Centre for Gene Therapy, Institute of Child Health, University College London, London WC1N 1EH, UK.
Viruses. 2014 Jan 14;6(1):243-63. doi: 10.3390/v6010243.
Restriction factors are a collection of antiviral proteins that form an important aspect of the innate immune system. Their constitutive expression allows immediate response to viral infection, ahead of other innate or adaptive immune responses. We review the molecular mechanism of restriction for four categories of restriction factors; TRIM5, tetherin, APOBEC3G and SAMHD1 and go on to consider how the TRIM5 and TRIMCyp proteins in particular, show promise for exploitation using gene therapy strategies. Such approaches could form an important alternative to current anti-HIV-1 drug regimens, especially if combined with strategies to eradicate HIV reservoirs. Autologous CD4+ T cells or their haematopoietic stem cell precursors engineered to express TRIMCyp restriction factors, and provided in a single therapeutic intervention could then be used to restore functional immunity with a pool of cells protected against HIV. We consider the challenges ahead and consider how early clinical phase testing may best be achieved.
限制因子是一组抗病毒蛋白,是先天免疫系统的重要组成部分。它们的组成型表达允许在其他先天或适应性免疫反应之前,对病毒感染做出即刻反应。我们综述了四类限制因子(TRIM5、 tetherin、APOBEC3G 和 SAMHD1)的限制分子机制,并进一步探讨了 TRIM5 和 TRIMCyp 蛋白,特别是如何具有利用基因治疗策略进行开发的潜力。这些方法可能成为现有抗 HIV-1 药物治疗方案的重要替代方案,尤其是如果与清除 HIV 储存库的策略相结合。经过基因工程改造表达 TRIMCyp 限制因子的自体 CD4+T 细胞或其造血干细胞前体,并在单次治疗干预中提供,然后可用于用一群对 HIV 具有保护作用的细胞来恢复功能性免疫。我们考虑了未来的挑战,并考虑了如何最好地进行早期临床阶段测试。