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Behold the Possibilities of Emerging Therapies

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.

  • Gene replacement therapy, also known as augmentation therapy, works by inserting normal copies of the mutated gene into the host cells

  • Gene editing corrects the gene variant directly within the host DNA

  • RNA (ribonucleic acid) editing edits the RNA, not the DNA

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See what’s possible when you test or retest.
Ask your eye specialist about genetic testing today.