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Genes are now being used for healing

Posted: Nov 23, 2009 9:07 AM
Updated: Nov 23, 2009 9:07 AM

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Around the world, there are more than 1,500 gene therapy trials going on to treat everything from Parkinson's to blindness to clogged arteries. Could the key to healing be in the body's building blocks?

Dale Turner was barely old enough to read when doctors told him his world would soon go dark. "The doctors said I would be completely blind by age 10," Turner recalled.

He has an inherited form of blindness called Leber's Congenital Amaurosis. A defective gene prevents his retina from producing the nutrients his eyes need. His vision was like looking down a gun barrel.

Turner got used to the fact he'd be legally blind for the rest of his life, until his parents heard about an experimental therapy, injecting new, healthy genes into the eye. "You always have the hope that there will be something for your incurable condition," Turner said. "Growing up, that there will be something for my eyes someday that will allow me to see."

In a trial, doctors injected hundreds of billions of copies of the working gene beneath the retina in the back of the eye. Four out of six patients regained vision.

"The improvement in vision corresponded exactly where the genes were replacing the defective gene," said William Hauswirth, Ph.D., Professor of Ophthalmic Molecular Genetics at the University of Florida College of Medicine in Gainesville, Florida.

To test the success of the surgery, patients in a British study walked through a maze. Before treatment, one patient bumped into walls six times and took almost a minute and a half to get through the course. After surgery, no bumps, and he finished in 14 seconds.

"It's likely the treatment will last for a long time, if not for the life of these patients," Dr. Hauswirth said.

Turner will never forget the first time he went outside after surgery. "I could see colors like never before, and it was just like a blue sky I had never seen before, and I had this feeling, like what have I been missing out on this whole time?" he said.

HIV is one of the latest targets for gene therapy. In a recent clinical trial, scientists found an injection of an anti-HIV gene could make the body resist the AIDS virus.

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