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用于基于CRISPR的基因编辑的病毒和非病毒纳米载体。

Viral and nonviral nanocarriers for CRISPR-based gene editing.

作者信息

Guo Zhongyuan, Zhu Audrey T, Fang Ronnie H, Zhang Liangfang

机构信息

Aiiso Yufeng Li Family Department of Chemical and Nano Engineering, University of California San Diego, La Jolla, CA 92093, USA.

Division of Host-Microbe Systems and Therapeutics, Department of Pediatrics, University of California San Diego, La Jolla, CA 92093, USA.

出版信息

Nano Res. 2024 Oct;17(10):8904-8925. doi: 10.1007/s12274-024-6748-5. Epub 2024 Jun 20.

Abstract

The continued development of clustered regularly interspaced short palindromic repeats (CRISPR) technology has the potential to greatly impact clinical medicine, particularly for disease diagnosis and treatment. Despite high demand for the delivery of CRISPR-based therapies, significant challenges persist. These include rapid degradation by enzymes, inefficient disease site targeting, and the risk of undesired off-target outcomes. Nanoparticulate platforms, with their tailorable properties, have been engineered to efficiently package CRISPR payloads in various formats, including as plasmid DNA, mRNA, and ribonucleoprotein complexes, for delivery. Among them, recombinant adeno-associated viruses, virus-like particles, and lipid nanoparticles have displayed exceptional promise. This review will discuss the development of these and other nanocarriers for CRISPR-based genome editing.

摘要

成簇规律间隔短回文重复序列(CRISPR)技术的持续发展有可能对临床医学产生重大影响,特别是在疾病诊断和治疗方面。尽管对基于CRISPR的疗法有很高的需求,但重大挑战依然存在。这些挑战包括被酶快速降解、疾病部位靶向效率低下以及出现意外脱靶结果的风险。具有可定制特性的纳米颗粒平台已被设计用于高效包装各种形式的CRISPR有效载荷,包括质粒DNA、信使核糖核酸和核糖核蛋白复合物,以便进行递送。其中,重组腺相关病毒、病毒样颗粒和脂质纳米颗粒已展现出非凡的前景。本综述将讨论这些以及其他用于基于CRISPR的基因组编辑的纳米载体的发展情况。

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