Chatterjee S, Johnson P R, Wong K K
Laboratory of Viral Diseases, National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health, Rockville, MD 20852.
Science. 1992 Nov 27;258(5087):1485-8. doi: 10.1126/science.1359646.
An adeno-associated virus vector encoding an antisense RNA was used to transduce stable intracellular resistance to human immunodeficiency virus-1 (HIV-1) in human hemopoietic and non-hemopoietic cell lines. The antisense targets are present in all HIV-1 transcripts and include the TAR sequence, which is critical for transcription and virus replication, and the polyadenylation signal. Cell lines expressing antisense RNA showed up to 95 percent inhibition of gene expression directed by the HIV-1 long terminal repeat and greater than 99 percent reduction in infectious HIV-1 production, with no detectable cellular toxicity. Because of their efficient transcription and inability to recombine with HIV-1, adeno-associated virus vectors represent a promising form of anti-retroviral gene therapy.
一种编码反义RNA的腺相关病毒载体被用于在人造血细胞系和非造血细胞系中诱导对人类免疫缺陷病毒1型（HIV-1）的稳定细胞内抗性。反义靶标存在于所有HIV-1转录本中，包括对转录和病毒复制至关重要的TAR序列以及聚腺苷酸化信号。表达反义RNA的细胞系对HIV-1长末端重复序列指导的基因表达显示出高达95%的抑制，并且传染性HIV-1产生减少超过99%，且未检测到细胞毒性。由于腺相关病毒载体具有高效转录能力且不能与HIV-1重组，它们代表了一种有前景的抗逆转录病毒基因治疗形式。
