Zhu Guanglin, Zhou Xinzhi, Wen Mingzhang, Qiao Jianjun, Li Guo, Yao Yuan
School of Chemical Engineering and Technology, Tianjin University, Tianjin 300072, China.
ZJU-Hangzhou Global Scientific and Technological Innovation Center, Zhejiang University, Hangzhou, Zhejiang 311200, China.
Biodes Res. 2024 Sep 3;6:0041. doi: 10.34133/bdr.0041. eCollection 2024.
The CRISPR-Cas13 system has emerged as a revolutionary tool for RNA editing, offering new opportunities for the development of nucleic acid therapeutics. Unlike DNA-targeting CRISPR-Cas9, Cas13 targets and cleaves RNA, enabling gene silencing and preventing genomic instability. Its applications include suppressing disease-causing genes, correcting splicing errors, and modulating immune responses. Despite these advances, challenges persist, such as the need to refine specificity, mitigate off-target impacts, and ensure effective delivery. This review provides an overview of the CRISPR-Cas13 mechanism, elucidating its role in RNA-targeted therapies and its transformative potential for disease treatment. Furthermore, it addresses the ongoing challenges that the scientific community is striving to overcome.
CRISPR-Cas13系统已成为RNA编辑的革命性工具,为核酸治疗的发展提供了新机遇。与靶向DNA的CRISPR-Cas9不同,Cas13靶向并切割RNA,实现基因沉默并防止基因组不稳定。其应用包括抑制致病基因、纠正剪接错误和调节免疫反应。尽管取得了这些进展,但挑战依然存在,例如需要提高特异性、减轻脱靶影响并确保有效递送。本综述概述了CRISPR-Cas13机制,阐明了其在RNA靶向治疗中的作用及其对疾病治疗的变革潜力。此外,它还探讨了科学界正在努力克服的持续挑战。