Grant: Operating Grant

Diseases such as retinitis pigmentosa (RP) and Leber`s hereditary optic neuropathy (LHON) can lead to devastating and irreversible blindness. Both of these diseases are hereditary, and to date, a great many genes have been identified that when mutated can cause these diseases. The existence of multiple gene mutations makes the development of effective therapies challenging. This is further complicated by the fact that there are multiple genetic and environmental factors that may also influence the disease process. As we learn more about these diseases, it becomes clearer that combination therapies that target more than one cause or risk factor may be necessary in order to obtain the best possible therapeutic outcome. While we may not know the exact triggers that initiate the disease process, it is generally accepted that many blinding diseases share two common mechanisms: the excess generation of free radicals (referred to as oxidative stress) and the death of the cells in the retina by a process called apoptosis. We hypothesize that a therapeutic strategy that targets both apoptosis and oxidative stress will provide improved clinical outcomes in many types of retinal disease. The current proposal will examine the effectiveness of this strategy in retinitis pigmentosa and in Leber's hereditary optic neuropathy.