{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,22]],"date-time":"2026-09-22T09:52:20Z","timestamp":1790070740617,"version":"4.0.1"},"reference-count":75,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2018,9,1]],"date-time":"2018-09-01T00:00:00Z","timestamp":1535760000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2018,9,1]],"date-time":"2018-09-01T00:00:00Z","timestamp":1535760000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2018,5,23]],"date-time":"2018-05-23T00:00:00Z","timestamp":1527033600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100002426","name":"Fondazione Telethon","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100002426","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003500","name":"Universit\u00e0 degli Studi di Padova","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003500","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007195","name":"Universit\u00e0 degli Studi di Napoli Federico II","doi-asserted-by":"publisher","award":["GTB12001"],"award-info":[{"award-number":["GTB12001"]}],"id":[{"id":"10.13039\/100007195","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2018,9]]},"DOI":"10.1016\/j.omtm.2018.05.002","type":"journal-article","created":{"date-parts":[[2018,6,18]],"date-time":"2018-06-18T12:53:53Z","timestamp":1529326433000},"page":"8-16","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":33,"special_numbering":"C","title":["Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses"],"prefix":"10.1016","volume":"10","author":[{"given":"Valeria","family":"De Pasquale","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patrizia","family":"Sarogni","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Valeria","family":"Pistorio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuliana","family":"Cerulo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simona","family":"Paladino","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Luigi Michele","family":"Pavone","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2018.05.002_bib1","series-title":"The Metabolic and Molecular Bases of Inherited Disease","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.omtm.2018.05.002_bib2","doi-asserted-by":"crossref","first-page":"e1","DOI":"10.1017\/S1462399408000550","article-title":"The mucopolysaccharidoses: a success of molecular medicine","volume":"10","author":"Clarke","year":"2008","journal-title":"Expert Rev. Mol. Med."},{"key":"10.1016\/j.omtm.2018.05.002_bib3","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1111\/jcpt.12136","article-title":"Current and potential therapeutic strategies for mucopolysaccharidoses","volume":"39","author":"Noh","year":"2014","journal-title":"J.\u00a0Clin. Pharm. Ther."},{"key":"10.1016\/j.omtm.2018.05.002_bib4","doi-asserted-by":"crossref","first-page":"587","DOI":"10.1007\/s10545-014-9718-3","article-title":"Treatment of lysosomal storage disorders: successes and challenges","volume":"37","author":"Hollak","year":"2014","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.omtm.2018.05.002_bib5","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1146\/annurev-med-122313-085916","article-title":"Lysosomal storage diseases: from pathophysiology to therapy","volume":"66","author":"Parenti","year":"2015","journal-title":"Annu. Rev. Med."},{"key":"10.1016\/j.omtm.2018.05.002_bib6","doi-asserted-by":"crossref","first-page":"1331","DOI":"10.1080\/13543784.2017.1397130","article-title":"Phase I and II clinical trials for the mucopolysaccharidoses","volume":"26","author":"Poswar","year":"2017","journal-title":"Expert Opin. Investig. Drugs"},{"key":"10.1016\/j.omtm.2018.05.002_bib7","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.ymgme.2013.11.015","article-title":"Early initiation of enzyme replacement therapy for the mucopolysaccharidoses","volume":"111","author":"Muenzer","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2018.05.002_bib8","first-page":"1895","article-title":"Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency","volume":"93","author":"Lutzko","year":"1999","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2018.05.002_bib9","first-page":"63","article-title":"Early umbilical cord blood-derived stem cell transplantation does not prevent neurological deterioration in mucopolysaccharidosis type III","volume":"18","author":"Welling","year":"2015","journal-title":"JIMD Rep."