CRISPR/Cas9系统RNP体内递送的挑战与解决策略

Challenges and strategies for in vivo delivery of CRISPR/Cas9-RNP

目的针对体内递送CRISPR/Cas9核酸蛋白复合物(ribonucleoprotein complex,RNP)面临的包封效率低,细胞靶向性差,体内滞留时间短,存在脱靶效应等多种挑战,综述应对方案和研究进展。方法查阅国内外相关文献,对现有递送RNP的载体进行整理,分析其优劣并归类。结果从构建合适的递送载体和探索合理的改造方法两方面提供RNP体内递送问题的解决方案。结论递送RNP实现CRISPR/Cas9系统的基因编辑是一种直接有效的方法,通过RNP体内递送问题的解决方案,有助于RNP在体内发挥更高效精准的基因编辑作用。

Objective To review the solutions and research progress targeting the challenges of in vivo delivery of CRISPR/Cas9 ribonucleoprotein complex(RNP),including low encapsulation efficiency,poor cellular targeting,short retention time,and off-target effects.Methods Related literatures at home and abroad were reviewed to summarize and classify the pros and cons of existing carriers for delivering RNP.Results The solutions of RNP in vivo delivery were provided from two aspects:constructing a suitable delivery vector and exploring a reasonable modification method.Conclusion Delivering RNP to achieve gene editing of CRISPR/Cas9 system is a direct and effective method.The solution to the problem of RNP delivery will help RNP play a more efficient and precise gene editing role in vivo.