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, T., Sangiovanni, V., Biggi, S., Stincardini, C., Elezgarai, S.R., Maietta, G., Andreev, I.A., Ratmanova, N.K., Belov, D.S., Lukyanenko, E.R., Belov, G.M., Barreca, M.L., Altieri, A., Kurkin, A.V., Biasini, E.. - 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