},{"key":"10.1016\/j.omtm.2018.05.002_bib10","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1182\/blood.V88.1.377b.377b","article-title":"Humoral immune response limits gene therapy in canine MPS I","volume":"88","author":"Shull","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2018.05.002_bib11","doi-asserted-by":"crossref","first-page":"143","DOI":"10.1016\/j.omtm.2017.07.004","article-title":"Non-clinical safety and efficacy of an AAV2\/8 vector administered intravenously for treatment of Mucopolysaccharidosis type VI","volume":"6","author":"Ferla","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2018.05.002_bib12","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1016\/j.ymgme.2017.12.434","article-title":"Gene therapy for Mucopolysaccharidoses","volume":"123","author":"Sawamoto","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2018.05.002_bib13","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1016\/S1474-4422(17)30169-2","article-title":"Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1\/2 clinical trial","volume":"16","author":"Tardieu","year":"2017","journal-title":"Lancet Neurol."},{"key":"10.1016\/j.omtm.2018.05.002_bib14","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1089\/hum.2013.238","article-title":"Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I\/II trial","volume":"25","author":"Tardieu","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2018.05.002_bib15","doi-asserted-by":"crossref","first-page":"481","DOI":"10.1152\/physrev.1991.71.2.481","article-title":"Glycosaminoglycans: molecular properties, protein interactions, and role in physiological processes","volume":"71","author":"Jackson","year":"1991","journal-title":"Physiol. Rev."},{"key":"10.1016\/j.omtm.2018.05.002_bib16","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.matbio.2015.02.003","article-title":"Proteoglycan form and function: A comprehensive nomenclature of proteoglycans","volume":"42","author":"Iozzo","year":"2015","journal-title":"Matrix Biol."},{"key":"10.1016\/j.omtm.2018.05.002_bib17","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/j.gde.2013.02.004","article-title":"Extracellular modulation of Fibroblast Growth Factor signaling through heparan sulfate proteoglycans in mammalian development","volume":"23","author":"Matsuo","year":"2013","journal-title":"Curr. Opin. Genet. Dev."},{"key":"10.1016\/j.omtm.2018.05.002_bib18","doi-asserted-by":"crossref","first-page":"272","DOI":"10.3109\/03008207.2015.1045066","article-title":"Interactions of signaling proteins, growth factors and other proteins with heparan sulfate: mechanisms and mysteries","volume":"56","author":"Billings","year":"2015","journal-title":"Connect. Tissue Res."},{"key":"10.1016\/j.omtm.2018.05.002_bib19","doi-asserted-by":"crossref","first-page":"139","DOI":"10.1530\/JOE-10-0377","article-title":"Extracellular matrix and cell signalling: the dynamic cooperation of integrin, proteoglycan and growth factor receptor","volume":"209","author":"Kim","year":"2011","journal-title":"J.\u00a0Endocrinol."},{"key":"10.1016\/j.omtm.2018.05.002_bib20","doi-asserted-by":"crossref","first-page":"266","DOI":"10.1002\/jcp.10030","article-title":"Proteoglycans of the extracellular matrix and growth control","volume":"189","author":"Kresse","year":"2001","journal-title":"J.\u00a0Cell. Physiol."},{"key":"10.1016\/j.omtm.2018.05.002_bib21","doi-asserted-by":"crossref","first-page":"6009","DOI":"10.1242\/dev.01522","article-title":"Functions of heparan sulfate proteoglycans in cell signaling during development","volume":"131","author":"Lin","year":"2004","journal-title":"Development"},{"key":"10.1016\/j.omtm.2018.05.002_bib22","doi-asserted-by":"crossref","first-page":"3456","DOI":"10.1242\/dev.098178","article-title":"Heparan sulfate proteoglycans: a sugar code for vertebrate development?","volume":"142","author":"Poulain","year":"2015","journal-title":"Development"},{"key":"10.1016\/j.omtm.2018.05.002_bib23","doi-asserted-by":"crossref","first-page":"512","DOI":"10.1016\/j.ydbio.2006.08.076","article-title":"On the role of glypicans in the process of morphogen gradient formation","volume":"300","author":"Hufnagel","year":"2006","journal-title":"Dev. Biol."},{"key":"10.1016\/j.omtm.2018.05.002_bib24","doi-asserted-by":"crossref","first-page":"2408","DOI":"10.1002\/1873-3468.12280","article-title":"Minireview: Syndecans and their crucial roles during tissue regeneration","volume":"590","author":"Chung","year":"2016","journal-title":"FEBS Lett."