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聚合物作为高效的非病毒基因传递载体：大分子化学和物理结构的作用

Polymers as Efficient Non-Viral Gene Delivery Vectors: The Role of the Chemical and Physical Architecture of Macromolecules.

作者信息

Khan Majad

机构信息

Department of Chemistry, King Fahd University of Petroleum & Minerals KFUPM, Dahran 31261, Saudi Arabia.

Interdisciplinary Research Center for Hydrogen Technologies and Carbon Management (IRC-HTCM), King Fahd University of Petroleum & Minerals KFUPM, Dahran 31261, Saudi Arabia.

出版信息

Polymers (Basel). 2024 Sep 18;16(18):2629. doi: 10.3390/polym16182629.

DOI:10.3390/polym16182629
PMID:39339093
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11435517/
Abstract

Gene therapy is the technique of inserting foreign genetic elements into host cells to achieve a therapeutic effect. Although gene therapy was initially formulated as a potential remedy for specific genetic problems, it currently offers solutions for many diseases with varying inheritance patterns and acquired diseases. There are two major groups of vectors for gene therapy: viral vector gene therapy and non-viral vector gene therapy. This review examines the role of a macromolecule's chemical and physical architecture in non-viral gene delivery, including their design and synthesis. Polymers can boost circulation, improve delivery, and control cargo release through various methods. The prominent examples discussed include poly-L-lysine, polyethyleneimine, comb polymers, brush polymers, and star polymers, as well as hydrogels and natural polymers and their modifications. While significant progress has been made, challenges still exist in gene stabilization, targeting specificity, and cellular uptake. Overcoming cytotoxicity, improving delivery efficiency, and utilizing natural polymers and hybrid systems are vital factors for prospects. This comprehensive review provides an illuminating overview of the field, guiding the way toward innovative non-viral-based gene delivery solutions.

摘要

基因治疗是将外源遗传元件导入宿主细胞以达到治疗效果的技术。尽管基因治疗最初被设想为治疗特定遗传问题的潜在方法，但目前它为许多具有不同遗传模式的疾病和后天性疾病提供了解决方案。基因治疗有两大类载体：病毒载体基因治疗和非病毒载体基因治疗。本综述探讨了大分子的化学和物理结构在非病毒基因递送中的作用，包括它们的设计与合成。聚合物可以通过多种方法促进循环、改善递送并控制货物释放。所讨论的突出例子包括聚-L-赖氨酸、聚乙烯亚胺、梳状聚合物、刷状聚合物和星状聚合物，以及水凝胶和天然聚合物及其修饰。虽然已经取得了重大进展，但在基因稳定、靶向特异性和细胞摄取方面仍然存在挑战。克服细胞毒性、提高递送效率以及利用天然聚合物和混合系统是未来发展的关键因素。这篇全面的综述对该领域进行了具有启发性的概述，为创新的非病毒基因递送解决方案指明了方向。

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