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Gene Therapy Effective In Treating Parkinson's Disease

March 19th 2011 04:27

gene therapy research parkinsons brain








BOSTON -- Could gene therapy be the key to finding a cure for Parkinson's disease? In a new study, doctors successfully treated Parkinson's patients by injecting genetic material into an area of the brain in charge of motor function.


"It's about 6 millimeters long and (they) infuse basically an altered virus that has a gene in it into this area. So it made an area of the brain less active," said Dr. Samuel Frank, from Boston Medical Center's Department of Neurology.

Frank said certain cells within the brain act like brakes on a car by controlling the way we move our bodies. In Parkinson's, those brakes don't work, causing the uncontrolled tremors and stiffness the disease is famous for.

"This basically put the brakes back on the motor system," Frank said.

After the one-time treatment, patients showed a 23 percent improvement in their symptoms.

"That's pretty dramatic versus taking a medication four or five times a day or having the surgery that leaves a wire in the deep parts of your brain," Frank said.

Parkinson's disease affects an estimated 1 million people in the U.S., mainly adults over the age of 65. This study is the first time gene therapy has successfully treated any neurological disease.

"If we can show that we can treat symptoms based on gene therapy for one disorder, then it has the promise for a lot of different neurological diseases," Frank said.


Frank also said the treatment could be available to patients within five years. The next step is testing the therapy in a much larger population in order to be eligible for FDA-approval.





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