Specific HIV-1 env gene silencing by small interfering RNAs in human peripheral blood mononuclear cells

Gene Ther. 2003 Nov;10(24):2046-50. doi: 10.1038/sj.gt.3302099.

Abstract

RNA interference (RNAi) is triggered by the presence of a double-stranded RNA (dsRNA) in the cell, and results in the silencing of homologous gene expression by the specific degradation of an mRNA containing the same sequence. dsRNA-mediated RNAi can be used in a wide variety of eucaryotes to induce the sequence-specific inhibition of gene expression. Synthetic 21-23 nucleotide (nt) small interfering RNAs (siRNAs) with 2-nt 3' overhangs were recently found to mediate efficient sequence-specific mRNA degradation in mammalian cells. Here, we show that synthetic siRNAs targeted against the viral structural Env proteins encoded by HIV-1 can specifically suppress the expression of HIV-1 genes. The siRNA-mediated RNAi also had advantages over antisense RNA-mediated inhibition, in terms of both the ease of designing effective antiviral agents and their potency. Especially, our best env-specific siRNAs, E7145 targeted to the central region of the V3 loop and E7490 targeted to the CD4 binding site of conserved regions on gp120, significantly inhibited the HIV-1 gene expression. Furthermore, E7145 and E7490 were effective against HIV-1(NL4-3) replication in PBMCs for a relatively long time (14 days). Therefore, the use of synthetic siRNAs provides a simple, rapid, and cost-effective tool for new anti-HIV-1 gene therapeutics.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • COS Cells
  • Cells, Cultured
  • Gene Silencing*
  • Genes, env / genetics*
  • Genetic Therapy / methods
  • HIV-1 / genetics*
  • HIV-1 / physiology
  • Humans
  • Leukocytes, Mononuclear / virology*
  • Molecular Sequence Data
  • RNA Interference
  • RNA, Messenger / genetics
  • RNA, Small Interfering / genetics*
  • RNA, Viral / genetics
  • Virus Replication

Substances

  • RNA, Messenger
  • RNA, Small Interfering
  • RNA, Viral