Mautino M R, Morgan R A
Clinical Gene Therapy Branch, National Human Genome Research Institute, National Institutes of Health, Bethesda, MD, USA.
Gene Ther. 2002 Apr;9(7):421-31. doi: 10.1038/sj.gt.3301674.
Retroviral vectors expressing transdominant negative mutants of Rev (TdRev) inhibit HIV-1 replication by preventing the nuclear export of unspliced viral transcripts, thus inhibiting the synthesis of Gag-Pol, Env and reducing the levels of genomic RNA available for packaging. Due to these effective mechanisms of inhibition, production of HIV-1-based lentiviral vectors expressing TdRev has been difficult. Here we describe HIV-based vectors in which expression of TdRev is negatively regulated by Rev expression. In these vectors, we maintained the wild-type HIV-1 Tat/Rev exons and intron configuration and its mode of splicing regulation. The second Rev exon was mutated to encode TdRev. Inhibition of TdRev expression by Rev during vector production yields high titer vector preparations. A second vector containing an additional anti-HIV gene (env-antisense) was constructed by flipping a 1.2-kb env fragment contained within the Tat/TdRev intron. SupT1 cells and primary CD4+ lymphocytes transduced with these vectors inhibit HIV-1 replication and show a preferential advantage for survival. Although these vectors are poorly mobilized to secondary target cells by wild-type HIV-1, they reduce the infectivity of the wild-type virions escaping inhibition.
表达Rev反式显性负突变体(TdRev)的逆转录病毒载体通过阻止未剪接病毒转录本的核输出抑制HIV-1复制,从而抑制Gag-Pol、Env的合成并降低可用于包装的基因组RNA水平。由于这些有效的抑制机制,生产表达TdRev的基于HIV-1的慢病毒载体一直很困难。在此,我们描述了基于HIV的载体,其中TdRev的表达受Rev表达的负调控。在这些载体中,我们保留了野生型HIV-1 Tat/Rev外显子和内含子结构及其剪接调控模式。第二个Rev外显子发生突变以编码TdRev。在载体生产过程中Rev对TdRev表达的抑制产生了高滴度的载体制剂。通过翻转Tat/TdRev内含子中包含的一个1.2 kb env片段构建了第二个含有额外抗HIV基因(env反义)的载体。用这些载体转导的SupT1细胞和原代CD4+淋巴细胞抑制HIV-1复制,并显示出存活的优先优势。尽管这些载体很难被野生型HIV-1转运到次级靶细胞,但它们降低了逃脱抑制的野生型病毒粒子的感染性。