Current developments in gene transfection agents

Current developments in gene transfection agents
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DOI:
10.2174/1567201043479902
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发表时间:
2004-04-01
影响因子:
2.4
通讯作者:
Domb, Abraham J.
Domb, Abraham J.
中科院分区:
医学4区
文献类型:
--
作者:
Azzam, Tony;Domb, Abraham J.

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DNA可以使用物理手段或将基因携带到细胞中用于基因表达的特定载体递送到细胞核中)。已经研究了用于递送H基因的各种载体,其可以分为两个主要组:其中待递送的DNA插入病毒中的病毒载体,和与DNA形成静电相互作用的阳离子分子载体。成功的基因治疗取决于将遗传物质有效地递送到细胞核中及其在这些细胞内的有效表达。尽管目前合成分子基因载体的体内表达水平低于病毒载体,并且基因表达是瞬时的,但这些载体可能具有几个优点,包括安全性、低免疫原性、递送大基因的能力和低成本的大规模生产。目前研究最多的两大类合成基因传递系统是阳离子脂质和阳离子聚合物,本文综述了近年来在病毒载体、物理手段和分子基因载体方面的研究进展。最后一部分重点介绍了我们最近在开发一系列用于体外和体内基因转染的可生物降解聚阳离子方面的研究。
DNA can be delivered into the cell nucleus either using physical means or specific carriers that carry the genes into the cells for gene expression). Various carriers for delivering H genes have been investigated which can be divided into two main groups: viral carriers where the DNA to be delivered is inserted into a virus, and cationic molecular carriers that form electrostatic interactions with DNA). Successful gene therapy depends on the efficient delivery of genetic materials into the cells nucleus and its effective expression within these cells). Although at present the in vivo expression levels of synthetic molecular gene vectors are lower than for viral vectors and gene expression is transient, these vehicles are likely to present several advantages including safety, low-immunogenicity, capacity to deliver large genes and large-scale production at low-cost). The two leading classes of synthetic gene delivery systems that have been mostly investigated are cationic lipids and cationic polymers).This review discusses recent developments in viral vectors, physical means and molecular gene carriers). The last part focuses on our recent studies in developing a new series of biodegradable polycations for in vitro and in vivo gene transfection).