HIV-1基因治疗研究进展

Advances in Research on HIV-1 Gene Therapy

针对HIV感染当前主要使用抗逆转录病毒药物治疗。尽管抗逆转录病毒治疗在一段时间内可抑制病毒的复制,但并不能彻底清除HIV感染,对大多数患者来说须终身服药,这不仅给患者带来极大的经济负担,还易引起药物不良反应及病毒耐药性变异等。基因治疗可以针对病毒或宿主的靶标,抑制病毒的入侵、复制和病毒基因的表达,使用基因编辑技术等还有望清除整合的前病毒DNA,或使宿主细胞获得长期抗HIV感染的抗性。对当前HIV-1基因治疗的研究进展作一概述,并重点介绍已经进入临床研究的基因治疗药物。

Anti-retroviral drugs are the mainstay of current treatment for HIV-1. Although anti-retroviral therapies can temporarily inhibit viral replication, they can not eradicate latent HIV reservoirs. Therefore, continuous and life-long treatment is necessary for most patients, bringing huge economic burden as well as the risk of side effects and resistant mutation. Gene therapy can inhibit viral invasion, replication and gene expression by targeting virus gene or host co-receptors. Gene editing technique is promising to eliminate the integrated provirus HIV-1 DNA, or to make host cells acquire long-term resistance to HIV infection. This review introduces the research progress in current gene therapy with special focus on those therapies undergoing clinical research.