Yamada O, Yu M, Yee J K, Kraus G, Looney D, Wong-Staal F
Department of Medicine, University of California, San Diego, La Jolla 92093-0665, USA.
Gene Ther. 1994 Jan;1(1):38-45.
T-cell lines (Jurkat and Molt-4) were transduced with retroviral vectors containing a hairpin ribozyme that targets a conserved sequence in the 5' transcribed leader sequence of human immunodeficiency virus (HIV) type 1. Stable cell lines were generated which constitutively and persistently expressed the ribozyme gene driven by either the Moloney retroviral long terminal repeat (LTR) or an internal human tRNA(val) promoter. There was no apparent deleterious effect of long-term ribozyme expression on cell proliferation or viability. Cells expressing ribozyme were resistant to challenge from diverse strains of HIV, including an uncloned clinical isolate. No reverse transcriptase activity or virus infectivity was detectable in the culture supernatants of Jurkat cells expressing the ribozyme driven by the tRNA(val) promoter up to 35 days after challenge with HIV-1/HXB2. Expression of the ribozyme also significantly decreased (by approximately 50- to 100-fold) the efficiency of incoming virus to synthesize viral DNA. These and previously reported results indicate that transfer and expression of the ribozyme gene interfere with both early and late events in the HIV replication cycle and confer long-term resistance to HIV-1 infection.
用含有靶向人类免疫缺陷病毒1型(HIV-1)5'转录前导序列中保守序列的发夹状核酶的逆转录病毒载体转导T细胞系(Jurkat和Molt-4)。构建了稳定细胞系,这些细胞系组成性且持续性地表达由莫洛尼逆转录病毒长末端重复序列(LTR)或人tRNA(val)内部启动子驱动的核酶基因。长期核酶表达对细胞增殖或活力没有明显的有害影响。表达核酶的细胞对多种HIV毒株的攻击具有抗性,包括未克隆的临床分离株。在用HIV-1/HXB2攻击后长达35天,在表达由tRNA(val)启动子驱动的核酶的Jurkat细胞的培养上清液中未检测到逆转录酶活性或病毒感染性。核酶的表达还显著降低(约50至100倍)了进入病毒合成病毒DNA的效率。这些以及先前报道的结果表明,核酶基因的转移和表达干扰了HIV复制周期中的早期和晚期事件,并赋予对HIV-1感染的长期抗性。