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Gene therapy shown to protect and reverse the debilitating effects of Parkinson's disease in pre-clinical studies

Researchers at Rush-Presbyterian-St. Luke's Medical Center, Chicago, and Lausanne, Switzerland, have successfully used gene therapy to reverse the anatomical, cellular changes that occur in the brains of primates with Parkinson's disease. The researchers also report success in preventing the disease from progressing and reversing functional deficits or symptoms associated with the disease in monkeys displaying early signs of Parkinson's disease.

Results of the research are published in the Oct. 27 issue of the journal Science.

Rush scientists used a special virus (lenti-GDNF), which was developed by colleagues in Switzerland to deliver the gene for glial-derived neurotrophic factor (GDNF) directly to the brain cells of monkeys.

GDNF is a nutrient that strengthens and protects brain cells that would normally die in this disease. GDNF also increases production of the chemical neurotransmitter dopamine, which sends signals in the brain that enable people to move smoothly and normally. The loss of dopamine in the brain causes the symptoms of Parkinson's disease.

"The lentiviral vector delivery system was effective in getting GDNF to the specific sites needed to rescue the cells and enhance the production of dopamine. By giving GDNF, we can stimulate dopamine production and prevent both the structural and functional consequences of cell degeneration that are characteristic of Parkinson's disease," said Jeffrey H. Kordower, PhD, the principal author of study. He is professor of neurological sciences and director of the Research Center for Brain Repair at Rush-Presbyterian-St. Luke's Medical Center in Chicago. Study collaborators at the Lausanne University Medical School are Patrick Aebischer, MD, PhD, and Nicole Dglon. PhD.

Two groups of monkeys were involved in the research. The first group was studied to determine if and how the lentivirus delivery system affected the anatomy of the aged brain cells. The second group was
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Contact: John Pontarelli
jpontare@rush.edu
312-942-5949
Rush University Medical Center
26-Oct-2000


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