Gene Therapy for Inherited Retinal Diseases

Candidates for Ocular Gene Therapy
Gene therapy is a new and promising treatment for some people with retinitis pigmentosa and Leber congenital amaurosis. To qualify, you or your child must have two copies of a mutated gene called RPE65 and enough functioning cells left in the retina for the treatment to be effective. Adults and children over one year old can receive the treatment.

Luxturna Injection
Ocular gene therapy is performed in an operating room under anesthesia. An eye surgeon removes the clear gel inside the eye and injects a drug called Luxturna under the retina. It contains a healthy copy of the RPE65 gene. This one-time treatment restores the retina's ability to respond to light, usually beginning a month after the injection. Six days following the first injection, the second eye is treated. 

Ocular Gene Therapy Recovery 
Ocular gene therapy is an out-patient procedure, meaning you or your child can go home the same day. After the procedure, the eye will be covered with a patch for 24 to 48 hours.
 

Comprehensive Eye Exam
Our inherited retinal disease doctors perform an in-depth, dilated eye exam. This includes retinal imaging and electroretinography, a test that measures the electrical response of the eye’s light-sensitive cells. With young children, these tests are often performed under sedation. We also assess visual acuity and your ability to see in low light.

Genetic Testing
If you or someone in your family has an inherited retinal disease, you or your child may be at risk for developing the condition and should be tested. Genetic testing is essential for accurately diagnosing your disease and determining if treatment is possible. Our genetic counselors will ask about your family history and perform specialized genetic testing. 

Sophisticated Retinal Imaging
Our researchers are national leaders in the use of sophisticated imaging technology and interpreting results. Our years of experience and extensive resources allow us to recommend the best treatment options for you or your child.

One of a Handful of Centers to Offer Ocular Gene Therapy
Luxturna, the only FDA-approved gene therapy for inherited retinal diseases, can only be administered at ocular gene therapy treatment centers where retinal specialists, genetic counselors, pharmacists, and nurses have specialized training and experience caring for patients with inherited retinal diseases. Duke is one of the select centers in the U.S. able to offer ocular gene therapy.

Respected Retinal Leaders
Our retinal specialists include nationally renowned experts who deliver lectures, train others on the latest advances in eye care, and help develop treatment guidelines that are followed by eye-care professionals around the world. Our doctors are also recognized research leaders who focus their efforts on learning more about the causes of retinal diseases and developing promising new therapies and imaging methods. The field of retinal surgery was pioneered at Duke by the former chair of our ophthalmology department. 
 
Compassionate, Supportive Environment
To help you and your loved ones manage your retinal condition and maximize your quality of life, we offer patient support services, educational materials, low vision rehabilitation program, and a dedicated social worker.