The Compelling Demand for an Effective PrP^C-Directed Therapy against Prion Diseases

Decades of research efforts have conclusively provided overwhelming evidence that the cellular prion protein (PrP^C) plays a central role in prion diseases, a set of fatal and incurable neurodegenerative disorders for which no therapy is yet available. In this Viewpoint, we provide an overview of the drug discovery strategies in the field, highlighting the current therapeutic hypotheses targeting, whether directly or indirectly, PrP^C as well as the antiprion agents closest to clinical application.