ADENOASSOCIATED VIRUS GENE-TRANSFER TO MOUSE RETINA

Ocular gene transfer may provide a means for arresting the retinal deg eneration characteristic of many inherited causes of blindness, includ ing retinitis pigmentosa (RP). Previously, we have shown in immunodefi cient animals that recombinant adeno-associated virus (rAAV) mediates transduction of photoreceptors as well as the retinal pigment epitheli um (RPE) following subretinal injection. In this study we extend these observations and show that highly purified recombinant AAV vectors en coding the reporter gene LacZ transduce photoreceptors in an immunocom petent mouse strain following subretinal injection and efficiently tra nsduce ganglion cells after intravitreal injection. Levels of transduc tion increase over time. Sublethal gamma-irradiation is shown to facil itate this process.