Gene therapy produces dramatic improvement in vision

A benign adenovirus armed with a genetic payload was used to correct the sight of a dozen people suffering from a rare genetic disorder called Leber's congenital amaurosis. And their work could have broader implications in fighting macular degeneration.

Only a handful of new cases of Leber's are reported every year. Patients share a defective RPE65 gene, which produces a derivative of Vitamin A the eyes need to be sensitive to light. The research team from Children's Hospital of Philadelphia and the University of Pennsylvania School of Medicine inserted a good copy of the gene in the retina of the worst eye, producing a dramatic improvement in eyesight--especially for the youngest study participants. One 9-year-old boy who was legally blind can now see.

Stephen Rose, chief scientific officer of Foundation Fighting Blindness, called the results "astounding. The big take-home message from this is that every individual in the group had improvement... and there were no safety issues at all." Researchers say they'll explore how this work could be adapted for use in macular degeneration, a condition that affects some 1.25 million people in the U.S.

- here's the press release
- read the article in the Los Angeles Times

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