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Gene therapy Beneficial in Parkinson's Disease

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Same technology was used by both teams for performing gene therapy, wherein a desired gene is inserted into a common virus called adeno-associated virus, which is known to readily infect humans. This virus has never been shown to cause disease. The genes used by both teams were quite different.

The team, led by Dr. Matthew J During from the Weill Cornell Medical Center in New York, used a gene responsible for the synthesis of enzyme, glutamic acid decarboxylase. This enzyme in turn converts compounds in the cell into the neurotransmitter, GABA that controls muscle movements. Parkinson’s disease has been known to be deficient in GABA in the subthalamic nucleus part of the brain.

Although injecting GABA directly into the brain eases symptoms of the disease, the hormone is quickly cleared, which is therefore a limits its benefits. During's team injected one side of the brain of 12 patients with one of three different concentrations of the gene therapy agent.

All 12 patients were found to have an improvement of at least 25 percent on a conventional scoring system that assesses the severity of Parkinson's symptoms, nine of them showed an improvement of at least 37 percent, while five of them were seen to have an improvement between 40 percent and 65 percent. Moreover the patients have observed these benefits to persist for a year unlike the benefits from a placebo effect which are mostly transient.

During said that sophisticated imaging of the patients' brains showed an increase in metabolism on the side where gene therapy was performed, and the amount of increase correlated with the degree of improvement in symptoms.

Neurologix Inc. of Fort Lee, N.J., a company created by During and his colleagues to commercialize the technology are the manufacturers of the gene therapy agent. The study was funded by Neurologix.

The second study was led by Dr. William J. Marks Jr.
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