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Gene therapy restores sight
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A TEAM of US scientists helped three dogs born blind see the world for the first time through gene therapy, an achievement that would someday restore vision in children born with genetic disorders that cause blind.
In a study carried in the May issue of Nature Genetics, scientists from the University of Pennsylvania and Cornell University said the new work was done on Briard dogs, which in the course of long-term breeding by humans have acquired a blinding genetic mutation in the RPE65 gene, which helps convert light into electrical signals in the eye. Mutation in any of a dozen or so others also cause blind.
In the study, thousands of viruses were injected directly into the eyes of three blind Briards. The viruses had been genetically engineered by scientists at the University of Florida to contain healthy versions of the RPE65 gene, which the viruses then delivered to the dogs' ailing retinal cells.
The dogs' left eyes received injections into one part of the eye, an area distant from the retina, but the therapy resulted in no effect. Then the dogs' right eyes received injections directly behind the retina, very close to the retinal pigment epithelial cells where the RPE65 gene does its job. Before long, all three dogs had vision in their right eyes.
However, the work done in puppies three or four months old does not prove the method will work in children unless clinical trials are done in humans. Scientists say the best time to intervene is birth to 12 years old, but conducting experiments on children is widely disagreed in the United States.(SD-Agencies)
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