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2007 Grant - Gordon
Cell-Based Therapies Using Transgenic Mice
Marcia Gordon, Ph.D.
University of South Florida
2007 Investigator-Initiated Research Grant
One strategy that has been proposed for treating Alzheimer's disease is to utilize enzymes to remove beta-amyloid from the brain. Beta-amyloid is a protein fragment that is a primary component of amyloid plaques, one of the characteristic features of Alzheimer's disease. One enzyme that degrades beta-amyloid is neprilysin, but methods to introduce this enzyme into the brain have been hampered by the risks of surgery or by immune reactions and cancer risks associated with molecular methods.
Marcia Gordon, Ph.D., and colleagues have proposed to introduce neprilysin into the brain by using a patient's own cells to carry the gene for the protein. The researchers plan to evaluate the use of monocytes, which are a type of cell found in the blood, for carrying the neprilysin gene into the brain and expressing it there. Monocytes have a number of advantages that make them well-suited for this purpose.
To test the feasibility of this method, Dr. Gordon and colleagues plan to study mice that express genes that make them susceptible to an Alzheimer-like condition and that cause deposition of beta-amyloid in the brain. Using these mice, they will retrieve the animal's own monocytes, introduce the gene for neprilysin into the cells, and then reintroduce the cells into the animal's blood stream. They will then evaluate the efficacy and safety of this procedure for removing beta-amyloid from the brain. These studies could lead to the development of methods to remove beta-amyloid from the brain of people affected by Alzheimer's disease.