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2013 Grants - Okun
Developing a DNA Vaccine for Alzheimer's Disease in People with Down Syndrome
Eitan Okun, Ph.D.
2013 Down Syndrome/Alzheimer's Disease New Investigator Program
People who have Down syndrome have a high risk of developing Alzheimer's disease, and the disease begins to develop when they are still in their 30s and 40s. Clinical trials have attempted to find a vaccine to slow or prevent Alzheimer's disease, but to date there is no treatment to stop or slow disease progression. Most vaccines have been designed to use the immune system to remove beta-amyloid from the brain. Beta-amyloid is a protein fragment that is toxic to nerve cells and forms amyloid plaques, one of the characteristic features of Alzheimer's disease.
Eitan Okun, Ph.D., and colleagues are working to develop a new type of vaccine for Alzheimer's disease, including people who have Down syndrome and who are at risk of Alzheimer's at a very young age. Their strategy is to use molecular techniques to modify a virus so it produces a short version of beta-amyloid. The strategy is to stimulate the immune system to remove several types of beta-amyloid in the brain, but may reduce the chances of side effects.
Dr. Okun and colleagues have proposed a series of experiments to further develop their vaccine and test its effectiveness. Once an optimal vaccine is developed, the researchers will study its ability to remove different forms of beta-amyloid from mice that have a condition resembling Down syndrome and Alzheimer's disease. The researchers will also test the mice to determine if the vaccine prevents or reverses impairments in brain function or behavior associated with Alzheimer's disease. These studies may advance the development of a new type of vaccine to slow or prevent the progression of Alzheimer's disease, including Alzheimer's in people who have Down syndrome.