| Vision Restored Through Gene Therapy |
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The promise of gene therapy is renewed with the announcement that partial vision has been restored to three individuals with retinal disease.
Although AMD patients are not part of this clinical trial, the success generated here will lead to research that will include people with AMD.
The first three patients to be enrolled in this Phase I clinical trial have Leber congenital amaurosis, a severe form of retinitis pigmentosa. They had severely abnormal vision before entering the study and can now read several lines on an eye chart and are able to see better in dimly lit settings. The Phase I clinical trial will involve a total of nine people and was originally designed to test the safety of the treatment. A Phase II study is being planned. The investigators believe the treatment has the potential to give near-normal vision to children born with this disease. The gene for Leber's is better understood than the genetics of age-related macular degeneration. The research began with a type of dog that is born blind and more than 50 dogs have received the treatment. All are seeing well, including the first dog, who received a single treatment 8 years ago. This research shows great promise, not only for children born with Leber's, but also for people with other retinal disorders like AMD. This is a remarkable breakthrough in gene therapy. May 2008 |
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