A Second Chance At Sight

In what could be a major milestone for gene therapy, for the first time, researchers have used gene therapy to improve vision in blind patients.

Homegrown (for VOXUS) Seattle’s Targeted Genetics conducted part of the study of six patients with an extremely rare form of blindness called Leber's Congenital Amaurosis. According to the study which appears in the New England Journal of Medicine, four of the six people who received gene therapy had some vision restored. If this test is successful on a larger scale, researchers say it could help people with more common types of blindness. Macular degeneration, the leading cause of blindness for those under 20 years old in the United States, affects 1.25 million Americans; the number is expected to grow to 3 million by 2020 as the population continues to age.

Gene therapy involves replacing defective genes with normal versions. The research marks a major milestone for gene therapy, a discipline many scientists find promising but so far has failed to produce a marketable product in the U.S.

-posted by Andrew