Today, the first gene therapy for an inherited retinal disease caused by variants in the RPE65 gene is available
Over 30 clinical trials are either completed or underway for different types of
inherited retinal diseases, such as retinitis pigmentosa,
X-linked retinitis
pigmentosa, Leber congenital amaurosis, achromatopsia, Usher syndrome,
and Stargardt disease. These trials explore the potential of gene therapy and
several other technologies
After receiving your results, your genetic counselor and eye specialist may recommend extra steps to find out if you are eligible for a clinical trial or approved therapy.
Learn more about ongoing trials here.
Science is advancing the future of inherited retinal disease treatment
Many gene therapy innovations are now being studied. Each therapy (below) is designed to affect genes associated with inherited retinal disease in a special way.
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Gene replacement therapy, also known as augmentation therapy, works by inserting normal copies of the mutated gene into the host cells
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Gene editing corrects the gene variant directly within the host DNA
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RNA (ribonucleic acid) editing edits the RNA, not the DNA
See what’s possible when you test or retest.
Ask your eye specialist about genetic testing today.
More answers lie in our genes
While you can’t change what you inherit, you may find answers that can help put you in control of your future.
Discover potential benefits
4 simple steps to test
Know what to expect before, during, and after your genetic test or retest.
View steps to genetic test