Gene therapy injection could improve sight for thousands

A SINGLE injection which restores the sight of people with genetic blindness has been successfully tested for the first time.

Scientists have hailed the results of the world’s first gene therapy trial for blindness after 14 patients who have one of the most common inherited forms of the disease experienced “significant” gains or arrested deterioration of their vision.

Oxford University researchers working with the NHS injected a virus containing a missing gene into the rear of the eyes of patients suffering from choroideremia.

Of the 12 who received the treatment without suffering any complications, all have demonstrated improved or maintained vision since having the injection, some as long as five years ago.

Among the best results were patients who gained more than one line of vision on the standard eyesight letters chart. Without the therapy, three quarters of the patients would have been expected to deteriorate.

Gene therapy works by altering inherited diseases at the level of DNA and, if successful, a single treatment can have lifelong effects.

Choroideremia is one form of a spectrum of inherited eye diseases known as retinitis pigmentosa, which have become the most common cause of untreatable blindness in young people.

It affects approximately 1,200 people in the UK, but the scientists who led the trial have said their success could pave the way for the adoption of gene therapies for blindness more widely.

Published in the journal Nature Medicine, the study has enabled the start of a larger trial with more than 100 patients across Europe and North America, run by Oxford University spin-out company Nightstar Therapeutics.