Adeno-associated virus gene transfer to mouse retina

RR Ali, MB Reichel, M De Alwis, N Kanuga… - Human gene …, 1998 - liebertpub.com
RR Ali, MB Reichel, M De Alwis, N Kanuga, C Kinnon, RJ Levinsky, DM Hunt
Human gene therapy, 1998liebertpub.com
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 …
Abstract
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.
Mary Ann Liebert