The CRISPR (clustered regularly interspaced short palindromic repeats)-based genome editing technology is an emerging RNA-guided nuclease system initially identified from the microbial adaptive immune systems. In recent years, the CRISPR system has been reprogrammed to target specific regions of the eukaryotic genome and has become a powerful tool for genetic engineering. Researchers have explored many approaches to improve the genome editing activity of the CRISPR-Cas system and deliver its components both ex vivo and in vivo. Moreover, these strategies have been applied to genome editing in preclinical research and clinical trials. In this review, we focus on representative strategies for regulation and delivery of the CRISPR-Cas system, and outline current therapeutic applications in their clinical translation.

中文翻译：

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基于 CRISPR 的治疗策略。

基于 CRISPR（成簇规律间隔的短回文重复序列）的基因组编辑技术是一种新兴的 RNA 引导的核酸酶系统，最初是从微生物适应性免疫系统中鉴定出来的。近年来，CRISPR 系统经过重新编程以靶向真核基因组的特定区域，并已成为基因工程的有力工具。研究人员已经探索了许多方法来提高 CRISPR-Cas 系统的基因组编辑活性，并在体外和体内递送其组分。此外，这些策略已应用于临床前研究和临床试验中的基因组编辑。在这篇综述中，我们重点关注 CRISPR-Cas 系统调控和递送的代表性策略，并概述当前在其临床转化中的治疗应用。