Gene therapy success 'reverses' blindness

Experimental gene therapy trials have improved the vision of four people who suffer from hereditary blindness.

The preliminary results of two independent studies suggest that "repair" genes delivered to the eye might one day cure Leber congenital amaurosis (LCA), a rare disease that strikes about 1 in 80,000 people in the UK, and 2,000 Americans in total.

Equally important, say researchers, the treatments proved safe in the six patients who received the genes – delivered by a disabled virus – via eye surgery.

"This is really an exciting result for gene therapy as a field," says Katherine High, of the University of Pennsylvania Medical School in Philadelphia, who was part of an international team that presented the findings at a conference yesterday.

Another team led by Robin Ali, of University College London, presented similar results.

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