Beckman Research Institute of the City of Hope, Duarte, CA, USA.
Curr Opin Immunol. 2012 Oct;24(5):625-32. doi: 10.1016/j.coi.2012.08.013. Epub 2012 Sep 15.
HIV/AIDS remains a chronic and incurable disease, in spite of the notable successes of combination antiretroviral therapy. Gene therapy offers the prospect of creating genetic resistance to HIV that supplants the need for antiviral drugs. In sight of this goal, a variety of anti-HIV genes have reached clinical testing, including gene-editing enzymes, protein-based inhibitors, and RNA-based therapeutics. Combinations of therapeutic genes against viral and host targets are designed to improve the overall antiviral potency and reduce the likelihood of viral resistance. In cell-based therapies, therapeutic genes are expressed in gene modified T lymphocytes or in hematopoietic stem cells that generate an HIV-resistant immune system. Such strategies must promote the selective proliferation of the transplanted cells and the prolonged expression of therapeutic genes. This review focuses on the current advances and limitations in genetic therapies against HIV, including the status of several recent and ongoing clinical studies.
尽管联合抗逆转录病毒疗法取得了显著成功，但艾滋病毒/艾滋病仍然是一种慢性且无法治愈的疾病。基因治疗提供了创造对 HIV 的遗传抗性的前景，从而取代了对抗病毒药物的需求。有鉴于此，各种抗 HIV 基因已进入临床测试，包括基因编辑酶、基于蛋白质的抑制剂和基于 RNA 的治疗方法。针对病毒和宿主靶标的治疗基因的组合旨在提高整体抗病毒效力并降低病毒耐药性的可能性。在基于细胞的疗法中，治疗基因在经过基因修饰的 T 淋巴细胞或产生 HIV 抗性免疫系统的造血干细胞中表达。这些策略必须促进移植细胞的选择性增殖和治疗基因的长期表达。本综述重点介绍了针对 HIV 的基因治疗的当前进展和局限性，包括最近和正在进行的几项临床研究的现状。
