Kodama Kota, Katayama Yoshiki, Shoji Yoko, Nakashima Hideki
Department of Microbiology, St. Marianna University School of Medicine, Sugao Miyamae-ku Kawasaki, Japan.
Curr Med Chem. 2006;13(18):2155-61. doi: 10.2174/092986706777935276.
Since the viral vector for gene therapy has serious problems, including oncogenesity and other adverse effects, non-viral carriers have attracted a great deal of attention. Non-viral carriers are expected to achieve gene therapy without serious side effects. However, the most critical issue of gene delivery by non-viral carriers is the low-expression efficiencies of the desired gene. In order to apply non-viral carriers for gene therapy in practical clinical usage, further understanding of the cellular barriers against gene delivery is a prerequisite. Moreover, additional intelligent concepts for gene delivery are also needed. We will summarize the features and shortcomings of currently developed non-viral delivery systems. Especially, we will address the current progress of cationic lipids (lipoplex) and cationic polymers (polyplex) in terms of transfection efficiency. Furthermore, our group has developed a system that responds to the particular intracellular signals of target disease cells. We have named this gene delivery system a drug delivery system based on responses cellular signal (D-RECS). We will introduce this new concept of intelligent non-viral delivery system that our group recently developed.
由于用于基因治疗的病毒载体存在严重问题，包括致癌性和其他副作用，非病毒载体已引起了广泛关注。非病毒载体有望实现无严重副作用的基因治疗。然而，非病毒载体进行基因递送最关键的问题是所需基因的低表达效率。为了在实际临床应用中应用非病毒载体进行基因治疗，进一步了解基因递送的细胞屏障是一个先决条件。此外，还需要用于基因递送的其他智能概念。我们将总结当前开发的非病毒递送系统的特点和缺点。特别是，我们将从转染效率方面阐述阳离子脂质（脂质体复合物）和阳离子聚合物（多聚体复合物）的当前进展。此外，我们小组开发了一种响应靶疾病细胞特定细胞内信号的系统。我们将这个基因递送系统命名为基于细胞信号响应的药物递送系统（D-RECS）。我们将介绍我们小组最近开发的这种智能非病毒递送系统的新概念。
