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A Small-Molecule Inhibitor of Prion Replication and Mutant Prion Protein Toxicity.

Tania MassignanValeria SangiovanniSilvia BiggiClaudia StincardiniSaioa R ElezgaraiGiulia MaiettaIvan A AndreevNina K RatmanovaDmitry S BelovEvgeny R LukyanenkoGrigory M BelovMaria Letizia BarrecaAndrea AltieriAlexander V KurkinEmiliano Biasini
Published in: ChemMedChem (2017)
Into the fold: Prion diseases are neurodegenerative disorders characterized by the accumulation in the brain of a self-replicating, misfolded isoform (PrPSc ) of the cellular prion protein (PrPC ). No therapies are available for these pathologies. We capitalized on previously described cell-based assays to screen a library of small molecules, and identified 55, a compound capable of counteracting both prion replication and toxicity. Compound 55 may represent the starting point for the development of a completely new class of therapeutics for prion diseases.
Keyphrases
  • small molecule
  • protein protein
  • high throughput
  • amino acid
  • bone marrow