纳米粒子在CRISPR/Cas9基因治疗中的应用

Application of nanoparticles in CRISPR/Cas9-based gene therapy

摘要:

CRISPR/Cas9基因编辑技术已成为基因治疗领域最有前景的工具。在临床应用中，对CRISPR/Cas9进行安全有效的递送一直是亟待解决的问题。纳米粒子，如脂基纳米粒子、聚合物纳米粒子、纳米金颗粒以及生物膜类纳米粒子等，因其生物相容性、安全性和可设计性等特点有望为基因治疗带来新的突破。文中首先对纳米粒子的特性和基因治疗中CRISPR/Cas9的发展进行了概述，然后详细归纳了纳米粒子在递送不同形式的CRISPR/Cas9中的应用，最后对纳米粒子介导的基因治疗的递送在未来面临的挑战和安全性等方面作出总结论述。

Abstract:

CRISPR/Cas9 is a cutting-edge gene-editing technology that has emerged as a promising tool for gene therapy. Nevertheless, the safe and efficient delivery of CRISPR/Cas9 is still an urgent issue in clinical application. Nanoparticles, such as lipid-based nanoparticles, polymer nanoparticles, gold nanoparticles, and biofilm nanoparticles, are expected to bring new opportunities for CRISPR/Cas9-based gene therapy because of their biocompatibility, safety and designability. This review briefly introduced the characteristics of nanoparticles and the development of CRISPR/Cas9 in gene therapy. Moreover, the application of nanoparticles in the delivery of different forms of CRISPR/Cas9 were elaborated. Finally, the challenges and safety of nanoparticle-based gene therapy were discussed.