},{"key":"10.1016\/j.omtm.2018.05.002_bib25","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1016\/j.matbio.2016.09.004","article-title":"The function of heparan sulfate during branching morphogenesis","volume":"57-58","author":"Patel","year":"2017","journal-title":"Matrix Biol."},{"key":"10.1016\/j.omtm.2018.05.002_bib26","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1111\/j.1747-0285.2008.00741.x","article-title":"The structure of glycosaminoglycans and their interactions with proteins","volume":"72","author":"Gandhi","year":"2008","journal-title":"Chem. Biol. Drug Des."},{"key":"10.1016\/j.omtm.2018.05.002_bib27","doi-asserted-by":"crossref","first-page":"13110","DOI":"10.1074\/jbc.271.22.13110","article-title":"Hepatocyte growth factor (HGF)\/NK1 is a naturally occurring HGF\/scatter factor variant with partial agonist\/antagonist activity","volume":"271","author":"Cioce","year":"1996","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib28","doi-asserted-by":"crossref","first-page":"6311","DOI":"10.1074\/jbc.M807671200","article-title":"The binding properties of minimal oligosaccharides reveal a common heparan sulfate\/dermatan sulfate-binding site in hepatocyte growth factor\/scatter factor that can accommodate a wide variety of sulfation patterns","volume":"284","author":"Deakin","year":"2009","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib29","doi-asserted-by":"crossref","first-page":"269","DOI":"10.2147\/TACG.S57672","article-title":"Sanfilippo syndrome: causes, consequences, and treatments","volume":"8","author":"Fedele","year":"2015","journal-title":"Appl. Clin. Genet."},{"key":"10.1016\/j.omtm.2018.05.002_bib30","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1186\/s13023-017-0675-4","article-title":"Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type III","volume":"12","author":"Ghosh","year":"2017","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2018.05.002_bib31","doi-asserted-by":"crossref","first-page":"9378","DOI":"10.1038\/srep09378","article-title":"Modulation of expression of genes involved in glycosaminoglycan metabolism and lysosome biogenesis by flavonoids","volume":"5","author":"Moskot","year":"2015","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2018.05.002_bib32","doi-asserted-by":"crossref","first-page":"282","DOI":"10.1006\/abio.1993.1197","article-title":"Simultaneous preparation and quantitation of proteoglycans by precipitation with alcian blue","volume":"210","author":"Bj\u00f6rnsson","year":"1993","journal-title":"Anal. Biochem."},{"key":"10.1016\/j.omtm.2018.05.002_bib33","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1590\/S0001-37652009000300007","article-title":"Heparan sulfate proteoglycans: structure, protein interactions and cell signaling","volume":"81","author":"Dreyfuss","year":"2009","journal-title":"An. Acad. Bras. Cienc."},{"key":"10.1016\/j.omtm.2018.05.002_bib34","doi-asserted-by":"crossref","first-page":"8716","DOI":"10.1016\/S0021-9258(19)38948-3","article-title":"The core protein of the matrix-associated heparan sulfate proteoglycan binds to fibronectin","volume":"265","author":"Heremans","year":"1990","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib35","doi-asserted-by":"crossref","first-page":"10953","DOI":"10.1074\/jbc.R112.437038","article-title":"Human genetic disorders caused by mutations in genes encoding biosynthetic enzymes for sulfated glycosaminoglycans","volume":"288","author":"Mizumoto","year":"2013","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib36","doi-asserted-by":"crossref","first-page":"20488","DOI":"10.1016\/S0021-9258(17)30530-6","article-title":"The polylactosaminoglycans of human lysosomal membrane glycoproteins lamp-1 and lamp-2. Localization on the peptide backbones","volume":"265","author":"Carlsson","year":"1990","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib37","doi-asserted-by":"crossref","first-page":"595","DOI":"10.1111\/jcmm.12346","article-title":"bFGF attenuates endoplasmic reticulum stress and mitochondrial injury on myocardial ischaemia\/reperfusion via activation of PI3K\/Akt\/ERK1\/2 pathway","volume":"19","author":"Wang","year":"2015","journal-title":"J.\u00a0Cell. Mol. Med."