Gene therapy vectors: the prospects and potentials of the cut-and-paste transposons
Gene therapy vectors: the prospects and potentials of the cut-and-paste transposons
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DOI：
10.1007/s10709-009-9391-x
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发表时间：
2010-05-01
期刊：
影响因子：
1.5
通讯作者：
Chalmers, Ronald M.
中科院分区：
文献类型：
--
作者：
Bouuaert, Corentin Claeys;Chalmers, Ronald M.
基因治疗应用需要有效的工具将遗传信息稳定传递到真核基因组中。目前大多数基因传递策略都是基于病毒载体。然而，一些缺点，例如有限的货物容量、宿主免疫反应和突变风险，凸显了对替代基因传递工具的需求。一个全面的基因治疗工具包应包含一系列可适应不同目标和目的的载体和技术。转座子提供了一种潜在的强大方法。然而，转座子包含大量不同的分子机制，其中一些比其他更适合基因递送应用。在这里，我们考虑各种机制的范围和潜力，重点关注剪切粘贴转座子作为基因治疗应用更有希望的途径之一。目前正在开发几种剪切粘贴转置系统。我们将首先考虑piggyBac和hAT家族元件Tol1和Tol2的机制，然后再关注水手家族元件，包括Mos1、Himar1和Hsmar1。
Gene therapy applications require efficient tools for the stable delivery of genetic information into eukaryotic genomes. Most current gene delivery strategies are based on viral vectors. However, a number of drawbacks, such as the limited cargo capacity, host immune response and mutational risks, highlight the need for alternative gene delivery tools. A comprehensive gene therapy tool kit should contain a range of vectors and techniques that can be adapted to different targets and purposes. Transposons provide a potentially powerful approach. However, transposons encompass a large number of different molecular mechanisms, some of which are better suited to gene delivery applications than others. Here, we consider the range and potentials of the various mechanisms, focusing on the cut-and-paste transposons as one of the more promising avenues towards gene therapy applications. Several cut-and-paste transposition systems are currently under development. We will first consider the mechanisms of piggyBac and the hAT family elements Tol1 and Tol2, before focusing on the mariner family elements including Mos1, Himar1 and Hsmar1.