UF researchers have developed a new gene therapy that could be used to treat a form of blindness.

The condition, called X-linked retinitis pigmentosa, is passed from mother to son and gradually deteriorates a person's vision. The genetic defect appears early in life, so even though affected children are often born with sight, they progressively lose their vision.

To battle the condition, researchers developed a technique that involves replacing a faulty gene in the eye with a working copy that supplies a necessary protein to light-sensitive cells, which enables the eye to function, said study co-author Dr. William Hauswirth, a UF ophthalmology professor.

"The treatment has a promise to fundamentally change their lives," Hauswirth said. "It's hoped that they can restore a significant portion of their vision and live their life in a much more functional way."

While women carry the trait, which affects about 10,000 people nationwide, they aren't affected as often as males are, he said. He said less than one percent of the patients are women.

UF researchers worked with colleagues at the University of Pennsylvania to clone a working copy of the affected gene inside a virus, which served as a vehicle to transport the gene to the eye.

The treatment cured dogs that had the disease. The injected genes made their way only to the afflicted area of the eye.

Dr. Alfred Lewin, who is also a UF ophthalmology professor and co-author of the study, said learning how to treat the condition could help doctors discover new treatments for common conditions.

"We're working on the rare diseases that are genetically understood," Lewin said, "but if we can develop the technology with this disease, we might be able to come up with treatments for more common diseases."

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Interesting work, it adds to several very interesting therapies involving gene therapy or stem cells that are currently being assessed in different forms of blindness.

Glad to see the role of animal research acknowledged in this report. It would be great if UF could take a more proactive role in educating the public on the role of animal research in developing new medicines, and in responding to the allegations made by certain animal rights extremists. The Speaking of Research blog makes this point very forcefully in a new post.

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