Gene Therapy for Eye Disease
... • For our gene therapy clinical trials, the surgical procedure
involves injecting the virus under the retina, producing a
temporary retinal detachment
• Recovery time is usually rapid and it is often possible to go
home the same day
• Quality of sight is reduced temporarily until recovery is
complet ...
Gene therapy of the human retina
Retinal gene therapy holds great promise in treating different forms of non-inherited and inherited blindness.In 2008, three independent research groups reported that patients with the rare genetic retinal disease Leber's Congenital Amaurosis had been successfully treated using gene therapy with adeno-associated virus (AAV). In all three studies, an AAV vector was used to deliver a functional copy of the RPE65 gene, which restored vision in children suffering from LCA. These results were widely seen as a success in the gene therapy field, and have generated excitement and momentum for AAV-mediated applications in retinal disease.In retinal gene therapy, the most widely used vectors for ocular gene delivery are based on adeno-associated virus. The great advantage in using adeno-associated virus for the gene therapy is that it poses minimal immune responses and mediates long-term transgene expression in a variety of retinal cell types. For example tight junctions that form the blood-retina barrier, separate subretinal space from the blood supply, providing protection from microbes and decreasing most immune-mediated damages.