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Adeno-Associated Virus Gene Transfer to Mouse Retina

1998; Mary Ann Liebert, Inc.; Volume: 9; Issue: 1 Linguagem: Inglês

10.1089/hum.1998.9.1-81

1557-7422

Robin R. Ali, Martin Reichel, Mahesh de Alwis, Naheed Kanuga, Christine Kinnon, Roland J. Levinsky, David M. Hunt, Shomi S. Bhattacharya, Adrian J. Thrasher,

interferon and immune responses

Ocular gene transfer may provide a means for arresting the retinal degeneration characteristic of many inherited causes of blindness, including retinitis pigmentosa (RP). Previously, we have shown in immunodeficient animals that recombinant adeno-associated virus (rAAV) mediates transduction of photoreceptors as well as the retinal pigment epithelium (RPE) following subretinal injection. In this study we extend these observations and show that highly purified recombinant AAV vectors encoding the reporter gene LacZ transduce photoreceptors in an immunocompetent mouse strain following subretinal injection and efficiently transduce ganglion cells after intravitreal injection. Levels of transduction increase over time. Sublethal γ-irradiation is shown to facilitate this process.