CRISPR–Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics

Author:

Zhu Guanglin1,Zhou Xinzhi23,Wen Mingzhang145,Qiao Jianjun145,Li Guo236,Yao Yuan234ORCID

Affiliation:

1. School of Chemical Engineering and Technology, Tianjin University, Tianjin 300072, China.

2. ZJU-Hangzhou Global Scientific and Technological Innovation Center, Zhejiang University, Hangzhou, Zhejiang 311200, China.

3. College of Chemical and Biological Engineering, Zhejiang University, Hangzhou, Zhejiang 310027, China.

4. Zhejiang Institute of Tianjin University, Shaoxing 312300, China.

5. Frontiers Science Center for Synthetic Biology (Ministry of Education), Tianjin University, Tianjin 300072, P. R. China.

6. Xianghu Laboratory, Hangzhou 311231, China.

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.

Funder

National Key Research and Development Program of China

Publisher

American Association for the Advancement of Science (AAAS)

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