In vivo gene transfer to the mouse eye using an HIV-based lentiviral vector; efficient long-term transduction of corneal endothelium and retinal pigment epithelium

We have evaluated the transduction profiles of an HIV-based lentiviral vect or delivered regionally to ocular tissues in vivo. Following subretinal inj ection, a green fluorescent protein (GFP) reporter gene was efficiently and stably expressed in retinal pigment epithelial (RPE) cells. Limited transd uction of adjacent photoreceptors occurred in newborn mice, but was ineffic ient in adult animals. Injection of the vector into the anterior chamber re sulted in efficient and stable transduction of corneal endothelial cells. E fficient in vivo gene transfer into cells of the corneal endothelium and re tinal pigment epithelium by lentiviral vectors may therefore offer a valuab le approach to the treatment of disorders of the cornea and outer retina.