关键词:  基因表达调控, 模型, 动物, 胚胎研究, 临床试验, CRISPR/Cas9, 基因编辑

Abstract: While the CRISPR/Cas9 gene editing technology has been continuously improved and reformed in recent years, it has been gradually replaced zinc-finger nuclease (ZFN) and transcription activator-like effector nuclease (TALEN) technology as the third generation gene editing technology with wide application prospect. CRISPR/Cas9 technology has been widely used in the cellular gene editing and gene regulation, gene knockout animal models construction and the human disease treatment research based on animal models. In addition, related experiments have already confirmed that this technology can play a role in gene editing of human embryos. There are two CRISPR/Cas9 clinical trials at present. It is worth looking forward to be applied in clinical targeted therapy. In this paper, the basic structure, the principle of action and potential application of CRISPR/Cas9 gene editing technology are briefly reviewed.

Key words: Gene expression regulation, Models, animal, Embryo research, Clinical trial, CRISPR/Cas9, Gene editing