Gene therapy for Parkinson's disease

Gene therapy for Parkinson's disease

Treat Parkinson's disease by gene therapy has proven successful in clinical trials for the first time, U.S. researchers said. This disease causes uncontrollable motor movements, tremor, rigidity and slow movements as part of the brain dies.

Small study in The Lancet Neurology used a virus to add genes to brain cells, resulting in less than half the patient's symptoms. Parkinson's UK warmly welcome this research, but they say more research is still needed. The disease affects 120,000 people in the UK, especially on over 50 years.

There is no cure for Parkinson's disease, although drugs and deep brain stimulation has shown a reduction in symptoms.

Gene therapy (gene therapy)

In patients with Parkinson's, a decline in the levels of chemicals are: GABA is located in the brain known as the nucleus subtalamikus. The researchers created a virus that "infects" cells with genes to increase the production of GABA (gamma aminobutyric acid).

In a trial of 22 patients, virus was injected into their brains, while 23 patients were "sham surgery", to make them think that they had injected the virus, their motor function and then monitored for six months.

Patients who had performed the gene therapy showed an increase of 23.1% at the level of their motor, while carried out with a "sham surgery" increased by 12.7%. Professor Nicholas Mazarakis, who is a specialist in gene therapy at Imperial College London, told the BBC a positive outcome was "very encouraging."

He added: "These results should be taken though with some caution, because even though the increase is rather small, only 10.4%, the clinical motor scale scores between patients who received gene therapy and placebo groups."

There are concerns about the safety of gene therapy. In 1999, Jesse Gelsinger died during the trial in the U.S. and there are cases of leukemia after treatment in France. The authors say the procedure is safe.

Dr Michelle Gardner, manager of the development of Parkinson's research in the UK, said: "This research shows promise of gene therapy for neurological conditions like Parkinson's, but further research is still needed. "We still do not know for how long the benefits of this treatment is over, or whether there may be long-term problems because the virus is inserted into the brain."
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