Wall Street Journal
January 15, 2014
by Gautam Naik

Scientists have improved the vision of a small number of patients suffering from a rare and incurable eye disease by replacing a defective gene with a healthy one—a boost for a technique known as gene therapy.

The patients have choroideremia, a degenerative disease caused by defects in a single gene that leads to blindness and affects 1 in 50,000 people. In an early-stage trial published Wednesday in the Lancet, the researchers used a deactivated virus to safely ferry billions of healthy, lab-made versions of the gene into the retina. That appeared to restore the function of light-sensitive cells, which the disease impairs.

“We were surprised by the magnitude of vision improvement” in the patients, said Robert MacLaren, a professor of ophthalmology at the University of Oxford in England and leader of the clinical trial.

The experiment marks one of the first times that gene therapy has targeted the main light-sensing cells in the retina. It thus offers a possible route for treating far more common causes of blindness that affect the same cells, such as retinitis pigmentosa and age-related macular degeneration.

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