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- Supporting Families Affected by Prion Disease
Today’s main therapeutic strategy for prion disease is to decrease PrP levels in the brain. From animal studies, we know that if PrP is completely absent, the disease never develops. Unfortunately, complete absence of PrP is also associated with mild defects in the protective sheath around nerves, called myelin. These defects could lead to side effects. On the other hand, if PrP levels are reduced by half, these myelin defects are not observed, but the disease is only slowed, not cured.
This raises two critical questions: how much do we need to lower PrP to halt or reverse prion disease, and is such a reduction associated with myelin defects? To address these questions, we engineered two mouse models expressing 5% and 15% of normal PrP levels. The goal of our study is to characterize these models so they can be used in future research. In the long term, these models will help determine how much PrP should be reduced in humans for future clinical trials.
Dr. Vanessa Laversenne was originally trained as an engineer in biotechnology at the École Polytechnique Fédérale de Lausanne (EPFL) in Switzerland. She then specialized in drug development for neurodegenerative diseases during her master’s and Ph.D. studies at EPFL. After completing her postdoctoral training at the Harvard School of Public Health, where she worked on Alzheimer’s disease and aging, she joined the Vallabh/Minikel Laboratory at the Broad Institute of Harvard and MIT to help develop effective therapies for prion disease.