Genome editing employs targeted nucleases as powerful tools to precisely alter the genome of target cells and regulate functional genes. Various strategies have been risen so far as the molecular scissors-mediated genome editing that includes zinc finger nuclease, transcription activator-like effector nucleases, and clustered regularly interspaced short palindromic repeats—CRISPR-related protein 9. These tools allow researchers to understand the basics of manipulating the genome, create animal models to study human diseases, understand host–pathogen interactions and design disease targets. Targeted genome modification utilizing RNA-guided nucleases are of recent curiosity, as it is a fast and effective strategy that enables the researchers to manipulate the gene of interest, carry out functional studies, understand the molecular basis of the disease and design targeted therapies. CRISPR-Cas9, a bacterial defense system employed against viruses, consists of a single-strand RNA-guided Cas9 nuclease connected to the corresponding complementary target sequence. This powerful and versatile tool has gained tremendous attention among the researchers, owing to its ability to correct genetic disorders. To help illustrate the potential of this gene editor in unexplored corners of oncology, we describe the history of CRISPR-Cas9, its rapid progression in cancer research as well as future perspectives.

基因组编辑使用靶向核酸酶作为强大的工具来精确改变靶细胞的基因组并调节功能基因。到目前为止，已经出现了各种策略，例如分子剪刀介导的基因组编辑，包括锌指核酸酶、转录激活因子样效应核酸酶和成簇的规则间隔短回文重复序列——CRISPR 相关蛋白 9。这些工具使研究人员能够了解基础知识操纵基因组，创建动物模型来研究人类疾病，了解宿主 - 病原体相互作用并设计疾病目标。利用 RNA 引导的核酸酶进行靶向基因组修饰最近引起了人们的好奇，因为它是一种快速有效的策略，使研究人员能够操纵感兴趣的基因，进行功能研究，了解疾病的分子基础并设计靶向治疗。CRISPR-Cas9 是一种针对病毒的细菌防御系统，由连接到相应互补靶序列的单链 RNA 引导的 Cas9 核酸酶组成。这种强大而多功能的工具因其纠正遗传疾病的能力而在研究人员中引起了极大的关注。为了帮助说明这种基因编辑器在肿瘤学未探索领域的潜力，我们描述了 CRISPR-Cas9 的历史、它在癌症研究中的快速进展以及未来的前景。这种强大而多功能的工具因其纠正遗传疾病的能力而在研究人员中引起了极大的关注。为了帮助说明这种基因编辑器在肿瘤学未探索领域的潜力，我们描述了 CRISPR-Cas9 的历史、它在癌症研究中的快速进展以及未来的前景。这种强大而多功能的工具因其纠正遗传疾病的能力而在研究人员中引起了极大的关注。为了帮助说明这种基因编辑器在肿瘤学未探索领域的潜力，我们描述了 CRISPR-Cas9 的历史、它在癌症研究中的快速进展以及未来的前景。