Modulating oxidative stress in the retina using gene therapy as a potential treatment for inherited retinopathies

Abstract

The focus of this thesis was the exploration of genetic modulators of oxidative stress in transgenic mouse models of Retinitis Pigmentosa (RP). This disease can be inherited both dominantly and recessively and can be either X-linked or autosomal depending of the disease causing mutation. Patients of RP gradually lose their peripheral eyesight over years as rod cells die in the retina. Cone cells then begin to die leading to complete blindness. Evidence amassed over the past ten years has suggested that increasing levels of oxidative stress in the retina of these patients is causing cell death of rod and cone photoreceptor cells. In particular, cone cells seem to die secondary to rods as the levels of oxidative stress dramatically increase when rods die. In addition, this increase in oxidative stress appears to be common in many inherited and non-inherited retinal degenerations.