Cheng Himchan, Jeong Euihwan, Cho Seung Woo
Department of Biomedical Engineering, College of Information and Biotechnology, Ulsan National Institute of Science and Technology, Ulsan, Republic of Korea.
CasCure Therapeutics, Ulsan, Republic of Korea.
Exp Mol Med. 2025 Jul;57(7):1373-1380. doi: 10.1038/s12276-025-01500-6. Epub 2025 Jul 31.
The CRISPR-Cas system has become a worldwide genome editing tool for various organisms. Its precision and efficiency have facilitated basic research, drug discovery and therapeutic interventions. In contrast to other genome editing agents, CRISPR-Cas is modulated by a short guide RNA. Due to its simplicity, CRISPR-Cas is recognized as the best candidate for multiplexed genome editing. With simultaneous targeting, efficient knockout of genes with large deletions is possible. In addition, CRISPR-Cas can induce complex structural variations, such as inversions, translocations and duplications. Moreover, by utilizing engineered CRISPR-Cas proteins specialized for direct repression or activation of gene expression, one can perform multiplexed epigenetic editing. Lastly, multiplexed targeting enables killing of specific types of cells by accumulating stress mediated by simultaneous DNA damages. Here we discuss how CRISPR-based editing technologies for multiple targets are applied in recent studies.
CRISPR-Cas系统已成为一种用于各种生物体的全球基因组编辑工具。其精确性和效率推动了基础研究、药物发现和治疗干预。与其他基因组编辑试剂不同，CRISPR-Cas由短引导RNA调控。由于其简单性，CRISPR-Cas被认为是多重基因组编辑的最佳候选者。通过同时靶向，可以高效敲除带有大片段缺失的基因。此外，CRISPR-Cas可以诱导复杂的结构变异，如倒位、易位和重复。而且，通过利用专门用于直接抑制或激活基因表达的工程化CRISPR-Cas蛋白，人们可以进行多重表观遗传编辑。最后，多重靶向能够通过累积由同时发生的DNA损伤介导的应激来杀死特定类型的细胞。在此，我们讨论基于CRISPR的多靶点编辑技术在近期研究中的应用情况。
