HIV gene therapy

Small interfering RNA (siRNA) can reduce the expression of specific cellular genes and inhibit viral replication, but their use against viral infections has been limited by the lack of an effective method for introducing siRNAs into primary cells. In the January 7 Proceedings of the National Academy of Sciences, Xiao-Feng Qin and colleagues at the California Institute of Technology, Pasadena, USA, show that lentiviral-mediated delivery of small interfering RNA against CCR5 can inhibit HIV-1 infection in human T cells (PNAS, 100:183-188, January 7, 2003).

Qin et al. used a siRNA expression vector derived from FUGW — an HIV-1-based lentivirus vector originally designed for germ-line gene transduction. They observed that with this vector more than 40% of the peripheral blood T lymphocytes could be transduced in vitro. This resulted in a stable expression of CCR5-siRNA and in an up to 10-fold inhibition of CCR5 expression on the cell surface, while the ...