Review Article | Open Access

Volume 2024 |Article ID 0041 | https://doi.org/10.34133/bdr.0041

CRISPR–Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics

Guanglin Zhu,^1,7 Xinzhi Zhou,^2,3,7 Mingzhang Wen ,^1,4,5 Jianjun Qiao ,^1,4,5 Guo Li ,^2,3,6 Yuan Yao ^2,3,4

¹School of Chemical Engineering and Technology, Tianjin University, Tianjin 300072, China
²ZJU-HangzhouGlobal Scientific and Technological Innovation Center, Zhejiang University, Hangzhou, Zhejiang 311200,China
³College of Chemical and Biological Engineering, Zhejiang University, Hangzhou, Zhejiang 310027,China
⁴Zhejiang Institute of Tianjin University, Shaoxing 312300, China
⁵Frontiers Science Centerfor Synthetic Biology (Ministry of Education), Tianjin University, Tianjin 300072, P. R. China
⁶XianghuLaboratory, Hangzhou 311231, China
⁷These authors contributed equally to this work

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.