In vivo gene transfer to the CNS using recombinant SV40-derived vectors

Abstract

Background: Gene transfer to the CNS has been approached using various vectors. Objective: We illustrate how SV40-derived vectors may be useful to deliver long-term gene expression to the brain, locally or diffusely. Results/conclusion: SV40-derived vectors transduce neurons and microglial cells. The potential utility of both localized and widespread gene delivery in treating neuroAIDS and other CNS diseases characterized by excessive oxidative stress is demonstrated. Finally, direct injection of rSV40 vectors into rat femoral bone marrow (BM) led to transgene expression in CNS neurons and microglia, mostly in the dentate gyrus and in the periventricular subependymal zone, suggesting that BM-derived cells may be progenitors of some CNS cells in adult animals, and that gene delivery to BM may allow transgene expression in newly formed neurons.

Keywords::

• AIDS
• brain
• gene therapy
• gp120
• HIV
• stem cells
• SV40