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Gene therapy shows early promise as treatment for blindness

An initial gene therapy study published Thursday has shown that a genetic eye disease may be treatable. A half dozen patients afflicted with the extremely rare disease had improved vision after defective genes in their retinas were replaced with healthy ones.

The patients have choroideremia, a rare disease caused by a genetic mutation that leads to blindness. About 1 out of every 50,000 people are affected by it. Some who have the disease may be born with good vision, but begin to experience problems with light sensitivity and peripheral vision as they age.

In the trial, deactivated viruses implanted with healthy versions of the gene were injected into the patient’s retinas, resulting in sustained eyesight improvement for all six patients over a six month period. .

Oxford University professor Robert MacClaren said that he couldn’t have hoped for a better result.

“Now that we know the virus is doing what it should do, we need to follow the patients up and see if the improvement is correlated with the stopping of degeneration.We’re hoping to help more people with this disease, because it’s a terrible diagnosis to have,” MacClaren said in an interview with FoxNews.com. “The concept of this gene therapy can be applied before the onset of vision loss. So in this case, genetic modification is a good thing, not necessarily a bad thing.”

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