Louise Collins
Clinical Sciences, GKT School of Medicine, Kings College/The Rayne Institute, London, England.
Methods Mol Biol. 2006;333:201-26. doi: 10.1385/1-59745-049-9:201.
Gene therapy holds great promise for treating a variety of human diseases and conditions. The field of gene therapy has advanced rapidly in the last decade. However, a major limiting factor remains the lack of a suitable vector for gene delivery. Although viruses are currently the most commonly researched vector, because of continuing safety concerns research has broadened to developing nonviral alternatives. Nonviral vectors fall into several categories. They can be physical methods, which provide relatively crude delivery approaches, such as direct cell injection, or chemical delivery vehicles. Chemical vectors almost always include a polycation component to assist the passage of DNA to the cell's nucleus. The passage of the transgene through the cell to the nucleus is hampered by many obstacles. Approaches to overcome these, both intracellularly and extracellularly, in order to maximize gene expression are currently under investigation. Nonviral vectors offer a safe and versatile alternative to their viral counterparts. Although still in their infancy, the different nonviral approaches under development hold great potential for many clinical applications.
基因治疗在治疗多种人类疾病和病症方面具有巨大的前景。在过去十年中，基因治疗领域发展迅速。然而，一个主要的限制因素仍然是缺乏合适的基因递送载体。尽管病毒目前是研究最广泛的载体，但由于持续存在的安全问题，研究已扩展到开发非病毒替代物。非病毒载体可分为几类。它们可以是物理方法，提供相对粗糙的递送途径，如直接细胞注射，或者是化学递送载体。化学载体几乎总是包含一个聚阳离子成分以协助DNA进入细胞核。转基因穿过细胞进入细胞核受到许多障碍的阻碍。目前正在研究细胞内和细胞外克服这些障碍以最大化基因表达的方法。非病毒载体为病毒载体提供了一种安全且通用的替代物。尽管仍处于起步阶段，但正在开发的不同非病毒方法在许多临床应用中具有巨大潜力。
