Gene Therapy

Inherited eye disorders are caused by mutations or mis-spellings in genes that are needed for vision.

The goal of gene therapy is to add a correctly spelled copy of the affected gene to the cells of the eye in order to restore function, and hopefully preserve or perhaps even improve vision. This approach has already been used successfully in people with two types of inherited retinal degeneration, caused by mutations in genes called RPE65 and CHM. In these diseases, vision is lost over time due to progressive dysfunction and death of the light-sensitive cells of the retina.

Subjects treated with single injections of gene therapy drugs designed to deliver correct copies of the RPE65 or CHM genes to the retina experienced stabilization of their vision, and in some cases significant improvement in their visual function. These successes are based on many years of research by investigators around the world. Based on the promising results from these studies, there is optimism that gene therapy can be applied broadly to treat many different genetic forms of inherited eye disease.

Clinical trials of gene therapy for several other genetic forms of inherited eye disease are also in progress, including additional forms of inherited retinal degeneration, and inherited optic neuropathy. Gene therapies for a number of other genetic forms of eye disease have also been shown to be beneficial in laboratory-based studies, and it is hoped that these will come to clinical trials for patients in the next several years. Efforts are also underway to use gene therapies to treat other eye disorders, such as age-related macular degeneration, and additional inherited disorders that involve other organ systems, such as the brain and bone marrow.


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Please note that the information provided on this website is intended as a guide only, and should not replace treatment and advice from your medical or eye care practitioner. If you have any questions regarding your eye condition or potential interventions, please speak to your health care practitioner.

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