文章：

CRISPR/Cas9的脂质体递送

Liposomal delivery of CRISPR/Cas9 

原文发布日期：2019-11-02 

英文摘要：

Liposomes are one of the most widely investigated carriers for CRISPR/Cas9 delivery. The surface properties of liposomal carriers, including the surface charge, PEGylation, and ligand modification can significantly affect the gene silencing efficiency. Three barriers of systemic CRISPR/Cas9 delivery (long blood circulation, efficient tumor penetration, and efficient cellular uptake/endosomal escape) are analyzed on liposomal carriers with different surface charges, PEGylations, and ligand modifications. Cationic formulations dominate CRISPR/Cas9 delivery and neutral formulations also have good performance while anionic formulations are generally not proper for CRISPR/Cas9 delivery. The PEG dilemma (prolonged blood circulation vs. reduced cellular uptake/endosomal escape) and the side effect of repeated PEGylated formulation (accelerated blood clearance) were discussed. Effects of ligand modification on cationic and neutral formulations were analyzed. Finally, we summarized the achievements in liposomal CRISPR/Cas9 delivery, outlined existing problems, and provided some future perspectives. 

摘要翻译： 

脂质体是CRISPR/Cas9递送系统中研究最广泛的载体之一。脂质体载体的表面特性（包括表面电荷、PEG化修饰和配体修饰）会显著影响基因沉默效率。本文针对不同表面电荷、PEG化修饰及配体修饰的脂质体载体，系统分析了CRISPR/Cas9体内递送需要克服的三个屏障（长循环时间、高效肿瘤穿透性和高效细胞摄取/内体逃逸）。阳离子制剂在CRISPR/Cas9递送中占主导地位，中性制剂同样表现良好，而阴离子制剂通常不适用于CRISPR/Cas9递送。文中探讨了PEG化面临的困境（延长血液循环时间与降低细胞摄取/内体逃逸效率之间的矛盾）以及重复注射PEG化制剂引发的加速血液清除副作用，分析了配体修饰对阳离子和中性制剂的影响，最后总结了脂质体递送CRISPR/Cas9的研究成果，指出当前存在的问题并展望未来发展方向。

原文链接：

Liposomal delivery of CRISPR/Cas9