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Researchers Test New Gene Therapy To Treat Blindness

Researchers in the U.K. have tested a new technique that could cure a rare form of blindness and possibly help treat more common forms, as well.
Posted at 7:53 PM, Jan 16, 2014

Researchers at Oxford University have tested a new technique that could cure a rare form of blindness — and possibly help treat more common forms as well.

The disease is a rare genetic disorder called choroideremia, and there was no treatment for it before now. The researchers used a gene therapy technique to partially restore sight to six people with the disease. (Via Channel 5

The study's lead author told Fox News"Gene therapy is exciting; it's a new type of medicine. ... And what we're doing is it on a very small scale, because we're looking at a very straightforward gene to replace."

Choroideremia is caused by a mutation in the CHM gene on the X chromosome — which means males are most likely to inherit it. It affects the retina and choroid in the eye, which begin to deteriorate and eventually cause complete blindness, usually by middle age. (Via Choroideremia Research Foundation

Those with the disease typically have normal vision during childhood but start to have problems as they age. They begin to notice not being able to see in the dark, then experiencing tunnel-like vision. The tunnel gets smaller and smaller until all sight is lost. 

"Bright sunlight's a problem, glare-y days are a problem, and that small island of sight gets smaller every year. ... This gives me ... that I may not be totally blind in five years." (Via CTV)

The rare disease strikes 1 out of every 50,000 people in the world. And the treatment seems straightforward enough: Just replace the bad gene. (Via Wikimedia Commons / Greyson Orlando

"What they're doing is putting a working copy of that gene, injecting into the eye, and that seems to stop the gradual decline." (Via ITV)

The study showed that even just one injection caused dramatic improvements in a third of patients — progress that has so far been maintained for two years without any side effects. (Via BBC)

Researchers hope the procedure can be used to help treat other, more common forms of blindness in the future.