Clustered regularly interspaced short palindromic repeat-CRISPR-associated protein (CRISPR-Cas) systems, found in nature as microbial adaptive immune systems, have been repurposed into an important tool in biological engineering and genome editing, providing a programmable platform for precision gene targeting. These tools have immense promise as therapeutics that could potentially correct disease-causing mutations. However, CRISPR-Cas gene editing components must be transported directly to the nucleus of targeted cells to exert a therapeutic effect. Thus, efficient methods of delivery will be critical to the success of therapeutic genome editing applications. Here, we review current strategies available for in vivo delivery of CRISPR-Cas gene editing components and outline challenges that need to be addressed before this powerful tool can be deployed in the clinic.

成簇的规则间隔短回文重复序列 CRISPR 相关蛋白 (CRISPR-Cas) 系统在自然界中被发现为微生物适应性免疫系统，已被重新利用为生物工程和基因组编辑的重要工具，为精确基因靶向提供了可编程平台。这些工具作为治疗方法具有巨大的前景，有可能纠正引起疾病的突变。然而，CRISPR-Cas基因编辑组件必须直接转运至靶细胞的细胞核才能发挥治疗效果。因此，有效的递送方法对于治疗性基因组编辑应用的成功至关重要。在这里，我们回顾了目前可用于体内递送 CRISPR-Cas 基因编辑组件的策略，并概述了在临床上部署这一强大工具之前需要解决的挑战。