Gene therapy trial sees success

World’s first gene therapy trial for choroideremia delivers results

Positive results of the world’s first gene therapy trial for choroideremia have been reported by publication Nature Medicine.

The trial began at Oxford Eye Hospital in 2011 and involved 14 patients receiving an injection into the back of their eyes of a virus containing the missing gene.

Results from the trial showed that there was a significant gain in vision across the group of patients. Out of the 12 patients who had no complications from the treatment, 100% either maintained or gained vision, which remained for up to five years. A total of 25% of untreated eyes sustained vision.

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