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Gene Therapy Reduces Amyloid Plaques In Mice With Model Of Alzheimer Disease

A new gene therapy technique has been shown to reduce the amount of amyloid-beta protein (which forms the plaques found in the brains of people with Alzheimer's disease) in the brains of mice. In a paper published this week in the open access medical journal PLoS Medicine Matthew Hemming, Dennis Selkoe and colleagues from Harvard Medical School generated a secreted form of neprilysin, a protease that can break down amyloid-beta protein, and used primary fibroblasts to introduce this soluble protease into the brains of mice who had advanced plaque deposition.

The pathologic hallmarks of Alzheimer disease are extracellular plaques of amyloid-beta protein and intraneuronal neurofibrillary tangles of tau protein, both of which accumulate in the regions of the brain that mediate memory and thought. Current treatments for Alzheimer disease affect only the symptoms. Ultimately it is to be hoped that it would be possible to develop disease-modifying interventions that would lower the production of amyloid-beta protein or enhance its clearance.

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