{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T12:05:09Z","timestamp":1790510709161,"version":"4.1.0"},"reference-count":39,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2020,4,1]],"date-time":"2020-04-01T00:00:00Z","timestamp":1585699200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2020,2,10]],"date-time":"2020-02-10T00:00:00Z","timestamp":1581292800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"Cure Sanfilippo Foundation"},{"name":"Telethon"},{"name":"NIH\/NIA","award":["R01AG050721"],"award-info":[{"award-number":["R01AG050721"]}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2020,4]]},"DOI":"10.1016\/j.ymthe.2020.02.005","type":"journal-article","created":{"date-parts":[[2020,2,12]],"date-time":"2020-02-12T11:11:24Z","timestamp":1581505884000},"page":"1167-1176","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":41,"title":["The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease"],"prefix":"10.1016","volume":"28","author":[{"given":"Antonio","family":"Monaco","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veronica","family":"Maffia","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicolina Cristina","family":"Sorrentino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Irene","family":"Sambri","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yulia","family":"Ezhova","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Teresa","family":"Giuliano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincenzo","family":"Cacace","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Edoardo","family":"Nusco","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"De Risi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elvira","family":"De Leonibus","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Schrader","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Frank-Gerrit","family":"Kl\u00e4rner","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gal","family":"Bitan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessandro","family":"Fraldi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2020.02.005_bib1","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1016\/j.tins.2011.05.006","article-title":"Clarifying lysosomal storage diseases","volume":"34","author":"Schultz","year":"2011","journal-title":"Trends Neurosci."},{"key":"10.1016\/j.ymthe.2020.02.005_bib2","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1083\/jcb.201208152","article-title":"The cell biology of disease: lysosomal storage disorders: the cellular impact of lysosomal dysfunction","volume":"199","author":"Platt","year":"2012","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib3","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1146\/annurev-neuro-070815-014031","article-title":"Brain Disorders Due to Lysosomal Dysfunction","volume":"39","author":"Fraldi","year":"2016","journal-title":"Annu. Rev. Neurosci."},{"key":"10.1016\/j.ymthe.2020.02.005_bib4","doi-asserted-by":"crossref","first-page":"719","DOI":"10.4161\/auto.19469","article-title":"Autophagy in lysosomal storage disorders","volume":"8","author":"Lieberman","year":"2012","journal-title":"Autophagy"},{"key":"10.1016\/j.ymthe.2020.02.005_bib5","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1093\/hmg\/ddm289","article-title":"A block of autophagy in lysosomal storage disorders","volume":"17","author":"Settembre","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2020.02.005_bib6","doi-asserted-by":"crossref","first-page":"e147","DOI":"10.1038\/emm.2014.117","article-title":"Degradation of misfolded proteins in neurodegenerative diseases: therapeutic targets and strategies","volume":"47","author":"Ciechanover","year":"2015","journal-title":"Exp. Mol. Med."},{"key":"10.1016\/j.ymthe.2020.02.005_bib7","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1016\/j.cell.2011.06.001","article-title":"Gaucher disease glucocerebrosidase and \u03b1-synuclein form a bidirectional pathogenic loop in synucleinopathies","volume":"146","author":"Mazzulli","year":"2011","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2020.02.005_bib8","doi-asserted-by":"crossref","first-page":"8332","DOI":"10.1073\/pnas.0903223106","article-title":"Sanfilippo syndrome type B, a lysosomal storage disease, is also a tauopathy","volume":"106","author":"Ohmi","year":"2009","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2020.02.005_bib9","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1016\/j.expneurol.2017.06.010","article-title":"Axonal dystrophy in the brain of mice with Sanfilippo syndrome","volume":"295","author":"Beard","year":"2017","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib10","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1002\/path.4328","article-title":"Neuronal inclusions of \u03b1-synuclein contribute to the pathogenesis of Krabbe disease","volume":"232","author":"Smith","year":"2014","journal-title":"J.