Gene therapy for inherited blindness

Leber hereditary optic neuropathy (LHON) is a rare inherited form of blindness that mainly affects younger men. It is usually caused by a spelling mistake (mutation) in the genetic code (DNA).

We used gene therapy to inject a working copy of the mutated code via injection into patients’ eyes, under local anaesthetic.

The treatment was well tolerated and around 80% of patients noticed an improvement in their vision.

This treatment is now being evaluated by the European Medicines Agency (EMA) and the National Institute for Health and Care Excellence (NICE). We have also received funding from the NIHR to conduct another study to see if patients if this treatment benefits patients with LHON who have lost vision for longer than one year.

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