Gene therapy hope for eye disease

Thursday January 16th, 2014

British doctors have successfully used gene therapy to improve the eyesight of people with an incurable disorder, it was announced today.

So far the treatment has been tested on six patients with choroideremia by researchers at Oxford University.

The genetic condition, untreatable until now, affects about one in every 50,000 people and leads to blindness.

Two patients experienced "substantial improvement" following treatment, the researchers say. One could read an extra three lines on an eye chart.

The other four also showed improvements in sight. The patients were aged between 35 and 63.

Researcher Professor Gordon McLaren has reported his findings in The Lancet.

Professor McLaren said today: "This is first time that gene therapy has been used to treat patients with normal visual acuity before the onset of clinically significant retinal thinning.

"Our findings hold great promise for gene therapy to prevent loss of sight in other retinal diseases such as age-related macular degeneration."

Writing in the journal, José Sahel from the Institut de la Vision, Paris, France, warns that the researchers have yet to show the treatment prevents long-term degeneration of the eye.

The Lancet 16 January 2104 [abstract]

Tags: Eye Health | Genetics | UK News

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