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Is gene therapy a cure for Alzheimer’s disease? The Alzheimer’s Society comments

Monday 10 October 2016

Following recent media headlines claiming that gene therapy might be able to cure Alzheimer’s disease, our friends at the Alzheimer’s Society (UK) commented on the stories and the original study behind the headlines. Here is what they had to say:

New research published today (Monday 10 October 2016) in the journal of Proceedings of the National Academy, holds potential to treat Alzheimer’s disease.

The research shows how the development of Alzheimer’s disease can be prevented in mice by delivering a gene into the brain using a specially adapted virus.

Scientists from Imperial College London suggest that the gene, called PGC1-alpha, may prevent the formation of a protein called amyloid-beta. Amyloid-beta is the main component of the amyloid plaques, the sticky clumps of protein found in the brains of people with Alzheimer’s disease. Mice that were treated with the gene therapy showed less amyloid in their brain and performed better on memory tests.

Commenting on the research, Dr Doug Brown, Director of Research and Development at Alzheimer’s Society said:  

“This research takes a new approach to tackling the underlying causes of Alzheimer’s disease – using a technique called gene therapy to interrupt the production of amyloid protein, one of the key hallmarks of Alzheimer’s. So far, potential treatments that directly target amyloid build-ups in the brain have mostly had disappointing results in clinical trials, whereas this study could pave the way for a new plan of attack.”

“It’s still early days for this gene therapy approach – while it has shown promise in mice, we’re still not sure whether this will be suitable for people. Using gene therapy could prove to be useful further down the line in dementia research and we look forward to seeing if this will be an effective way of developing future treatments.”

Link to original study: