7th Australasian gene therapy society meeting

• Book ID: 102891690
• Publisher: John Wiley and Sons
• Year: 2011
• Tongue: English
• Weight: 848 KB
• Volume: 13
• Category: Article
• ISSN: 1099-498X
• DOI: 10.1002/jgm.1582

✦ Synopsis

in which both the aptamer and the siRNA portions have potent anti-HIV activities. The envelope glycoprotein is expressed on the surface of HIV-1 infected cells, allowing binding and internalization of the aptamer-siRNA chimeric molecules. The Dicersubstrate siRNA delivered by the aptamers is functionally processed by Dicer, resulting in specific inhibition of HIV-1 replication and infectivity in cultured CEM T-cells and primary blood mononuclear cells.

A third approach uses a PAMAM G5 dendrimer for non targeted delivery of Dicer substrate small interfering RNAs in human CD4+ T-lymphocytes. Our results show efficient nanoparticle formation of G5 dendrimers with our siRNAs, effective delivery to the target cells and the release of siRNAs that are processed by Dicer into functional 21-22mer siRNAs which are incorporated into the RNA induced silencing complex (RISC) and guide sequence specific degradation of the target transcripts.