Genetics of Blindness And The Inherited Retinal Diseases

On the bright side, there are some promising therapeutic approaches on the horizon. Gene therapy has shown promise in patients with Leber congenital amaurosis, a form of severe, recessively-inherited retinal degeneration. Mouse models of induced pluripotent stem (IPS) cells have demonstrated the feasibility of regenerating photoreceptor cells and/or retinal tissue. These may be on the horizon, but they’re still a long way off. At best, a distant glimmer of hope for the thousands of patients living with retinal disease.