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Gene therapy shows promise in model of Parkinson's disease

Scientists at the Ecole Polytechnique Federale de Lausanne (EPFL), Switzerland, have conducted novel experiments that might one day lead to gene therapy treatment options for patients with Parkinson's disease.

In research published this week in the Proceedings of the National Academy of Sciences, the research team, led by EPFL President Patrick Aebischer, found that viral delivery of a gene associated with Parkinson's disease protected neurons from degeneration.

Parkinson's disease is a progressive, degenerative neurological disorder in which dopamine-producing neurons in the part of the brain responsible for coordinating muscle movement die or become so damaged that they are no longer able to function. Dopamine is a neurotransmitter, a "chemical messenger" that transfers information from neuron to neuron, ultimately allowing us to use our muscles in a smooth, coordinated way. Disease symptoms, such as tremor, rigidity, difficulty coordinating movement and difficulty with balance begin to manifest themselves when about 80% of a victim's dopamine-producing neurons have died.

Scientists estimate that Parkinson's disease affects 1-3 % of people over the age of 60. In the United States alone, 1.5 million people suffer from Parkinson's disease, and about 60,000 new patients are diagnosed every year. Although it is commonly thought of as a disease of the elderly, 15% of Parkinson's victims are under the age of 50.

Scientists are not sure what causes Parkinson's disease. There is no way to prevent its onset and once diagnosed, it has no cure. Treatment is limited to ameliorating symptoms with a variety of therapies, including dopamine-based drug therapy.

Only about 5-10 % of Parkinson's appears to be inherited, and to date five genes have been implicated in patients with a familial history of the disease. Studies of these inherited forms of Parkinson's have led to insights on its pathogenesis in sporadic
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Contact: Mary Parlange
mary.parlange@epfl.ch
41-21-693-7022
Ecole Polytechnique Fdrale de Lausanne
29-Nov-2004


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