Progress in healing eye problems with genetics

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Gene therapy restores sight
By Michelle Robertson
An amazing breakthrough in medical science uses gene therapy to give sight back to people who suffer from genetic blindness. The Oxford Eye Hospital used injections of altered genes to reverse a rare genetic disease that causes progressive loss of vision. They had an almost 100% success rate as all those who took part either “gained or maintained vision”. Professor Robert MacLaren, the ophthalmologist who led the trial, explained, “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 five years ago. The trial has made a big difference to their lives.”

As a result of this success, the European Medicines Agency has approved the first gene therapy treatments for other more common eye diseases. Dr Neil Ebenezer, Director of Research, Policy and Innovation at Fight for Sight, said: “All research breakthroughs are made by standing on the shoulders of others. This technique could transform how we treat diseases and could have broad applicability to a range of other conditions.”


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