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Gene therapy ‘reduces Parkinson’s symptoms’

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Parkinson’s disease sufferers have been given fresh hope that a cure could be found in the future after a study revealed that a particular type of gene therapy can significantly improve motor function in patients.

A research team from the Feinstein Institute for Medical Research in New York discovered that the injection of the glutamic acid decarboxylase (GAD) gene directly into the brain was safe and could lead to significant improvement in the condition of those who have not responded to drug treatment.

It is thought that the findings, which are published in the current edition of the Lancet Neurology journal, show the potential for gene therapy to also alleviate the symptoms of other brain disorders as the GAD gene produces a brain chemical called GABA, which is responsible for coordinating movement.

The treatment works by inserting the gene into a part of the brain called the subthalamic nucleus with the aim of increasing GABA production and therefore restoring motor function.

Patients who underwent the gene therapy and had been off their medication for 12 hours enjoyed a 23.1% improvement in motor function on average, compared with a 12.7% increase in the placebo group. More importantly, all patients survived the surgery with only minimal side effects, such as headaches and nausea.

They also showed improvement in other clinical assessments of motor function, particularly those with advanced Parkinson’s disease who are resistant to drug treatment.

Andrew Feigin and the research team behind the gene therapy trial concluded: “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.”

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