Ohlfest John R, Freese Andrew B, Largaespada David A
Department of Neurosurgery, University of Minnesota, Minneapolis, MN 55455, USA.
Curr Gene Ther. 2005 Dec;5(6):629-41. doi: 10.2174/156652305774964749.
Gene therapy has the potential to improve the clinical outcome of many cancers by transferring therapeutic genes into tumor cells or normal host tissue. Gene transfer into tumor cells or tumor-associated stroma is being employed to induce tumor cell death, stimulate anti-tumor immune response, inhibit angiogenesis, and control tumor cell growth. Viral vectors have been used to achieve this proof of principle in animal models and, in select cases, in human clinical trials. Nevertheless, there has been considerable interest in developing nonviral vectors for cancer gene therapy. Nonviral vectors are simpler, more amenable to large-scale manufacture, and potentially safer for clinical use. Nonviral vectors were once limited by low gene transfer efficiency and transient or steadily declining gene expression. However, recent improvements in plasmid-based vectors and delivery methods are showing promise in circumventing these obstacles. This article reviews the current status of nonviral cancer gene therapy, with an emphasis on combination strategies, long-term gene transfer using transposons and bacteriophage integrases, and future directions.
基因治疗有潜力通过将治疗性基因导入肿瘤细胞或正常宿主组织来改善许多癌症的临床治疗效果。将基因导入肿瘤细胞或肿瘤相关基质正被用于诱导肿瘤细胞死亡、刺激抗肿瘤免疫反应、抑制血管生成以及控制肿瘤细胞生长。病毒载体已被用于在动物模型中实现这一原理验证,并且在某些情况下也用于人类临床试验。然而,开发用于癌症基因治疗的非病毒载体一直备受关注。非病毒载体更简单,更适合大规模生产,并且临床使用可能更安全。非病毒载体曾因基因转移效率低以及基因表达短暂或持续下降而受到限制。然而,基于质粒的载体和递送方法的最新改进在克服这些障碍方面显示出前景。本文综述了非病毒癌症基因治疗的现状,重点关注联合策略、使用转座子和噬菌体整合酶的长期基因转移以及未来方向。