Gene Therapy and Animal Models for Retinal Disease

Those plagued by retinal diseases are often robbed of their vision, as often, effective treatments do not exist. Knowledge of the pathophysiology of retinal diseases stems from research on available animal models. Gene therapy may be useful for both genetic and acquired retinal diseases. This review will focus on retinal diseases for which gene therapy has demonstrated promise. The diseases are presented in order of the age at which they are generally first symptomatic and include retinopathy of prematurity, Leber congenital amaurosis, mucopolysaccharidoses, retinoblastoma, retinitis pigmentosa, diabetic retinopathy, glaucoma and age-related macular degeneration. We will describe the animal models used to study these disorders and emphasize the progress that has been made in using gene therapy for the treatment of retinal disease.