},{"key":"10.1016\/j.omtm.2018.05.002_bib38","doi-asserted-by":"crossref","first-page":"316","DOI":"10.1161\/01.RES.0000112965.70691.AC","article-title":"Heparan sulfate proteoglycans function as receptors for fibroblast growth factor-2 activation of extracellular signal-regulated kinases 1 and 2","volume":"94","author":"Chua","year":"2004","journal-title":"Circ. Res."},{"key":"10.1016\/j.omtm.2018.05.002_bib39","doi-asserted-by":"crossref","first-page":"1961","DOI":"10.1016\/j.cellsig.2011.07.005","article-title":"Intracellular signaling cascades triggered by the NK1 fragment of hepatocyte growth factor in human prostate epithelial cell line PNT1A","volume":"23","author":"Pavone","year":"2011","journal-title":"Cell. Signal."},{"key":"10.1016\/j.omtm.2018.05.002_bib40","doi-asserted-by":"crossref","first-page":"1643","DOI":"10.1093\/hmg\/ddx069","article-title":"Perturbations in cell signaling elicit early cardiac defects in mucopolysaccharidosis type II","volume":"26","author":"Costa","year":"2017","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2018.05.002_bib41","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.bone.2016.01.029","article-title":"Altered interaction and distribution of glycosaminoglycans and growth factors in mucopolysaccharidosis type I bone disease","volume":"88","author":"Kingma","year":"2016","journal-title":"Bone"},{"key":"10.1016\/j.omtm.2018.05.002_bib42","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1038\/nrm3012","article-title":"MET signalling: principles and functions in development, organ regeneration and cancer","volume":"11","author":"Trusolino","year":"2010","journal-title":"Nat. Rev. Mol. Cell Biol."},{"key":"10.1016\/j.omtm.2018.05.002_bib43","doi-asserted-by":"crossref","first-page":"26166","DOI":"10.1016\/S0021-9258(18)35731-4","article-title":"Expression and characterization of hepatocyte growth factor receptor-IgG fusion proteins. Effects of mutations in the potential proteolytic cleavage site on processing and ligand binding","volume":"267","author":"Mark","year":"1992","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib44","doi-asserted-by":"crossref","first-page":"129","DOI":"10.1146\/annurev-biochem-060713-035314","article-title":"Demystifying heparan sulfate-protein interactions","volume":"83","author":"Xu","year":"2014","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.omtm.2018.05.002_bib45","doi-asserted-by":"crossref","first-page":"1999","DOI":"10.1242\/jcs.112.12.1999","article-title":"Differential regulation of hepatocyte growth factor\/scatter factor by cell surface proteoglycans and free glycosaminoglycan chains","volume":"112","author":"Deakin","year":"1999","journal-title":"J.\u00a0Cell Sci."},{"key":"10.1016\/j.omtm.2018.05.002_bib46","doi-asserted-by":"crossref","first-page":"1040","DOI":"10.1074\/jbc.M107506200","article-title":"The mode of action of heparan and dermatan sulfates in the regulation of hepatocyte growth factor\/scatter factor","volume":"277","author":"Lyon","year":"2002","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib47","doi-asserted-by":"crossref","first-page":"32977","DOI":"10.1074\/jbc.M105486200","article-title":"Dissociation of heparan sulfate and receptor binding domains of hepatocyte growth factor reveals that heparan sulfate-c-met interaction facilitates signaling","volume":"276","author":"Rubin","year":"2001","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib48","doi-asserted-by":"crossref","first-page":"17094","DOI":"10.1074\/jbc.M000237200","article-title":"Stimulation of DNA synthesis and cell proliferation of human mammary myoepithelial-like cells by hepatocyte growth factor\/scatter factor depends on heparan sulfate proteoglycans and sustained phosphorylation of mitogen-activated protein kinases p42\/44","volume":"275","author":"Sergeant","year":"2000","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib49","doi-asserted-by":"crossref","first-page":"11216","DOI":"10.1016\/S0021-9258(19)78113-7","article-title":"Interaction of hepatocyte growth factor with heparan sulfate. Elucidation of the major heparan sulfate structural determinants","volume":"269","author":"Lyon","year":"1994","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib50","doi-asserted-by":"crossref","first-page":"6003","DOI":"10.1021\/bi972468t","article-title":"Hepatocyte growth factor\/scatter factor has distinct classes of binding site in heparan sulfate from mammary cells","volume":"37","author":"Rahmoune","year":"1998","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtm.2018.05.002_bib51","doi-asserted-by":"crossref","first-page":"271","DOI":"10.1074\/jbc.273.1.271","article-title":"Hepatocyte