Scherer L, Rossi J J, Weinberg M S
Division of Molecular Biology, City of Hope Beckman Research Institute, Duarte, CA 91010, USA.
Gene Ther. 2007 Jul;14(14):1057-64. doi: 10.1038/sj.gt.3302977.
The current treatment regimen for HIV-infected individuals combines two or more drugs targeting different viral proteins such as RT and gag. Resistance to conventional drugs can develop quickly, and typically persists. The prospect of longer, continuous antiretroviral therapy brings with it the need for new antiretroviral drugs and approaches. In this context, gene therapies have the potential to prolong life and quality of life as an additional therapeutic class and may serve as an adjuvant to traditional treatments. This review focuses on RNA-based hematopoietic cell gene therapy for treatment of HIV infection. Recent advances in our understanding of RNA interference (RNAi) make this an especially attractive candidate for anti-HIV gene therapy although ribozyme and RNA decoy/aptamer approaches can be combined with RNAi to make a combinatorial therapy akin to highly active anti-retroviral therapy.
目前针对HIV感染者的治疗方案是将两种或更多种针对不同病毒蛋白(如逆转录酶和核衣壳蛋白)的药物联合使用。对传统药物的耐药性可能迅速产生,并且通常会持续存在。长期持续进行抗逆转录病毒治疗的前景带来了对新型抗逆转录病毒药物和治疗方法的需求。在这种背景下,基因治疗作为一种额外的治疗类别,有潜力延长生命并提高生活质量,并且可以作为传统治疗的辅助手段。本综述聚焦于基于RNA的造血细胞基因治疗以治疗HIV感染。尽管核酶和RNA诱饵/适配体方法可与RNA干扰(RNAi)相结合以形成类似于高效抗逆转录病毒治疗的联合疗法,但我们对RNA干扰(RNAi)理解的最新进展使其成为抗HIV基因治疗特别有吸引力的候选方法。