lady having eye examination

Gene therapy for rare eye disease set to be offered on NHS

A gene therapy for a rare inherited eye disorder which can improve vision could soon be available on the NHS.

Today the National Institute for Health and Care Excellence (NICE) has recommended Luxturna (voretigene neparvovec) for use in the NHS in England, making it the first available treatment for an inherited retinal dystrophy.

People with a mutation of the RPE65 gene will be suitable for the new treatment.

In the gene therapy, a healthy copy of the gene is injected directly into the eye so a working protein can be produced. Patients have to have some functioning retinal cells for it to work. The gene should provide the instructions to make a protein that is key to normal vision.

Research has shown that, in the short term that the drug improves vision and prevents the condition from getting worse.

This treatment is only for the treatment of Leber congenital amaurosis type 2 (LCA2) and severe early-onset Retinitis Pigmentosa caused by mutations in a specific gene called RPE65.

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