Gene Thereapy Experiements Improve Vision In Nearly Blind
For the first time scientists have used gene therapy to dramatically improve sight in people with a rare form of blindness.
In four of the six young people who received the treatment, teams of researchers in the United States and Britain reported sight improvement. Two of the volunteers who could only see hand motions were able to read a few lines of an eye chart within weeks.
“It’s a phenomenal breakthrough,” said Stephen Rose, chief research officer of the Foundation Fighting Blindness.
If successful in larger numbers, experts said, the technique has the potential to reverse blindness from other kinds of inherited eye diseases.
The two teams of scientists, working separately, each tested gene replacement therapy in three patients with a form of a rare hereditary eye disease called Leber’s congenital amaurosis. There’s no treatment for the disease, which appears early in infancy and causes severe vision loss, especially at night.
An estimated 2,000 Americans have the form of the disease they targeted.
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