The recently developed CRISPR/Cas9 technology has revolutionized the genome engineering field. Since 2016, increasing number of studies regarding CRISPR therapeutics have entered clinical trials, most of which are focusing on the ex vivo genome editing. In this review, we highlight the ex vivo cell-based CRISPR/Cas9 genome editing for therapeutic applications. In these studies, CRISPR/Cas9 tools were used to edit cells in vitro and the successfully edited cells were considered as therapeutics, which can be introduced into patients to treat diseases. Considering a large number of previous reviews have been focused on the CRISPR/Cas9 delivery methods and materials, this review provides a different perspective, by mainly introducing the targeted conditions and design strategies for ex vivo CRISPR/Cas9 therapeutics. Brief descriptions of the history, functionality and applications of CRISPR/Cas9 systems will be introduced first, followed by the design strategies and most significant results from previous research that used ex vivo CRISPR/Cas9 genome editing for the treatment of conditions or diseases. The last part of this review includes general information about the status of CRISPR/Cas9 therapeutics in clinical trials. We also discuss some of the challenges as well as the opportunities in this research area.

最近开发的 CRISPR/Cas9 技术彻底改变了基因组工程领域。自2016年以来，越来越多有关CRISPR疗法的研究进入临床试验，其中大部分集中于离体基因组编辑。在这篇综述中，我们重点介绍了用于治疗应用的离体细胞 CRISPR/Cas9 基因组编辑。在这些研究中，CRISPR/Cas9工具被用来在体外编辑细胞，成功编辑的细胞被认为是治疗剂，可以引入患者体内治疗疾病。考虑到之前的大量综述都集中在 CRISPR/Cas9 递送方法和材料上，本综述提供了不同的视角，主要介绍了离体CRISPR/Cas9 疗法的靶向条件和设计策略。首先将简要介绍 CRISPR/Cas9 系统的历史、功能和应用，然后介绍设计策略和先前使用离体CRISPR/Cas9 基因组编辑治疗病症或疾病的研究的最重要结果。本综述的最后一部分包括有关 CRISPR/Cas9 疗法在临床试验中的现状的一般信息。我们还讨论了该研究领域的一些挑战和机遇。