{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,16]],"date-time":"2026-10-16T14:04:52Z","timestamp":1792159492468,"version":"4.4.0"},"reference-count":46,"publisher":"Public Library of Science (PLoS)","issue":"8","license":[{"start":{"date-parts":[[2010,8,16]],"date-time":"2010-08-16T00:00:00Z","timestamp":1281916800000},"content-version":"unspecified","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["www.plosone.org"],"crossmark-restriction":false},"short-container-title":["PLoS ONE"],"DOI":"10.1371\/journal.pone.0012194","type":"journal-article","created":{"date-parts":[[2010,8,16]],"date-time":"2010-08-16T17:06:35Z","timestamp":1281978395000},"page":"e12194","update-policy":"https:\/\/doi.org\/10.1371\/journal.pone.corrections_policy","source":"Crossref","is-referenced-by-count":56,"title":["Enzyme Replacement in a Human Model of Mucopolysaccharidosis IVA In Vitro and Its Biodistribution in the Cartilage of Wild Type Mice"],"prefix":"10.1371","volume":"5","author":[{"given":"Melita","family":"Dvorak-Ewell","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Wendt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuck","family":"Hague","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terri","family":"Christianson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vish","family":"Koppaka","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Danielle","family":"Crippen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emil","family":"Kakkis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michel","family":"Vellard","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"340","published-online":{"date-parts":[[2010,8,16]]},"reference":[{"key":"ref1","doi-asserted-by":"crossref","first-page":"357","DOI":"10.1007\/BF01799267","article-title":"Mucopolysaccharidosis type IVA (Morquio syndrome): a clinical review.","volume":"19","author":"H Northover","year":"1996","journal-title":"J Inherit Metab Dis"},{"key":"ref2","article-title":"The mucopolysaccharidoses.","author":"E Neufeld","year":"1995"},{"key":"ref3","doi-asserted-by":"crossref","first-page":"621","DOI":"10.1093\/glycob\/cwh076","article-title":"Glycosaminoglycans and proteoglycans in normal mitral valve leaflets and chordae: association with regions of tensile and compressive loading.","volume":"14","author":"KJ Grande-Allen","year":"2004","journal-title":"Glycobiology"},{"key":"ref4","doi-asserted-by":"crossref","first-page":"243","DOI":"10.1002\/ajmg.1320280529","article-title":"Electron microscopy in the diagnosis of lysosomal storage diseases.","volume":"Suppl 3","author":"C Vogler","year":"1987","journal-title":"Am J Med Genet"},{"key":"ref5","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/BF00432778","article-title":"Coronary intimal sclerosis in Morquio's syndrome.","volume":"379","author":"SM Factor","year":"1978","journal-title":"Virchows Arch A Pathol Anat Histol"},{"key":"ref6","article-title":"Implications of absence of clinical phenotype on Morquio A mice: Why rodents do not require skeletal keratan sulfate?","author":"AM Montano"},{"issue":"Suppl 2","key":"ref7","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1023\/A:1012463605992","article-title":"Variable clinical presentation in lysosomal storage disorders.","volume":"24","author":"M Beck","year":"2001","journal-title":"J Inherit Metab Dis"},{"key":"ref8","doi-asserted-by":"crossref","first-page":"851","DOI":"10.1302\/0301-620X.73B5.1910048","article-title":"The odontoid process in Morquio-Brailsford's disease. The effects of occipitocervical fusion.","volume":"73","author":"JM Stevens","year":"1991","journal-title":"J Bone Joint Surg Br"},{"key":"ref9","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1007\/s004310050872","article-title":"Cardiovascular changes in children with mucopolysaccharide storage diseases and related disorders\u2013clinical and echocardiographic findings in 64 patients.","volume":"157","author":"JH Dangel","year":"1998","journal-title":"Eur J Pediatr"},{"key":"ref10","doi-asserted-by":"crossref","first-page":"295","DOI":"10.2106\/00004623-196244020-00006","article-title":"Morquio's disease and dysplasia epiphysalis multiplex. A study of epiphyseal cartilage in seven cases.","volume":"44-A","author":"CE Anderson","year":"1962","journal-title":"J Bone Joint Surg Am"},{"key":"ref11","doi-asserted-by":"crossref","first-page":"217","DOI":"10.3109\/00313028609059462","article-title":"The histological and ultrastructural features of the epiphyseal plate in Morquio type A syndrome (mucopolysaccharidosis type IVA).","volume":"18","author":"J McClure","year":"1986","journal-title":"Pathology"},{"key":"ref12","first-page":"176","article-title":"The Morquio syndrome (mucopolysaccharidosis IV): Morphologic and