Efficient gene transfer of HIV-1-specific short hairpin RNA into human lymphocytic cells using recombinant adeno-associated virus vectors

Mol Ther. 2004 Mar;9(3):396-402. doi: 10.1016/j.ymthe.2003.11.025.

Abstract

The cellular introduction of short, interfering RNA leads to sequence-specific degradation of homologous mRNA, a process termed RNA interference (RNAi). Here, we report that recombinant adeno-associated virus 2 (rAAV-2) can be used to transfer short hairpin (sh) RNA expression cassettes genetically into human cells. HIV-1 replication was suppressed by >95% in H9 cells and primary human lymphocytes that expressed shRNA targeting the first exon of the viral transactivator protein tat compared to control cells. rAAV-2 integrated stably into the host genome, leading to long-term expression of tat shRNA. Our findings demonstrate the utility of rAAV-2 for the genetic transfer of shRNA expression cassettes into human cells, providing an alternative to using retroviral vectors as RNAi delivery systems.

Publication types

  • Research Support, Non-U.S. Gov't
  • Research Support, U.S. Gov't, P.H.S.

MeSH terms

  • Blotting, Northern
  • Cell Line
  • Dependovirus / genetics*
  • Enzyme-Linked Immunosorbent Assay
  • Exons
  • Gene Products, tat / genetics
  • Gene Transfer Techniques*
  • Genetic Therapy / methods*
  • Genetic Vectors*
  • HIV-1 / genetics*
  • Humans
  • Lymphocytes / metabolism*
  • Lymphocytes / virology*
  • Models, Genetic
  • Polymerase Chain Reaction
  • Promoter Regions, Genetic
  • RNA / chemistry*
  • RNA Interference
  • Retroviridae / genetics
  • Time Factors
  • Transcriptional Activation
  • tat Gene Products, Human Immunodeficiency Virus

Substances

  • Gene Products, tat
  • tat Gene Products, Human Immunodeficiency Virus
  • RNA