[关键词]
[摘要]
基因编辑技术能够对基因组序列进行准确、稳定的遗传改造,给生命科学和临床治疗带来革命性的变革。CRISPR/Cas9技术是基因编辑领域一个突破性进展,它操作简单、编辑效率高,极大地扩展了科学家对基因的操控能力。本文中,笔者将介绍基因编辑技术的分类、CRISPR/Cas9技术的应用(基因敲除、基因敲入、CRISPR/Cas9导入细胞的方法、提高CRISPR/Cas9特异性和调控CRISPR/Cas9系统)、CRISPR/cas9系统的其他应用(碱基转换、表观遗传调控、内源基因的转录调控及全基因组范围内的遗传筛选)及CRISPR/Cas9系统在癌症研究中的应用(建立癌症的小鼠模型、建立染色体重排的癌症模型、高通量筛查与肿瘤细胞转移相关的基因和癌症治疗),着重介绍其在癌症领域的应用和发展状况,使读者对该技术有一个总体的把握。
[Key word]
[Abstract]
Genome-editing technologies that enable precise and stable modification of the genome are sparking a revolution in life science research and disease management. The discovery of the CRISPR/Cas9 technology repre-sents a huge breakthrough in the field of genome editing due to its easy operation and high efficiency, empowering scientists to manipulate genome editing to a broader extension. In this review, the classification of genome-editing technologies, the potential applications of the CRISPR/Cas9 system (including gene knockout, knock-in, methods of delivering CRISPR/Cas9 into cells, enhancing specificity of the CRISPR/Cas9, and accurate regulation of the CRIS-PR/Cas9) as well as broader applications of the CRISPR/Cas9 system (including base replacement, epigenetic regu-lation, regulation of endogenous gene expression, and genome-wide genetic screening) will be elaborated, respec-tively. Moreover, CRISPR/Cas9 in the field of cancer research, including generation of mouse models of cancer, gen-eration of cancer models of chromosome rearrangement, high-throughput screening of genes associated with tumor metastasis, as well as treatment of cancer will also be illustrated, providing readers with a general understanding of the CRISPR/Cas9 technology.
[中图分类号]
[基金项目]
国家自然科学基金资助项目(No. 81370302)