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Gene Therapy Found Effective in Monkeys With Parkinson’s

Last Updated: October 16, 2009.

Injecting three genes critical for producing dopamine into the brains of a monkey model of Parkinson's disease corrects the motor problems without inducing the abnormal involuntary movements seen with other treatments, according to a study in the Oct. 14 issue of Science Translational Medicine.

FRIDAY, Oct. 16 (HealthDay News) -- Injecting three genes critical for producing dopamine into the brains of a monkey model of Parkinson's disease corrects the motor problems without inducing the abnormal involuntary movements seen with other treatments, according to a study in the Oct. 14 issue of Science Translational Medicine.

Bechir Jarraya, M.D., from CEA in Fontenay-aux-Roses, France, and colleagues simulated Parkinson's disease in macaque monkeys by treating them with a drug that destroyed their dopamine-producing neurons, then injected them in the brain with the three genes critical for dopamine synthesis.

The researchers found that the gene therapy restored extracellular dopamine to about 50 percent of normal levels and corrected the motor defects for one year without inducing the abnormal involuntary movements known as dyskinesias, which have been previously observed after treatment with drugs such as L-dopa that transiently raise brain dopamine levels. Significant improvements in akinesia and posture were observed starting two weeks after injection.

"Gene therapy-mediated dopamine replacement may be able to correct Parkinsonism in patients without the complications of dyskinesias," Jarraya and colleagues conclude.

Several authors are employees of Oxford BioMedica, which is conducting a Phase I/II safety and efficacy trial of the gene therapy in Parkinson's patients.

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