First Gene Therapy Approved to Treat Inherited Retinal Dystrophy

Gene Therapy Scientist Holding Test TubeOne of the most promising developments in treatment of vision loss is gene therapy. Recent research shows significant advances in the field. For example, in a 2014 study, researchers suggested that specialized proteins can remove genes that code for age-related macular degeneration and replace them with healthy genes. Additionally, in 2017, a team from the University of Iowa published a study using a gene editing tool called CRISPR-Cas9 to isolate a gene in mice that causes specific types of glaucoma. Scientists are even making advancements in the treatment of another disease that can lead to blindness: Leber congenital amaurosis (LCA).

New Treatment for Leber Congenital Amaurosis

Recently, the U.S. Food & Drug Administration approved the sale of the first gene therapy treatment for Leber congenital amaurosis. LCA is an inherited retinal dystrophy that causes blindness. It affects an estimated 1,000 to 2,000 Americans. The disease begins in infancy and slowly causes the retinal cells to die until the patient goes blind.

The therapy is called Luxturna and requires retinal injections of healthy genes that replace diseased genes. Currently, there is no alternative therapy for this disease.

How Luxturna Works to Treat Leber Congenital Amaurosis

Luxturna is only effective in treating patients whose vision loss is caused by the RPE65 gene. To qualify for the treatment, individuals must undergo genetic testing. Luxturna doesn’t help patients regain lost vision, but it helps them see light and the shape of objects, so they don’t have to rely on a service animal or cane.

Healthy vision requires regular visits to an ophthalmologist for yearly comprehensive eye exams. Many degenerative eye conditions lack noticeable symptoms until permanent vision loss occurs, so be proactive in your eye care. Click here to find an ophthalmologist in your area and preserve your vision.

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