ACS Nano：新型纳米颗粒运输系统或能克服CRISPR基因编辑障碍

Direct Cytosolic Delivery of CRISPR/Cas9-Ribonucleoprotein for Efficient Gene Editing

近日，一项刊登在国际杂志ACSNano上的研究报告中，来自马萨诸塞大学阿默斯特分校的研究人员通过研究利用纳米颗粒设计出了一种新系统能够帮助CRISPR／Cas9系统跨过细胞膜进入到细胞核中，同时还能够避免被细胞器所捕获。研究者Rubul Mout说道，CRISPR由两种组分组成，包括名为Cas9的剪刀样蛋白和名为sgRNA的RNA分子，sgRNA分子能够引导Cas9进入到靶点位置。

Genome editing through the delivery of CRISPR/Cas9-ribonucleoprotein (Cas9-RNP) reduces unwanted gene targeting and avoids integrational mutagenesis that can occur through gene delivery strategies. Direct and efficient delivery of Cas9-RNP into the cytosol followed by translocation to the nucleus remains a challenge. Here, we report a remarkably highly efficient (∼90%) direct cytoplasmic/nuclear delivery of Cas9 protein complexed with a guide RNA (sgRNA) through the coengineering of Cas9 protein and carrier nanoparticles. This construct provides effective (∼30%) gene editing efficiency and opens up opportunities in studying genome dynamics.