靶问题目前是制约基因组编辑技术介导人类疾病治疗的重要瓶颈。本文将分别从基因组编辑工具的介绍、脱靶的现状、解决优化的方案和检测方法进行总结与探讨，通过比较，进一步了解基因组编辑工具的优缺点及相关脱靶检测方法的适用性。

Abstract:

Breakthroughs of genome-editing in recent years have paved the way to develop new therapeutic strategies. These genome-editing tools mainly include Zinc-finger nucleases (ZFNs), Transcription activator-like effector nucleases (TALENs), and clustered regulatory interspaced short palindromic repeat (CRISPR)/Cas-based RNA-guided DNA endonucleases. However, off-target effects are still the major issue in genome editing, and limit the application in gene therapy. Here, we summarized the cause and compared different detection methods of off-targets.