CRISPR/Cas9 systems: Delivery technologies and biomedical applications

IF 10.7 1区 医学 Q1 PHARMACOLOGY & PHARMACY Asian Journal of Pharmaceutical Sciences Pub Date : 2023-11-01 DOI:10.1016/j.ajps.2023.100854
Yimin Du , Yanfei Liu , Jiaxin Hu , Xingxing Peng , Zhenbao Liu
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Abstract

The emergence of the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated protein 9 (Cas9) genome-editing system has brought about a significant revolution in the realm of managing human diseases, establishing animal models, and so on. To fully harness the potential of this potent gene-editing tool, ensuring efficient and secure delivery to the target site is paramount. Consequently, developing effective delivery methods for the CRISPR/Cas9 system has become a critical area of research. In this review, we present a comprehensive outline of delivery strategies and discuss their biomedical applications in the CRISPR/Cas9 system. We also provide an in-depth analysis of physical, viral vector, and non-viral vector delivery strategies, including plasmid-, mRNA- and protein-based approach. In addition, we illustrate the biomedical applications of the CRISPR/Cas9 system. This review highlights the key factors affecting the delivery process and the current challenges facing the CRISPR/Cas9 system, while also delineating future directions and prospects that could inspire innovative delivery strategies. This review aims to provide new insights and ideas for advancing CRISPR/Cas9-based delivery strategies and to facilitate breakthroughs in biomedical research and therapeutic applications.

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CRISPR/Cas9系统:传递技术和生物医学应用
聚类规则间隔短回文重复序列(CRISPR)/CRISPR相关蛋白9 (Cas9)基因组编辑系统的出现,在人类疾病管理、建立动物模型等领域带来了重大变革。为了充分利用这种强大的基因编辑工具的潜力,确保高效和安全的递送到目标位点是至关重要的。因此,为CRISPR/Cas9系统开发有效的递送方法已成为一个关键的研究领域。在这篇综述中,我们全面概述了递送策略,并讨论了它们在CRISPR/Cas9系统中的生物医学应用。我们还提供了物理,病毒载体和非病毒载体递送策略的深入分析,包括质粒,mRNA和蛋白质为基础的方法。此外,我们说明了CRISPR/Cas9系统的生物医学应用。这篇综述强调了影响递送过程的关键因素和CRISPR/Cas9系统当前面临的挑战,同时也描绘了未来的方向和前景,可以激发创新的递送策略。本综述旨在为推进基于CRISPR/ cas9的递送策略提供新的见解和思路,促进生物医学研究和治疗应用的突破。
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来源期刊
Asian Journal of Pharmaceutical Sciences
Asian Journal of Pharmaceutical Sciences Pharmacology, Toxicology and Pharmaceutics-Pharmaceutical Science
CiteScore
18.30
自引率
2.90%
发文量
11
审稿时长
14 days
期刊介绍: The Asian Journal of Pharmaceutical Sciences (AJPS) serves as the official journal of the Asian Federation for Pharmaceutical Sciences (AFPS). Recognized by the Science Citation Index Expanded (SCIE), AJPS offers a platform for the reporting of advancements, production methodologies, technologies, initiatives, and the practical application of scientific knowledge in the field of pharmaceutics. The journal covers a wide range of topics including but not limited to controlled drug release systems, drug targeting, physical pharmacy, pharmacodynamics, pharmacokinetics, pharmacogenomics, biopharmaceutics, drug and prodrug design, pharmaceutical analysis, drug stability, quality control, pharmaceutical engineering, and material sciences.
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