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Gene therapy hope for Parkinson's disease

Gene therapy hope for parkinson s disease Patients showed improved motor function

Gene therapy has been shown to improve the symptoms of patients with Parkinson's disease.

Parkinson's disease is a progressive incurable condition which affects the brain and nervous system, causing shaking, muscle stiffness and difficulty moving. Around 120,000 people in the UK are affected by the condition.

Research has already shown that Parkinson's disease patients have substantially lower levels of a neurotransmitter - GABA - in the part of the brain known as the subthalamic nucleus. GABA is responsible for coordinating movement.

In the trial, a team of scientists used an inert modified virus carrying a gene for glutamic acid decarboxylase (GAD) to infect brain cells in the subthalamic nucleus. They hoped the GAD gene would instruct brain cells to start making more GABA.

They injected the virus into the brains of 22 patients with the disease while another 23 patients received "sham surgery", which included pretending to drill into the patients skull.

The motor function of all patients was then monitored at regular intervals up to 6 months after surgery.

The findings showed that patients who had gene therapy had an average 23 per cent improvement in motor function compared with 12.7 per cent in those given the sham surgery.

Andrew Feigin and colleagues at The Feinstein Institute for Medical Research in New York wrote: "This study... justifies the continued development of AAV2-GAD for treatment of Parkinson's disease... and shows the promise of gene therapy for other neurological disorders."

The findings are published in the journal Lancet Neurology.

This article was published on Thu 17 March 2011

Image © James Steidl -

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