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

D Boden, O Pusch, F Lee, L Tucker, B Ramratnam - Molecular Therapy, 2004 - cell.com
D Boden, O Pusch, F Lee, L Tucker, B Ramratnam
Molecular Therapy, 2004cell.com
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 …
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.
cell.com