growth factor\/scatter factor binds with high affinity to dermatan sulfate","volume":"273","author":"Lyon","year":"1998","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib52","doi-asserted-by":"crossref","first-page":"48341","DOI":"10.1074\/jbc.M108395200","article-title":"Endocan is a novel chondroitin sulfate\/dermatan sulfate proteoglycan that promotes hepatocyte growth factor\/scatter factor mitogenic activity","volume":"276","author":"B\u00e9chard","year":"2001","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib53","doi-asserted-by":"crossref","first-page":"9457","DOI":"10.1074\/jbc.272.14.9457","article-title":"Heparin binding and oligomerization of hepatocyte growth factor\/scatter factor isoforms. Heparan sulfate glycosaminoglycan requirement for Met binding and signaling","volume":"272","author":"Sakata","year":"1997","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib54","doi-asserted-by":"crossref","first-page":"1383","DOI":"10.1016\/S0969-2126(98)00138-5","article-title":"Crystal structure of the NK1 fragment of human hepatocyte growth factor at 2.0 A resolution","volume":"6","author":"Ultsch","year":"1998","journal-title":"Structure"},{"key":"10.1016\/j.omtm.2018.05.002_bib55","doi-asserted-by":"crossref","first-page":"1956","DOI":"10.1182\/blood-2005-02-0657","article-title":"Functional abnormalities of heparan sulfate in mucopolysaccharidosis-I are associated with defective biologic activity of FGF-2 on human multipotent progenitor cells","volume":"106","author":"Pan","year":"2005","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2018.05.002_bib56","doi-asserted-by":"crossref","first-page":"36194","DOI":"10.1074\/jbc.M114.599944","article-title":"Heparan sulfate inhibits hematopoietic stem and progenitor cell migration and engraftment in mucopolysaccharidosis I","volume":"289","author":"Watson","year":"2014","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib57","doi-asserted-by":"crossref","first-page":"40","DOI":"10.1038\/s41419-017-0187-0","article-title":"EGFR activation triggers cellular hypertrophy and lysosomal disease in NAGLU-depleted cardiomyoblasts, mimicking the hallmarks of mucopolysaccharidosis IIIB","volume":"9","author":"De Pasquale","year":"2018","journal-title":"Cell Death Dis."},{"key":"10.1016\/j.omtm.2018.05.002_bib58","doi-asserted-by":"crossref","first-page":"37515","DOI":"10.1074\/jbc.M111.287474","article-title":"Mucopolysaccharidosis type I, unique structure of accumulated heparan sulfate and increased N-sulfotransferase activity in mice lacking \u03b1-l-iduronidase","volume":"286","author":"Holley","year":"2011","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2018.05.002_bib59","doi-asserted-by":"crossref","first-page":"46576","DOI":"10.1038\/srep46576","article-title":"Neurodevelopmental changes in excitatory synaptic structure and function in the cerebralcortex of Sanfilippo syndrome IIIA mice","volume":"7","author":"Dwyer","year":"2017","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2018.05.002_bib60","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.ymgme.2014.09.012","article-title":"Extracellular matrix disruption is an early event in the pathogenesis of skeletal disease in mucopolysaccharidosis I","volume":"114","author":"Heppner","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2018.05.002_bib61","doi-asserted-by":"crossref","first-page":"1030","DOI":"10.1038\/nature05817","article-title":"Heparan sulphate proteoglycans fine-tune mammalian physiology","volume":"446","author":"Bishop","year":"2007","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2018.05.002_bib62","doi-asserted-by":"crossref","first-page":"177","DOI":"10.1007\/BF00437470","article-title":"Involvement of heparan sulfate and related molecules in sequestration and growth promoting activity of fibroblast growth factor","volume":"15","author":"Vlodavsky","year":"1996","journal-title":"Cancer Metastasis Rev."},{"key":"10.1016\/j.omtm.2018.05.002_bib63","doi-asserted-by":"crossref","first-page":"13862","DOI":"10.1021\/bi801007p","article-title":"Minimum FGF2 binding structural requirements of heparin and heparan sulfate oligosaccharides as determined by NMR spectroscopy","volume":"47","author":"Guglier","year":"2008","journal-title":"Biochemistry"},{"key":"10.1016\/j.omtm.2018.05.002_bib64","doi-asserted-by":"crossref","first-page":"857","DOI":"10.1016\/0955-0674(90)90084-R","article-title":"The affinity of fibroblast growth factors (FGFs) for heparin; FGF-heparan sulfate interactions in cells and extracellular matrix","volume":"2","author":"Klagsbrun","year":"1990","journal-title":"Curr. Opin. Cell Biol."