Identification of anti-prion drugs and targets using toxicity-based assays

Prion diseases are untreatable and invariably fatal, making the discovery of effective therapeutic interventions a priority. Most candidate molecules have been discovered based on their ability to reduce the levels of PrPSc, the infectious form of the prion protein, in cultured neuroblastoma cells. We have employed an alternative assay, based on an abnormal cellular phenotype associated with a mutant prion protein, to discover a novel class of...