Rodent of the Week: Gene therapy stops Alzheimer’s in mice

Disease-modifying treatments for Alzheimer’s disease have proved elusive. However, a study in mice published this week suggests that a treatment strategy relying on gene therapy may be worth pursuing.

Researchers at the Gladstone Institute of Neurological Disease in San Francisco found that mice and humans with Alzheimer’s disease have unusually low levels of an enzyme called EphB2 in the parts of the brain that control memory. EphB2 plays an important role in fostering communication between brain cells. The scientists used gene therapy to experimentally change EphB2 levels in the mice. When levels were reduced in healthy mice, they had memory problems similar to those seen in mice with Alzheimer’s. Increasing levels of the enzyme in the mice with a predisposition to Alzheimer’s disease, however, prevented memory and behavior problems.

The study showed that amyloid proteins, the substance that causes the sticky plaque that is a hallmark characteristic of Alzheimer’s disease, bind to EphB2 and cause it to degrade and levels to drop.

“Based on our results, we think that blocking amyloid proteins from binding to EphB2 and enhancing EphB2 levels or functions with drugs might be of benefit in Alzheimer’s disease,” the senior author of the study, Dr. Lennart Mucke, said in a news release. The study was published in the Nov. 28 issue of Nature.

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