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First Gene Therapy Trial for a Genetic Cause of Blindness
blindnessFindings published in Nature Medicine reveal positive takeaways from a gene therapy trial for choroideremia, a genetic cause of blindness. Among the 14 participants in the trial, the entire group showed a significant gain in vision. Patients were treated with an injection to the eye that contained a virus hosting the missing gene. Of the 12 patients that had no complications, 100% either gained or maintained vision for at least five years (the time of the last follow up).

“The early results of vision improvement we saw have been sustained for as long as we have been following up these patients and in several the gene therapy injection was over 5 years ago,” said Professor Robert MacLaren, the ophthalmologist who led the trial. “The trial has made a big difference to their lives.” 

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Blind Gain Sight in Gene Therapy Trial


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