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2012 Grants - Gong
hFbx2/APP, a Novel Transgenic Mouse Model in the Study of BACE1 Degradation
Bing Gong, M.D., Ph. D.
Mount Sinai School of Medicine
New York, New York
2012 Investigator-Initiated Research Grant
A common approach scientists use to study diseases such as Alzheimer's disease is to use genetic engineering techniques to create animal models that mimic the disease characteristics. Such animals are known as transgenic animals.
BACE1 is an enzyme in the brain thought to play an important role in Alzheimer's disease. It is a key enzyme in the production of beta-amyloid, a protein fragment that aggregates into amyloid plaque in the brain, one of the characteristic features of Alzheimer's disease. BACE1 is part of a biochemical pathway that cuts beta-amyloid from its parent protein, amyloid precursor protein.
Bing Gong, M.D., Ph.D., and colleagues have been studying the role of BACE1 in the production of beta-amyloid. They have developed a transgenic mouse strain that produces high levels of another protein, hFBX2, which destroys BACE1. Dr. Gong and colleagues have proposed a series of studies to characterize these transgenic mice, to determine if destruction of BACE1 reduces amyloid plaque formation in the brain. These studies will answer key questions about the role of BACE1 in the disease process, and may identify targets for drugs that block the actions of BACE1.