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HOMENEWSRESEARCHERS SAY THERAPY PREVENTS ALZHEIMER’S IN MICE RESEARCHERS SAY THERAPY PREVENTS ALZHEIMER’S IN MICE
Gene therapy in mice resulted in a "revved up" protein disposal process, protecting brain cells from dying and consequently preventing the development of Alzheimer's disease.
MARCH 11, 2011 – GENE THERAPY THAT BOOSTS the ability of brain cells to consume toxic proteins prevents development of Alzheimer’s disease in mice predestined to the illness, according to researchers at Georgetown University Medical Center (GUMC).
The researchers say the one-time treatment could potentially have the same function in people at the beginning stages of the disease.
The study, published online in Human Molecular Genetics, demonstrates that giving mouse brain cells an extra type of protein promotes removal of particles believed to destroy neurons from the inside out. This “revved up” protein disposal process, as the researchers call it, prevents the cells from dying and spewing proteins into the brain that stick together and clump into plaque.
GARBAGE IN, GARBAGE OUT
“At its core, this is a simple garbage in, garbage out therapy,” says the study’s lead investigator, GUMC neuroscientist Charbel E-H Moussa. “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.”
Moussa believes diseases such as Alzheimer’s start when neurons are unable to get rid of toxic peptide called amyloid beta that begins to build up inside neurons. He says the idea remains controversial, but is rapidly gaining acceptance among neuroscientists.
Previous research by Moussa has found that is a buildup of amyloid beta is present among victims of Alzheimer’s, Parkinsonism (including Dementia with Lewy Bodies) and Down’s syndrome.
If human 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.
Co-authors of the study, supported by the National Institutes of Health, include Preeti Khandelwa and Hyang-Sook Hoe of GUMC, and Alexander Herman and William Rebeck.
More information on this research is available on the GUMC website.
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