UNMET NEED
Millions of North Americans suffer from irreversible vision loss due to retinal degenerative diseases such as retinitis pigmentosa, age-related macular degeneration, cone-rod dystrophies, Leber congenital amaurosis, Stargardt disease, and Usher syndrome. The common cause sight impairments in these diseases is progressive death of the light-sensing cells of the retina, the rod and cone photoreceptors. While rod photoreceptor degeneration leads to night blindness and reduced peripheral vision, it is the loss of cones that is the most devastating to patients because these cells provide the most-important daylight and high acuity macular vision in humans. Although there are currently some treatments available to slow disease progression and cone loss in some conditions, there are no cures available for any retinal degenerative diseases.