| http://purl.uniprot.org/citations/32737286 | http://www.w3.org/1999/02/22-rdf-syntax-ns#type | http://purl.uniprot.org/core/Journal_Citation |
| http://purl.uniprot.org/citations/32737286 | http://www.w3.org/2000/01/rdf-schema#comment | "Amyotrophic Lateral Sclerosis (ALS) is a fatal disease characterized by the degeneration of upper and lower motor neurons (MNs). We find a significant reduction of the retromer complex subunit VPS35 in iPSCs-derived MNs from ALS patients, in MNs from ALS post mortem explants and in MNs from SOD1G93A mice. Being the retromer involved in trafficking of hydrolases, a pathological hallmark in ALS, we design, synthesize and characterize an array of retromer stabilizers based on bis-guanylhydrazones connected by a 1,3-phenyl ring linker. We select compound 2a as a potent and bioavailable interactor of VPS35-VPS29. Indeed, while increasing retromer stability in ALS mice, compound 2a attenuates locomotion impairment and increases MNs survival. Moreover, compound 2a increases VPS35 in iPSCs-derived MNs and shows brain bioavailability. Our results clearly suggest the retromer as a valuable druggable target in ALS."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.org/dc/terms/identifier | "doi:10.1038/s41467-020-17524-7"xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Mastrangelo E."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Milani M."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Quattrini A."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Martino G."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Comi G."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Ottoboni L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Riva N."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Marinelli L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Muzio L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Brancaccio D."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Manzoni L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Seneci P."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Eleuteri S."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Sorrentino L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Scalone E."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Sirtori R."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Feo L."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Fossaghi A."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/author | "Gornati D."xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/date | "2020"xsd:gYear |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/name | "Nat Commun"xsd:string |
| http://purl.uniprot.org/citations/32737286 | http://purl.uniprot.org/core/pages | "3848"xsd:string |