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CRISPR-Cas13:用于核酸治疗的开创性RNA编辑技术

CRISPR-Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics.

作者信息

Zhu Guanglin, Zhou Xinzhi, Wen Mingzhang, Qiao Jianjun, Li Guo, Yao Yuan

机构信息

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.

出版信息

Biodes Res. 2024 Sep 3;6:0041. doi: 10.34133/bdr.0041. eCollection 2024.

Abstract

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.

摘要

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。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/42aa/11371277/cd96de9a3e93/bdr.0041.fig.001.jpg

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