Abstract
Recent progress in gene therapy has opened the door for various human diseases. The greatest challenge that gene vectors still face is the ability to sufficiently deliver nucleic acid into target cells. To overcome various barriers, plenty of researches have been undertaken utilizing diverse strategies, among which a wide variety of polycation/pDNA vectors have been developed and explored frequently. For enhanced transfection efficiency, polycations are constantly utilized with covalent modifications, which however lead to reduced positive charge density and changed properties of polycation/pDNA complexes. Accordingly, non-covalent or ternary strategy is proposed. The cationic properties of polycations can be retained and the transfection efficiency can be enhanced by introducing additional polymers with functional groups via non-covalent assembly. This review will discuss the construction and advantages of ternary complexes gene delivery system, including low toxicity and enhanced gene expression both in vitro and in vivo. Recent progress and expectations with promising results that may have some reference for clinical application are also discussed.
Keywords: Gene vectors, non-covalent, polycations, ternary complexes, transfection.
Current Drug Metabolism
Title:Polycation-Based Ternary Gene Delivery System
Volume: 16 Issue: 2
Author(s): Shuai Liu and Tianying Guo
Affiliation:
Keywords: Gene vectors, non-covalent, polycations, ternary complexes, transfection.
Abstract: Recent progress in gene therapy has opened the door for various human diseases. The greatest challenge that gene vectors still face is the ability to sufficiently deliver nucleic acid into target cells. To overcome various barriers, plenty of researches have been undertaken utilizing diverse strategies, among which a wide variety of polycation/pDNA vectors have been developed and explored frequently. For enhanced transfection efficiency, polycations are constantly utilized with covalent modifications, which however lead to reduced positive charge density and changed properties of polycation/pDNA complexes. Accordingly, non-covalent or ternary strategy is proposed. The cationic properties of polycations can be retained and the transfection efficiency can be enhanced by introducing additional polymers with functional groups via non-covalent assembly. This review will discuss the construction and advantages of ternary complexes gene delivery system, including low toxicity and enhanced gene expression both in vitro and in vivo. Recent progress and expectations with promising results that may have some reference for clinical application are also discussed.
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Cite this article as:
Liu Shuai and Guo Tianying, Polycation-Based Ternary Gene Delivery System, Current Drug Metabolism 2015; 16 (2) . https://dx.doi.org/10.2174/138920021602150713115608
DOI https://dx.doi.org/10.2174/138920021602150713115608 |
Print ISSN 1389-2002 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5453 |
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