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Gene therapy for Parkinson's disease moves forward in animals

An international team of scientists has used gene therapy in two separate studies to renew brain cells and restore normal movements in monkeys and rats with a drug-induced form of Parkinson's disease.

The research, detailed online in the scientific publications Brain and The Journal of Neuroscience, essentially describes one strategy to halt Parkinson's disease at its onset and another strategy to treat the devastating side effects that occur when treating the disease in its later stages.

By inserting corrective genes into the brain, scientists studying small monkeys called marmosets prevented brain damage by producing therapeutic levels of a protein that helps nourish brain cells, said Ron Mandel, Ph.D., a scientist with the University of Florida's McKnight Brain Institute and Genetics Institute who was part of the research team.

The protein, called GDNF, short for glial cell line-derived neurotrophic factor, is believed to preserve brain cells and could provide protection against Parkinson's disease. But its use has been debated since trials in humans ended last year without showing clinical improvements.

Amgen, the world's largest biotechnology company, conducted the trials and later halted use of the drug because of safety concerns, creating an outcry from hopeful Parkinson's patients.

But the gene therapy used in monkeys represents a different way to deliver the GDNF to the brain, causing the body to produce it naturally. It also produces more manageable levels of the protein in the brain.

"Our strategy is a neuroprotective concept and would only be amenable for early stage patients to keep a good quality of life. It would be a huge change in the way treatment is done," said Mandel, a neuroscientist in UF's College of Medicine. "We know the GDNF protects the neurons in primates from the model that we use, so that's good. We now know we can use very low doses that are still effective, so that's good. But we need a s
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Source:University of Florida


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