作为非病毒载体高效传递 CRISPR/Cas9 的纳米粒子研究进展

IF 4.9 3区医学 Q1 PHARMACOLOGY & PHARMACY

Pharmaceutics Pub Date : 2024-09-11 DOI:10.3390/pharmaceutics16091197

Minse Kim, Youngwoo Hwang, Seongyu Lim, Hyeon-Ki Jang, Hyun-Ouk Kim

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引用次数: 0

摘要

簇状规则间隔短回文重复（CRISPR）/Cas9 系统是一种基因编辑技术。由于传统病毒载体的局限性，纳米颗粒递送系统备受关注。在这篇综述中，我们评估了各种纳米颗粒作为 CRISPR/Cas9 非病毒载体的效率，包括脂基、聚合物、无机和细胞外囊基系统。我们将讨论它们的优势、局限性和当前面临的挑战。本综述旨在通过总结最新进展和强调关键策略，全面概述非病毒递送系统在推进 CRISPR/Cas9 技术临床应用和基因治疗方面的作用。

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Advances in Nanoparticles as Non-Viral Vectors for Efficient Delivery of CRISPR/Cas9

The clustered regularly interspaced short palindromic repeat (CRISPR)/Cas9 system is a gene-editing technology. Nanoparticle delivery systems have attracted attention because of the limitations of conventional viral vectors. In this review, we assess the efficiency of various nanoparticles, including lipid-based, polymer-based, inorganic, and extracellular vesicle-based systems, as non-viral vectors for CRISPR/Cas9 delivery. We discuss their advantages, limitations, and current challenges. By summarizing recent advancements and highlighting key strategies, this review aims to provide a comprehensive overview of the role of non-viral delivery systems in advancing CRISPR/Cas9 technology for clinical applications and gene therapy.

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来源期刊

Pharmaceutics Pharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science

CiteScore

7.90

自引率

11.10%

发文量

2379

审稿时长

16.41 days

期刊介绍： Pharmaceutics (ISSN 1999-4923) is an open access journal which provides an advanced forum for the science and technology of pharmaceutics and biopharmaceutics. It publishes reviews, regular research papers, communications, and short notes. Covered topics include pharmacokinetics, toxicokinetics, pharmacodynamics, pharmacogenetics and pharmacogenomics, and pharmaceutical formulation. Our aim is to encourage scientists to publish their experimental and theoretical details in as much detail as possible. There is no restriction on the length of the papers. The full experimental details must be provided so that the results can be reproduced.