Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems.
Enhancing the In Vitro and In Vivo Stabilities of Polymeric Nucleic Acid Delivery Nanosystems.
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DOI：
10.1021/acs.bioconjchem.8b00749
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发表时间：
2018-12
影响因子：
4.7
通讯作者：
Yuyuan Wang;Mingzhou Ye;Ruosen Xie;S. Gong
中科院分区：
文献类型：
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作者：
Yuyuan Wang;Mingzhou Ye;Ruosen Xie;S. Gong
基因治疗在各种医学和生物医学应用中具有很大的前景。由阳离子聚合物和核酸形成的非病毒基因递送系统（例如，聚合复合物）已被广泛研究用于靶向基因治疗;然而，它们的体外和体内稳定性受它们的内在性质如化学组成（例如，聚合物分子量和结构，以及N/P比）和许多环境因素（例如，血流中循环期间的剪切应力、与血清蛋白的相互作用和生理离子强度）。在这篇综述中，我们调查了一些重要的内在和环境因素对聚合物基因传递系统的稳定性的影响，并讨论了各种策略，以提高聚合物基因传递系统的稳定性，从而使有效的基因传递到靶细胞。最后简要讨论了聚合物核酸纳米系统的发展前景和面临的挑战。
Gene therapy holds great promise for various medical and biomedical applications. Nonviral gene delivery systems formed by cationic polymer and nucleic acids (e.g., polyplexes) have been extensively investigated for targeted gene therapy; however, their in vitro and in vivo stability is affected by both their intrinsic properties such as chemical compositions (e.g., polymer molecular weight and structure, and N/P ratio) and a number of environmental factors (e.g., shear stress during circulation in the bloodstream, interaction with the serum proteins, and physiological ionic strength). In this review, we surveyed the effects of a number of important intrinsic and environmental factors on the stability of polymeric gene delivery systems, and discussed various strategies to enhance the stability of polymeric gene delivery systems, thereby enabling efficient gene delivery into target cells. Future opportunities and challenges of polymeric nucleic acid delivery nanosystems were also briefly discussed.