Gene therapy for Parkinson's

Wednesday September 23rd, 2015

British scientists have unveiled a radical new therapy for Parkinson's disease - based on gene therapy.

The treatment has so far been tested on rats - and the researchers say it is a long way from being usable on human patients.

It uses a virus to deliver a genetic switch to cholinergic neurons. This is then activated with a drug - with the effect of stimulating the neurons.

The research team, based at Imperial College, London, and Newcastle University, say their findings confirm the role of cholinergic neurons in Parkinson's disease and the benefits of developing treatments to target them.

Their findings have been published in the journal Molecular Neurodegeneration.

Researcher Dr Ilse Pienaar said: “This study confirms that cholinergic neurons are key to the gait problems and postural instability experienced by advanced Parkinson’s disease patients.

"It also suggests that it’s possible to target those cells that remain to compensate for those that are no longer functioning effectively, possibly due to weak communication between nerve cells.

"If we can transfer this technique into people, we believe this could help patients regain mobility.”

Fellow researcher Dr Joanna Elson, a geneticist in Newcastle, said: “The structure we studied is complex, very complex. Despite this complexity and the intricacy of the techniques and the brain region analysed, the results are exciting because of the potential to advance patient treatment.

"This paper will help us understand how deep-brain stimulation works, but more importantly it is a step towards offering less invasive treatment options to patients with Parkinson’s and other neurodegenerative disorders.”

Pharmacogenetic stimulation of cholinergic pedunculopontine neurons reverses motor deficits in a rat model of Parkinson’s disease. Molecular Neurodegeneration 23 September 2015; doi: 10.1186/s13024-015-0044-5

Tags: Brain & Neurology | Genetics | UK News

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