Cas9/sgRNA递送技术及其研究进展

Advances of Cas9/sgRNA delivery system for gene editing

摘要:

在与CRISPR/Cas9基因编辑技术相关的临床应用中，Cas9/sgRNA的递送是决定基因编辑治疗效果的关键技术之一。无需转录和翻译过程的Cas9蛋白/sgRNA复合物直接递送形式可能能够提供更高的特异性和安全性。文中通过对Cas9/sgRNA递送技术的研究现状及其在基因相关疾病治疗中的进展进行简要综述，为新型药物载体的设计和基因治疗的临床应用提供新思路。

Abstract:

In the application of CRISPR genome editing, direct cellular delivery of non-replicable Cas9/sgRNA may reduce unwanted gene targeting and integrational mutagenesis, thus offering greater specificity and safety. Cas9/sgRNA delivery system holds great potential for treating genetic diseases. This review summarizes the advances of Cas9/sgRNA delivery systems and its therapeutic applications, providing new understandings and inspirations for vector design and future clinical applications.