Hong Kong Med J 2013;19(Suppl 5):S24-6
Triple combination lentiviral vector- based haematopoietic stem cell gene therapy for inhibition of drug- resistant HIV-1
Y Chen, BC Wong, CH Li, X Zhang, SS Lee
School of Biomedical Sciences, The Chinese University of Hong Kong
1. A triple combination lentiviral vector Lenti-TriC was successfully constructed, in which lhRNA targeting HIV-1 was driven by the U6 promoter, shRNA targeting CCR5 was driven by the H1 promoter, and TRIM5α and EGFP were bicistronically expressed under the control of EF-1α promoter.
2. Lenti-TriC expressed lhRNA (targeting HIV-1) and inhibited HIV-1 gene expression. Lenti-TriC expressed shRNA (targeting CCR5) and significantly inhibited CCR5 gene expression.
3. Lenti-TriC inhibited HIV-1 replication in human CEM T cells, primary PBMC cells, and macrophages.
4. Lenti-TriC did not affect the differentiation potential of CD34+ cells.
5. Lenti-TriC could be used for haematopoietic stem cell gene therapy to inhibit HIV-1 replication.