CRISPR–Cas13: Pioneering RNA Editing for Nucleic Acid Therapeutics

作者：Guanglin Zhu, Xinzhi Zhou, Mingzhang Wen, Jianjun Qiao, Li Guo, Yuan Yao · 发表于：BioDesign Research · 年份：2024 · DOI：10.34133/bdr.0041 · 被引用次数：43 · 研究领域：CRISPR and Genetic Engineering、RNA regulation and disease、RNA and protein synthesis mechanisms

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.