McLaughlin Institute

MRI Collaborates with researchers at MIT/Harvard

An exciting new research collaboration with Eric Minikel and Sonia Vallabh at the Harvard/MIT Broad Institute involves testing a class of drugs called anti-sense oligonucleotides (ASOs) for efficacy against prion disease. Mouse models of prion disease are well characterized and recapitulate the effects of human prion diseases. We, along with Eric and Sonia, are testing ASOs developed by Ionis Pharmaceuticals in mouse models of prion disease, and have found that some ASOs significantly delay symptoms and extend lifespan. Pre-clinical work at MRI is continuing using mouse models to support an application to the Federal Drug Administration for testing ASOs against prion disease in humans. While prion diseases are rare in humans, more common neurodegenerative diseases work by the same mechanism. A therapy that works against human prion disease could be tailored to work against Alzheimer’s, Parkinson’s and Huntington’s disease, and we hope to extend the work on prion disease to animal models for the more common diseases.

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