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Positive results of the world’s first gene therapy trial for a genetic cause of blindness known as choroideremia have been reported in Nature Medicine.

Image credit: Prof Robert MacLaren

The trial involved 14 patients receiving a single injection into the back of the eye of a virus containing the missing gene and began in 2011 at the Oxford Eye Hospital - part of the Oxford University Hospitals NHS Foundation Trust. By the end of the study there was a significant gain in vision across the group of patients as a whole.

Furthermore, of the 12 patients who received the treatment without any complications, 100% either gained or maintained vision in their treated eyes, which was sustained for up to 5 years at the last follow up. During this time only 25% of the untreated eyes which acted as controls maintained vision. The gene therapy treatment was generally well tolerated and there were no significant safety concerns.

Read more (University of Oxford website)

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