\u00a0Pathol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib11","doi-asserted-by":"crossref","first-page":"2734","DOI":"10.1038\/ncomms3734","article-title":"Lysosomal NEU1 deficiency affects amyloid precursor protein levels and amyloid-\u03b2 secretion via deregulated lysosomal exocytosis","volume":"4","author":"Annunziata","year":"2013","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2020.02.005_bib12","doi-asserted-by":"crossref","first-page":"112","DOI":"10.15252\/emmm.201606965","article-title":"Lysosomal dysfunction disrupts presynaptic maintenance and restoration of presynaptic function prevents neurodegeneration in lysosomal storage diseases","volume":"9","author":"Sambri","year":"2017","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2020.02.005_bib13","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1002\/ana.22092","article-title":"Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations","volume":"68","author":"Valstar","year":"2010","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib14","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.ymthe.2020.02.005_bib15","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1093\/glycob\/9.12.1389","article-title":"A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome)","volume":"9","author":"Bhaumik","year":"1999","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymthe.2020.02.005_bib16","doi-asserted-by":"crossref","first-page":"E2292","DOI":"10.3390\/ijms19082292","article-title":"Protein Co-Aggregation Related to Amyloids: Methods of Investigation, Diversity, and Classification","volume":"19","author":"Bondarev","year":"2018","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2020.02.005_bib17","doi-asserted-by":"crossref","first-page":"16958","DOI":"10.1021\/ja206279b","article-title":"Lysine-specific molecular tweezers are broad-spectrum inhibitors of assembly and toxicity of amyloid proteins","volume":"133","author":"Sinha","year":"2011","journal-title":"J.\u00a0Am. Chem. Soc."},{"key":"10.1016\/j.ymthe.2020.02.005_bib18","doi-asserted-by":"crossref","first-page":"2469","DOI":"10.2174\/13816128113199990496","article-title":"Disrupting self-assembly and toxicity of amyloidogenic protein oligomers by \u201cmolecular tweezers\u201d - from the test tube to animal models","volume":"20","author":"Attar","year":"2014","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.ymthe.2020.02.005_bib19","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1186\/2050-6511-15-23","article-title":"Safety and pharmacological characterization of the molecular tweezer CLR01 - a broad-spectrum inhibitor of amyloid proteins\u2019 toxicity","volume":"15","author":"Attar","year":"2014","journal-title":"BMC Pharmacol. Toxicol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib20","doi-asserted-by":"crossref","first-page":"464","DOI":"10.1007\/s13311-012-0105-1","article-title":"A novel \u201cmolecular tweezer\u201d inhibitor of \u03b1-synuclein neurotoxicity in\u00a0vitro and in\u00a0vivo","volume":"9","author":"Prabhudesai","year":"2012","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.ymthe.2020.02.005_bib21","doi-asserted-by":"crossref","first-page":"1107","DOI":"10.1007\/s13311-017-0544-9","article-title":"A Molecular Tweezer Ameliorates Motor Deficits in Mice Overexpressing \u03b1-Synuclein","volume":"14","author":"Richter","year":"2017","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.ymthe.2020.02.005_bib22","doi-asserted-by":"crossref","first-page":"3735","DOI":"10.1093\/brain\/aws289","article-title":"Protection of primary neurons and mouse brain from Alzheimer\u2019s pathology by molecular tweezers","volume":"135","author":"Attar","year":"2012","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2020.02.005_bib23","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1007\/s13311-013-0256-8","article-title":"Molecular tweezers targeting transthyretin amyloidosis","volume":"11","author":"Ferreira","year":"2014","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.ymthe.2020.02.005_bib24","doi-asserted-by":"crossref","first-page":"6926","DOI":"10.1523\/JNEUROSCI.0800-08.2008","article-title":"Autophagy induction and autophagosome clearance in neurons: relationship to autophagic pathology in Alzheimer\u2019s disease","volume":"28","author":"Boland","year":"2008","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2020.02.005_bib25","doi-asserted-by":"crossref","first-page":"407","DOI":"10.1083\/jcb.201106120","article-title":"Autophagosomes initiate distally and mature during transport toward the cell soma in primary neurons","volume":"196","author":"Maday","year":"2012","journal-title":"J.