},{"key":"10.1016\/j.omtm.2018.05.002_bib65","doi-asserted-by":"crossref","first-page":"1542","DOI":"10.1016\/j.bbadis.2011.07.013","article-title":"Bone, joint and tooth development in mucopolysaccharidoses: relevance to therapeutic options","volume":"1812","author":"Oussoren","year":"2011","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtm.2018.05.002_bib66","doi-asserted-by":"crossref","DOI":"10.1093\/hmg\/ddy131","article-title":"FGF signaling deregulation is associated with early developmental skeletal defects in animal models for mucopolysaccharidosis type II (MPSII)","author":"Bellesso","year":"2018","journal-title":"Hum. Mol. Genet."},{"issue":"Suppl 1","key":"10.1016\/j.omtm.2018.05.002_bib67","doi-asserted-by":"crossref","first-page":"188","DOI":"10.1111\/j.1440-1746.2010.06549.x","article-title":"Hepatocyte growth factor twenty years on: Much more than a growth factor","volume":"26","author":"Nakamura","year":"2011","journal-title":"J.\u00a0Gastroenterol. Hepatol."},{"key":"10.1016\/j.omtm.2018.05.002_bib68","doi-asserted-by":"crossref","first-page":"e2","DOI":"10.5334\/ojb.ab","article-title":"Cell line and DNA biobank from patients affected by genetic diseases","volume":"1","author":"Filocamo","year":"2014","journal-title":"Open J. Bioresour."},{"key":"10.1016\/j.omtm.2018.05.002_bib69","series-title":"The Protein Protocols Handbook","first-page":"7","article-title":"The Lowry method for protein quantitation","author":"Waterborg","year":"2002"},{"key":"10.1016\/j.omtm.2018.05.002_bib70","article-title":"Labeling membranes with fluorescent phosphatidylethanolamine","volume":"2011","author":"Chazotte","year":"2011","journal-title":"Cold Spring Harb. Protoc."},{"key":"10.1016\/j.omtm.2018.05.002_bib71","doi-asserted-by":"crossref","first-page":"e0131662","DOI":"10.1371\/journal.pone.0131662","article-title":"The murine model of Mucopolysaccharidosis IIIB develops cardiopathies over time leading to heart failure","volume":"10","author":"Schiattarella","year":"2015","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2018.05.002_bib72","doi-asserted-by":"crossref","first-page":"496","DOI":"10.2478\/s11658-010-0019-z","article-title":"Integrin receptors play a role in the internalin B-dependent entry of Listeria monocytogenes into host cells","volume":"15","author":"Auriemma","year":"2010","journal-title":"Cell. Mol. Biol. Lett."},{"key":"10.1016\/j.omtm.2018.05.002_bib73","doi-asserted-by":"crossref","first-page":"549","DOI":"10.1016\/j.nmd.2012.01.009","article-title":"Role of serotonergic system in the pathogenesis of fibrosis in canine idiopathic inflammatory myopathies","volume":"22","author":"Pavone","year":"2012","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtm.2018.05.002_bib74","doi-asserted-by":"crossref","first-page":"384","DOI":"10.1002\/ar.21353","article-title":"Fate map of serotonin transporter-expressing cells in developing mouse thyroid","volume":"294","author":"Spina","year":"2011","journal-title":"Anat. Rec. (Hoboken)"},{"key":"10.1016\/j.omtm.2018.05.002_bib75","doi-asserted-by":"crossref","first-page":"530","DOI":"10.1038\/nrm1681","article-title":"Heparan sulphate proteoglycans: the sweet side of development","volume":"6","author":"H\u00e4cker","year":"2005","journal-title":"Nat. Rev. Mol. Cell Biol."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050118300482?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050118300482?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:23:34Z","timestamp":1762421014000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050118300482"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,9]]},"references-count":75,"alternative-id":["S2329050118300482"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2018.05.002","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2018,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Targeting Heparan Sulfate Proteoglycans as a Novel Therapeutic Strategy for Mucopolysaccharidoses","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2018.05.002","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2018 The Author(s).","name":"copyright","label":"Copyright"}]}}