Gene Therapy Breakthrough Could ‘Cure’ Blindness

Posted: January 16, 2014 at 10:47 am

By Thomas Moore, Health and Science Correspondent

Patients suffering from an inherited form of blindness have for the first time had their vision dramatically improved by gene therapy.

The first six patients to be given the experimental injections into the back of the eye were all able to see better in dim light.

And two of them were able to read more lines on an eye chart.

The patients were born with a genetic form of blindness called choroideremia, which affects 1 in 50,000 people, most of them boys, who start to lose their vision in late childhood.

But in a groundbreaking clinical trial, doctors at the Oxford Eye Hospital injected a harmless virus that had been engineered to carry a working copy of the gene that sufferers lack.

The trial was intended to confirm that the injections did not damage the delicate light-sensing cells in the retina.

But the gene therapy had an unexpected therapeutic benefit - and three more patients have now been treated with a higher dose.

Professor Robert MacLaren of Oxford University, who led the trial, said: "In truth we did not expect to see such dramatic improvements in visual acuity.

"It is still too early to know if the gene therapy treatment will last indefinitely, but we can say that the vision improvements have been maintained as long as we have been following up the patients, which is two years in one case."

Original post:
Gene Therapy Breakthrough Could 'Cure' Blindness

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