Genes and Gene Therapy in Inherited Retinal Disease

Inherited retinal diseases (IRDs) are a leading cause of legal blindness in children and working-age adults, accounting for up to 20% of all blind individuals aged 16 to 64.1 Consequently, though individually rare, IRDs as a whole represent a significant source of morbidity in the general population. Indeed, significant visual impairment is been consistently associated with lower quality of life2,3 and increased mortality.4 Moreover, while many common causes of visual impairment are preventable or treatable, most IRDs currently have no proven cure. Management is largely supportive, and the associated progressive vision loss is frequently permanent and disabling, with significant associated psychosocial and economic burden of disease.