UF researcher: gene therapy effective in animal studies of Parkinson's disease

An international team of scientists has reversed some of the effects of Parkinson's disease in rats with a drug-induced form of the progressive movement disorder.

By inserting corrective genes into the brain, researchers were able to trigger the regeneration of a critical bundle of nerve fibers. The new growth was linked to significant -- but not complete -- recovery of the animals' ability to use their paws spontaneously, said Ronald J. Mandel, a University of Florida scientist who was part of the team and a co-author of a recent Journal of Neuroscience article describing the research.

"For a long time, we had known that if the cells that produce the chemical dopamine died, then you would get the effects of Parkinson's disease. Researchers therefore focused on trying to save those cells. But when we were able to do so, we still saw all the signs of the disease," said Mandel, an associate professor in the College of Medicine's neuroscience department who is affiliated with UF's Brain and Genetics institutes.

"What we've shown here is that you can get real functional recovery by instead targeting the treatment to the forebrain. There you can see a regrowth of the fibers or axons that are connected to the dopamine-producing cells. Without that connection, those cells are incapable of supporting normal movement," Mandel said.

Parkinson's disease has been in the news in recent months with the revelation that actor Michael J. Fox has the disorder, which is characterized by rigidity in the arms and legs, tremors and movement difficulties. Some of the symptoms can be alleviated with medications such as levodopa, a drug that converts to dopamine in the brain, but their effectiveness lessens over time. Surgery also can minimize some effects, but there is no cure for the gradually worsening condition, which afflicts an estimated 1.2 million people in the United States and Canada.

The gene therapy experiments were conducted at Lund Uni

Contact: Victoria White
University of Florida

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