Therapeutic Approaches for Prion Diseases

Prion diseases are neurodegenerative disorders that affect both humans and animals and are characterized by the accumulation of a misfolded isoform of the host encoded prion protein (PrPC) and pathogenic prion protein (PrPSc) in the central nervous system and, in many instances, in the lymphoreticular system. These diseases have received great interest due to the fact that they can be transmissible among humans and, in certain conditions, from animals to humans. Polymorphism or mutations of the prion protein gene can influence the conversion of cellular prion protein into a protease-K-resistant form. A large number of studies have been performed in experimental models to develop effective therapeutics for the therapy of prion diseases that cause death after several months following a rapid disease progression. This review summarizes past and current assays for the development of prion disease therapeutics.