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Gene Therapy for Parkinsons Disease

e areas, on the side of the brain where the genes were infused. The study was designed to inject the genes into one side of the brain. Normally, Parkinsons patients have worsening symptoms on one side of the body.

The novel strategy included packing genes that make an inhibitory chemical called GABA into pieces of viruses that have been rendered non-infectious. They began studying the experimental treatment in Parkinsons patients in 2003. Some patients continue to show improvement. Parkinson's patients have been willing to step up to the operating table for relief from the tremors, stiffness and rigidity that characterize the disease.

Decades ago, surgeons began to make lesions in parts of the brain involved in the disease, which lessened symptoms. Fetal stem cell surgery was pioneered in Parkinson's patients. And in the past decade, the deep brain stimulation has worked in as many as 70 percent of patients who have opted for the surgical procedure. If it doesn't work, the electrodes can be removed.

Gene therapy could be a more natural way to treat the disease, said Dr. Feigin. This important study shows that gene therapy can be performed safely and may benefits patients. The gene therapy technique was developed by Neurologix, a New Jersey-based company.

Parkinson's is a movement disorder caused by a progressive depletion of the brain chemical dopamine in the substantia nigra. These dopamine-containing cells control movement. When 70 to 80 percent of these cells are destroyed, a person develops the first symptoms of disease: tremors, slowed movement, muscle rigidity and problems with balance.

The main medication used in Parkinson's is L-dopa, which replaces dopamine in cells that are still working normally. Over time, the cell death is so massive that the effects of the medication disappear.


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