PHILADELPHIA, April 28 (UPI) -- The New England Journal of Medicine said gene therapy was used for the first time to restore some sight to patients with congenital blindness.
While the treatment did not give the patients normal eyesight, preliminary results from the clinical trial hold promise for treatment of congenital retinal disease and other retinal diseases, The Children's Hospital of Philadelphia said.
The study, published in the New England Journal of Medicine, was led by the hospital, The University of Pennsylvania, the Second University of Naples, the Telethon Institute of Genetics and Medicine in Italy and several other U.S. institutions.
The scientists used a vector, a genetically engineered adeno-associated virus, to carry a normal version of the gene RPE65 that is mutated in Leber congenital amaurosis. Leber damages light receptors in the retina and usually begins stealing sight in early childhood, causing total blindness in adulthood, the hospital said.
Three patients received the gene therapy through a surgical procedure performed by Dr. Albert M. Maguire. Two weeks after the injections, all three patients reported improved vision in the injected eye. "Patients' vision improved from detecting hand movements to reading lines on an eye chart," Maguire said.
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