Gene therapy takes a step forward
MIL/USA Today, Apr 28, 2008.
April 28, 2008 - For the first time, doctors have used gene therapy to restore vision in patients with a rare and usually incurable form of blindness, researchers announced Sunday.
Experts say the results are a welcome success in gene therapy, a promising idea that has had several major setbacks during the past 15 years.
"It's a breakthrough," says Ronald Crystal, chairman of the department of genetic medicine at New York's Weill Medical College of Cornell University. "It really helps to validate that gene therapy can work and is going to be important."
None of the six patients treated regained normal vision, but four have much better eyesight than before, according to two studies published online in The New England Journal of Medicine.
The patients had a rare inherited disorder called Leber's congenital amaurosis, which begins eroding eyesight at birth and leaves them blind by their mid-20s.
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