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2005 Grant - Loring
Stem Cells as Delivery Vehicles to Target Amyloid Plaques and Tangles
Jeanne F. Loring, Ph.D.
The Burnham Institute
La Jolla, California
2005 Investigator-Initiated Research Grant
Amyloid plaque is a key pathological feature of Alzheimer's disease. The main component of a plaque is the beta-amyloid protein fragment, which may be the main toxic factor damaging cell-to-cell communication and causing the loss of cells. A large effort in Alzheimer's research is to develop treatments that clear beta-amyloid and amyloid plaques from the brain.
Jeanne F. Loring, Ph.D., and associates are investigating whether a neural stem cell can be used to deliver neprilysin, an amyloid-degrading brain protein, to plaque-laden sites in the brain. Neural stem cells are cells in the brain that have the potential to develop into any type of brain cell. Studies have shown that they tend to migrate to regions of the brain affected by inflammation. Because there is inflammation at the site of amyloid plaques, neural stem cells engineered to carry neprilysin should deliver the anti-amyloid protein to where it is needed.
The investigators will test this therapeutic strategy in genetically altered mice that develop an Alzheimer-like disorder and symptoms. They will assess variant forms of this strategy in the mice and determine the impact on amyloid concentrations and disease progression. The outcome of this work may demonstrate whether this treatment may be effective and safe for study in clinical trials.