Use of synthetic peptides for non-viral gene delivery

Use of synthetic peptides for non-viral gene delivery
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DOI:
10.1007/4-431-27879-6_8
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发表时间:
2005-01-01
期刊:
NON-VIRAL GENE THERAPY: GENE DESIGN AND DELIVERY
影响因子:
--
通讯作者:
Katayama, Y
Katayama, Y
中科院分区:
其他
文献类型:
--
作者:
Niidome, T;Katayama, Y

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非病毒基因传递系统的开发是解决病毒基因传递过程中出现的内源性病毒重组、致瘤效应和意外免疫反应等问题的重要关键。正如本书其他章节所讨论的,大多数非病毒基因传递系统的基本技术都依赖于脂质体和聚合物化学;然而,肽化学也为这一领域做出了巨大贡献。肽可以自动或人工合成,它们的化学性质允许设计和合成复杂的结构,例如配体修饰的肽可以被特定的细胞识别,染料修饰的肽可以追踪它们在细胞和组织中的位置,以及其他“智能”肽可以实现功能性基因传递。预计肽构建的相对容易将为研究人员提供广泛的分子以及关于功能基因传递的结构要求的重要信息。多肽在基因传递中发挥着许多作用,如提供一个简单的阳离子片段来结合DNA,作为配体或促进DNA释放到细胞质中,携带信号以运输到细胞核,或作为调节细胞中基因表达的传感器。在本章中,描述了在基因传递中使用肽的一些例子,并讨论了在基因治疗中利用这些肽的前景。
The development of non-viral gene delivery systems is an important key to solving several problems occurring in viral gene delivery, such as endogenous virus recombination, oncogenic effects and unexpected immune responses. As discussed in other chapters of this book, most of the basic techniques of non-viral gene delivery systems have relied on liposome and polymer chemistries; however, peptide chemistry has also contributed strongly to this field. Peptides can be synthesized automatically or manually and their chemistry allows the design and synthesis of complicated structures, eg ligand-modified peptides recognizable by specific cells, dye-modified peptides to trace their locations in cells and tissues, and other “intelligent” peptides to achieve functional gene delivery. It is expected that the relative ease of peptide construction will provide researchers with a wide range of molecules as well as important information about the structural requirements for functional gene delivery. Peptides play many roles in gene delivery, such as providing a simple cationic moiety to bind DNA, acting as a ligand or facilitating the release of DNA into the cytosol, carrying a signal for transport to the nucleus, or functioning as a sensor for the regulation of gene expression in cells. In this chapter, some examples of the use of peptides in gene delivery are described, and the prospects of utilizing such peptides in gene therapy are discussed.