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Safety study indicates gene therapy for blindness improves vision
Date:9/8/2008

GAINESVILLE, Fla. All three people who received gene therapy at the University of Florida to treat a rare, incurable form of blindness have regained some of their vision, according to a paper published online today in Human Gene Therapy.

The patients one woman and two men ranging from 21 to 24 years old with a type of hereditary blindness called Leber congenital amaurosis type 2 volunteered to test the safety of an experimental gene-transfer technique in a phase 1 clinical research study conducted by UF and the University of Pennsylvania with support from the National Eye Institute of the National Institutes of Health.

In this form of LCA disease, photoreceptor cells cannot respond to light because a gene called RPE65 does not properly produce a protein necessary for healthy vision. In the study, researchers used an adeno-associated virus an apparently harmless virus that already exists in most people to deliver RPE65 to a small area of the retina.

Not only were there no ill effects other than routine postsurgical soreness, the subjects said the vision in their treated eyes was slightly improved in dim lighting conditions.

"The patients report seeing brighter areas and perhaps some images, but basically the message is that this is treatment is fully safe," said William W. Hauswirth, Ph.D., a professor of ophthalmology and member of UF's Powell Gene Therapy Center and the UF Genetics Institute.

"One thing we did not do is suppress the patients' immune systems, which was done in two other LCA clinical trials that were under way," said Hauswirth, who began studying the adeno-associated virus as a vehicle to deliver genes into living animals more than 30 years ago. "Theoretically, the idea was that it might be necessary to suppress the immune system because we are using a vector that might activate the body's defenses and cause a harmful response. However, immune suppression itself carries a risk of infection
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Contact: John Pastor
jdpastor@ufl.edu
352-273-5815
University of Florida
Source:Eurekalert

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