Gene therapy might one day be an effective treatment for Alzheimer’s disease, new experiments in mice suggest.

The study found that gene delivery of a human protein called apolipoprotein E2 (apoE2) helped cut down amyloid-beta deposits in the brains of mice. The deposits are the hallmark of Alzheimer’s disease.

“Gene therapy offers the opportunity to influence the progression of this horrible disease,” Dr. Inder M. Verma, who led the study, told AMN Health.

Apolipoprotein E latches on to fats and clears them from the bloodstream. It is also found in the brain, where it is thought to perform a similar function by getting rid of unwanted substances.

People produce different forms of apolipoprotein E, and studies have shown that those with apoE2 tend to be protected against Alzheimer’s disease, whereas those with apoE4 are more likely to develop the disease.

Verma, who is based at The Salk Institute in La Jolla, California, and his colleagues investigated whether gene delivery of apoE2 could directly affect the deposits of amyloid-beta in mice that had been genetically altered to develop Alzheimer’s disease.

ApoE2 gene therapy resulted in a 30 percent to 50 percent reduction in amyloid-beta in the mice’s hippocampus, a region of the brain that helps control memory.

Verma and his colleagues reported their findings in the January 25 issue of the online journal Proceedings of the National Academy of Sciences Early Edition.

The team also plans to conduct additional studies in other animals. They hope to discover exactly how the treatment works to affect the amyloid-beta deposits.

SOURCE: Proceedings of the National Academy of Sciences Early Edition, January 25, 2005.