Claeys Bouuaert Corentin, Chalmers Ronald M
School of Biomedical Sciences, University of Nottingham, Queen's Medical Centre, Nottingham NG7 2UH, UK.
Genetica. 2010 May;138(5):473-84. doi: 10.1007/s10709-009-9391-x. Epub 2009 Aug 2.
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.
基因治疗应用需要高效的工具，以便将遗传信息稳定地传递到真核生物基因组中。目前大多数基因传递策略都基于病毒载体。然而，许多缺点，如有限的运载能力、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靶点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，其中一些比其他机制更适合基因传递应用。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，将其作为基因治疗应用中更有前景的途径之一。目前正在开发几种剪切粘贴转座系统。在重点讨论包括Mos1、Himar1和Hsmar1在内的水手家族元件之前，我们将首先考虑piggyBac以及hAT家族元件Tol1和Tol2的机制。
