Gene therapy clearing toxic proteins in brain may prevent Alzheimer’s disease

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Gene therapy that boosts the ability of brain cells to gobble up toxic proteins prevents development of Alzheimer’s disease in mice that are predestined to develop it, report researchers at Georgetown University Medical Center.

Researchers say the treatment ? which is given just once – could potentially do the same in people at the beginning stages of the disease.

The study, published in Human Molecular Genetics, demonstrates that giving brain cells extra parkin genes promotes efficient and effective removal of amyloid particles believed to be destroying the neurons from the inside. This revved up protein disposal process prevents the cells from dying and spewing amyloid proteins into the brain, where they stick together and clump into plaque, they say.

“At its core, this is a simple garbage in-garbage out therapy, and we are the first to show that this gene attacks amyloid beta inside brain cells for degradation,” says the study’s lead investigator, neuroscientist Charbel E-H Moussa, M.B., Ph.D.

He adds that the strategy may work for other brain disorders. “Many neurodegenerative diseases are characterized by a toxic build-up of one protein or another, and this approach is designed to prevent that process early-on,” he says.

The novelty of Moussa’s work is that he believes diseases like Alzheimer’s starts when neurons are unable to get rid of toxic amyloid beta that begins to build up inside neurons ? an idea that he says remains controversial, but is rapidly gaining acceptance among neuroscientists.

Moussa says the research team has done all the animal work necessary for an application to begin studying the treatment in humans, starting with an analysis of safety.

He adds that if these experiments are successful, the goal will be to use the treatment as early as possible in the course of a neurodegenerative disease. “Our hope is to stop the whole process early on, but if it is later, perhaps we can halt progression,” he says.

Source: Georgetown University Medical Center, USA

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