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By the time Corey Haas was 7, the retinal disease he was born with had already stolen much of his vision. “He always clung to me or my wife,” said Corey’s father, Ethan Haas. The boy relied on a cane and adults to guide him, and, unable to see blackboard writing, sat in back with a teacher’s aide, large-type computer screen and materials in Braille. Legally blind, Corey was expected eventually to lose all sight. Then, 13 months ago, after his eighth birthday, he underwent an experimental gene therapy procedure, receiving an injection in his left eye.
SAN FRANCISCO -- Gene therapy to counteract retinal degeneration from a rare inherited sight disorder improved vision in all patients, including one child who recovered near normal light sensitivity. Although the 8-year-old boy was the clearest success story, all the children with Leber's congenital amaurosis treated in the study regained sufficient vision to walk unaided, Jean Bennett, MD, of the University of Pennsylvania in Philadelphia, and colleagues reported here at the American Academy of Ophthalmology meeting.

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