Scientists using gene therapy to treat Parkinson's disease

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London, Oct 15 (ANI): Scientists from French Atomic Energy Commission's Institute of Biomedical Imaging in Orsay are using gene therapy to treat Parkinson's disease.

While the research conducted over monkeys has shown, trials over human patients are underway.

Currently, the remedy used for Parkinson's disease involves replacing dopamine - the neurotransmitter that is depleted in patients with the disease - by administering the dopamine precursor levodopa, or L-DOPA.

However, over a period of several years, this treatment has debilitating physical and psychological side effects.

During the study, lead researcher and neurosurgeon Stephane Palfi tested gene therapy as a potential treatment.

At first, they gave macaque monkeys a harsh neurotoxin that caused the animals to develop bodily tremors, unstable posture and severe joint rigidity - hallmarks of advanced Parkinson's disease.

The researchers then injected the monkeys' brains with three genes essential for synthesizing dopamine.

The study showed significant improvements in motor behaviour of the monkeys after just two weeks, without any visible adverse effects.

"We don't see any problems in these monkeys," Nature quoted Palfi as saying.

The monkeys did not show any jerky, uncontrolled movements that often occurs prolonged oral L-DOPA treatment.

One animal even exhibited sustained recovery more than 3.5 years later.

Initial results of the study conducted over human patients have also shown promise

Palfi's team has already tested two different doses of the three-gene-containing virus in six human patients, and is now investigating an intermediate dose that matches that used in the monkeys, with corrections for brain size.

The study results are published in journal Science Translational Medicine1. (ANI)

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