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Gene Therapy for Parkinson's Disease

by Rathi Manohar on March 17, 2011 at 11:10 PM
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 Gene Therapy for Parkinson's Disease

Parkinson's disease, can be successfully treated through gene therapy as proved by clinical trials, according to a research report in the US, published in The Lancet Neurology.

Researchers used a virus to introduce genes into brain cells, and when this was done, the symptoms of the disease get reduced in nearly 50 per cent of the patients. The disease manifests itself in uncontrolled shaking, stiffness and slow movement as part of the brain dies. Drugs and brain stimulation are used to keep the symptoms under control, as there is really no cure for the disease. The disease affects old people, mostly those who are over fifty. †

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In this study, the reduced level of a chemical - GABA - in the subthalamic nucleus of the brain, which is associated with the disease, was targeted.

A virus was created which was injected into the cells with a gene to increase GABA production.

22 patients had the virus injected into their brains while 23 patients were just made to believe that they had had the virus injected.
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Their motor function was then scored over six months.

Patients who had gene therapy showed a 23.1% improvement in their motor score, while the others improved by 12.7%.

Although the researchers have claimed success, experts in the UK talk of exercising caution as the study show very minimal difference in the groups under study. Professor Nicholas Mazarakis, who is a specialist in gene therapy at Imperial College London, told the BBC, "This result should be taken with some caution though, as it constitutes a rather small mean improvement, only 10.4... In addition, the absence of significant improvements in other secondary outcome measures such as dyskinesia and quality of life between the two groups, warrants further long-term evaluation of this treatment in more patients."

Concerns over safety issues, a lack of information on the efficacy of the therapy and how enduring it would be warrants more research on gene therapy.†

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Source: Medindia
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