Experimental gene therapy for Parkinson’s clears safety hurdle

By: Agence France-Presse January 10, 2014 8:15 AM

Singer Linda Ronstadt says Parkinson's Disease stole her voice (AFP file)

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PARIS -- A closely watched prototype therapy to inject corrective genes into the brain to treat Parkinson's disease has cleared an important safety hurdle, doctors said Friday.

Tested on 15 volunteers with an advanced form of the degenerative nerve disease, the technique proved safe and the results were encouraging, they said.

The experiment aims to reverse the lack of a brain chemical called dopamine, which is essential for motor skills.

It entails tucking three genes into a disabled horse virus of the family lentiviruses.

The modified virus is then injected directly into a specialized area of the brain, where it infiltrates cells. In doing so, it delivers corrective pieces of DNA, prompting defective brain cells to once again start producing dopamine.

Called ProSavin, the British-designed treatment was authorized for tests on humans after it was tried on lab monkeys.

It is being closely watched by specialists to see if it works better than conventional therapies -- the veteran drug levodopa or electrical stimulation of the brain -- or another experimental gene technique, which uses a modified cold virus.

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Experimental gene therapy for Parkinson's clears safety hurdle