\u00a0Cell Biol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib26","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1016\/j.nbd.2018.05.015","article-title":"Lysosome trafficking and signaling in health and neurodegenerative diseases","volume":"122","author":"Lie","year":"2019","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2020.02.005_bib27","doi-asserted-by":"crossref","first-page":"113","DOI":"10.4161\/auto.5227","article-title":"Lysosomal storage diseases as disorders of autophagy","volume":"4","author":"Settembre","year":"2008","journal-title":"Autophagy"},{"key":"10.1016\/j.ymthe.2020.02.005_bib28","doi-asserted-by":"crossref","first-page":"505","DOI":"10.1016\/j.molcel.2009.01.020","article-title":"A role for NBR1 in autophagosomal degradation of ubiquitinated substrates","volume":"33","author":"Kirkin","year":"2009","journal-title":"Mol. Cell"},{"key":"10.1016\/j.ymthe.2020.02.005_bib29","doi-asserted-by":"crossref","first-page":"e35787","DOI":"10.1371\/journal.pone.0035787","article-title":"Neuropathology in mouse models of mucopolysaccharidosis type I, IIIA and IIIB","volume":"7","author":"Wilkinson","year":"2012","journal-title":"PLoS ONE"},{"key":"10.1016\/j.ymthe.2020.02.005_bib30","doi-asserted-by":"crossref","first-page":"2693","DOI":"10.1093\/hmg\/ddm223","article-title":"Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes","volume":"16","author":"Fraldi","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2020.02.005_bib31","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1016\/j.omtm.2019.10.009","article-title":"Enhancing the Therapeutic Potential of Sulfamidase for the Treatment of Mucopolysaccharidosis IIIA","volume":"15","author":"Sorrentino","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2020.02.005_bib32","doi-asserted-by":"crossref","first-page":"jcs221739","DOI":"10.1242\/jcs.221739","article-title":"Lysosomal storage disorders - challenges, concepts and avenues for therapy: beyond rare diseases","volume":"132","author":"Marques","year":"2019","journal-title":"J.\u00a0Cell Sci."},{"issue":"R1","key":"10.1016\/j.ymthe.2020.02.005_bib33","doi-asserted-by":"crossref","first-page":"R54","DOI":"10.1093\/hmg\/ddr112","article-title":"Combination therapies for lysosomal storage disease: is the whole greater than the sum of its parts?","volume":"20","author":"Hawkins-Salsbury","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2020.02.005_bib34","doi-asserted-by":"crossref","first-page":"283","DOI":"10.1038\/nrm3565","article-title":"Signals from the lysosome: a control centre for cellular clearance and energy metabolism","volume":"14","author":"Settembre","year":"2013","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.ymthe.2020.02.005_bib35","doi-asserted-by":"crossref","first-page":"2510","DOI":"10.3390\/molecules20022510","article-title":"The effect of glycosaminoglycans (GAGs) on amyloid aggregation and toxicity","volume":"20","author":"Iannuzzi","year":"2015","journal-title":"Molecules"},{"key":"10.1016\/j.ymthe.2020.02.005_bib36","doi-asserted-by":"crossref","first-page":"332ra44","DOI":"10.1126\/scitranslmed.aad3650","article-title":"Neuronal heparan sulfates promote amyloid pathology by modulating brain amyloid-\u03b2 clearance and aggregation in Alzheimer\u2019s disease","volume":"8","author":"Liu","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2020.02.005_bib37","doi-asserted-by":"crossref","first-page":"657","DOI":"10.3389\/fchem.2019.00657","article-title":"Molecular Lysine Tweezers Counteract Aberrant Protein Aggregation","volume":"7","author":"Hadrovic","year":"2019","journal-title":"Front Chem."},{"key":"10.1016\/j.ymthe.2020.02.005_bib38","doi-asserted-by":"crossref","first-page":"1642","DOI":"10.1093\/brain\/awv040","article-title":"Direct visualization of alpha-synuclein oligomers reveals previously undetected pathology in Parkinson\u2019s disease brain","volume":"138","author":"Roberts","year":"2015","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2020.02.005_bib39","doi-asserted-by":"crossref","first-page":"185","DOI":"10.4161\/auto.27367","article-title":"The vacuole versus the lysosome: when size matters","volume":"10","author":"Klionsky","year":"2014","journal-title":"Autophagy"}],"updated-by":[{"DOI":"10.1016\/j.ymthe.2022.10.001","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2022,11,2]],"date-time":"2022-11-02T00:00:00Z","timestamp":1667347200000}}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620300915?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001620300915?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2022,11,6]],"date-time":"2022-11-06T03:10:42Z","timestamp":1667704242000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001620300915"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2020,4]]},"references-count":39,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2020,4]]}},"alternative-id":["S1525001620300915"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.02.005","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2020,4]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The Amyloid Inhibitor CLR01 Relieves Autophagy and Ameliorates Neuropathology in a Severe Lysosomal Storage Disease","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2020.02.005","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2020 The Author(s).","name":"copyright","label":"Copyright"}]}}