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首页> 外文期刊>Acta biomaterialia >Polysaccharide gene transfection agents
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Polysaccharide gene transfection agents

机译:多糖基因转染剂

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Gene delivery is a promising technique that involves in vitro or in vivo introduction of exogenous genes into cells for experimental and therapeutic purposes. Successful gene delivery depends on the development of effective and safe delivery vectors. Two main delivery systems, viral and non-viral gene carriers, are currently deployed for gene therapy. While most current gene therapy clinical trials are based on viral approaches, non-viral gene medicines have also emerged as potentially safe and effective for the treatment of a wide variety of genetic and acquired diseases. Non-viral technologies consist of plasmid-based expression systems containing a gene associated with the synthetic gene delivery vector. Polysaccharides compile a large family of heterogenic sequences of monomers with various applications and several advantages as gene delivery agents. This chapter, compiles the recent progress in polysaccharide based gene delivery, it also provides an overview and recent developments of polysaccharide employed for in vitro and in vivo delivery of therapeutically important nucleotides, e.g. plasmid DNA and small interfering RNA.
机译:基因递送是一种有前途的技术,其涉及将外源基因体外或体内引入细胞用于实验和治疗目的。成功的基因递送取决于有效和安全的递送载体的发展。目前已经部署了两种主要的递送系统,即病毒和非病毒基因载体,用于基因治疗。尽管目前大多数的基因疗法临床试验都基于病毒方法,但非病毒基因药物也已出现,对于治疗多种遗传和后天疾病均具有潜在的安全性和有效性。非病毒技术由基于质粒的表达系统组成,该系统包含与合成基因传递载体相关的基因。多糖具有多种用途,并具有许多作为基因传递剂的优势,可汇编出一大类单体的异源序列。本章汇编了基于多糖的基因递送的最新进展,也概述了用于体内和体内递送治疗上重要的核苷酸(例如核苷酸)的多糖的概况和最新进展。质粒DNA和小干扰RNA。

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