Into the fold: Prion diseases are neurodegenerative disorders characterized by the accumulation in the brain of a self-replicating, misfolded isoform (PrP(Sc) ) of the cellular prion protein (PrP(C) ). 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.
A Small-Molecule Inhibitor of Prion Replication and Mutant Prion Protein Toxicity / Massignan, Tania; Sangiovanni, Valeria; Biggi, Silvia; Stincardini, Claudia; Elezgarai, Saioa R.; Maietta, Giulia; Andreev, Ivan A.; Ratmanova, Nina K.; Belov, Dmitry S.; Lukyanenko, Evgeny R.; Belov, Grigory M.; Barreca, Maria Letizia; Altieri, Andrea; Kurkin, Alexander V.; Biasini, Emiliano. - In: CHEMMEDCHEM. - ISSN 1860-7179. - 12:16(2017), pp. 1286-1292. [10.1002/cmdc.201700302]
A Small-Molecule Inhibitor of Prion Replication and Mutant Prion Protein Toxicity
Massignan, Tania;Biggi, Silvia;Stincardini, Claudia;Maietta, Giulia;Biasini, Emiliano
2017-01-01
Abstract
Into the fold: Prion diseases are neurodegenerative disorders characterized by the accumulation in the brain of a self-replicating, misfolded isoform (PrP(Sc) ) of the cellular prion protein (PrP(C) ). 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.I documenti in IRIS sono protetti da copyright e tutti i diritti sono riservati, salvo diversa indicazione