biochemical studies.","volume":"137","author":"DW Hollister","year":"1975","journal-title":"Johns Hopkins Med J"},{"key":"ref13","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1111\/j.1651-2227.1982.tb09434.x","article-title":"Morphological and biochemical analysis of biopsy specimens in disorders of skeletal development.","volume":"71","author":"B Engfeldt","year":"1982","journal-title":"Acta Paediatr Scand"},{"key":"ref14","doi-asserted-by":"crossref","first-page":"196","DOI":"10.1016\/j.ymgme.2009.03.008","article-title":"Deficiency in N-acetylgalactosamine-6-sulfate sulfatase results in collagen perturbations in cartilage of Morquio syndrome A patients.","volume":"97","author":"RA Bank","year":"2009","journal-title":"Mol Genet Metab"},{"key":"ref15","doi-asserted-by":"crossref","first-page":"1311","DOI":"10.1016\/j.joca.2007.04.008","article-title":"A molecular and histological characterization of cartilage from patients with Morquio syndrome.","volume":"15","author":"L De Franceschi","year":"2007","journal-title":"Osteoarthritis Cartilage"},{"key":"ref16","first-page":"119","article-title":"Blood supply of the knee joint. A microangiographic study in children and adults.","author":"SS Shim","year":"1986","journal-title":"Clin Orthop Relat Res"},{"key":"ref17","first-page":"352","article-title":"Allograft cardiac valves. A view through the scanning electron microscope.","volume":"68","author":"JW Hammon Jr","year":"1974","journal-title":"J Thorac Cardiovasc Surg"},{"key":"ref18","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1038\/sj.bmt.1703839","article-title":"Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.","volume":"31","author":"C Peters","year":"2003","journal-title":"Bone Marrow Transplant"},{"key":"ref19","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1517\/13543784.11.5.675","article-title":"Enzyme replacement therapy for the mucopolysaccharide storage disorders.","volume":"11","author":"ED Kakkis","year":"2002","journal-title":"Expert Opin Investig Drugs"},{"key":"ref20","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1016\/S0092-8674(03)00348-9","article-title":"The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases.","volume":"113","author":"MP Cosma","year":"2003","journal-title":"Cell"},{"key":"ref21","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/S0378-1119(03)00746-7","article-title":"The human SUMF1 gene, required for posttranslational sulfatase modification, defines a new gene family which is conserved from pro- to eukaryotes.","volume":"316","author":"J Landgrebe","year":"2003","journal-title":"Gene"},{"key":"ref22","doi-asserted-by":"crossref","first-page":"305","DOI":"10.1042\/BJ20061783","article-title":"SUMF1 enhances sulfatase activities in vivo in five sulfatase deficiencies.","volume":"403","author":"A Fraldi","year":"2007","journal-title":"Biochem J"},{"key":"ref23","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1093\/oxfordjournals.jbchem.a123697","article-title":"N-acetylgalactosamine-6-sulfate sulfatase in human placenta: purification and characteristics.","volume":"110","author":"M Masue","year":"1991","journal-title":"J Biochem"},{"key":"ref24","doi-asserted-by":"crossref","first-page":"12115","DOI":"10.1016\/S0021-9258(18)63825-6","article-title":"Mannose 6-phosphate receptors and lysosomal enzyme targeting.","volume":"264","author":"NM Dahms","year":"1989","journal-title":"J Biol Chem"},{"issue":"Pt 1","key":"ref25","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1042\/bj3110333","article-title":"Expression, purification and characterization of recombinant human N-acetylgalactosamine-6-sulphatase.","volume":"311","author":"J Bielicki","year":"1995","journal-title":"Biochem J"},{"key":"ref26","doi-asserted-by":"crossref","first-page":"3349","DOI":"10.1093\/hmg\/ddg366","article-title":"Mouse model of N-acetylgalactosamine-6-sulfate sulfatase deficiency (Galns-\/-) produced by targeted disruption of the gene defective in Morquio A disease.","volume":"12","author":"S Tomatsu","year":"2003","journal-title":"Hum Mol Genet"},{"key":"ref27","doi-asserted-by":"crossref","first-page":"3321","DOI":"10.1093\/hmg\/ddi364","article-title":"Development of MPS IVA mouse (Galnstm(hC79S.mC76S)slu) tolerant to human N-acetylgalactosamine-6-sulfate sulfatase.","volume":"14","author":"S Tomatsu","year":"2005","journal-title":"Hum Mol Genet"},{"key":"ref28","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1016\/j.ymgme.2007.02.009","article-title":"Murine model (Galns(tm(C76S)slu)) of MPS IVA with missense mutation at the active site cysteine conserved among sulfatase proteins.","volume":"91","author":"S Tomatsu","year":"2007","journal-title":"Mol Genet Metab"},{"key":"ref29","doi-asserted-by":"crossref","first-page":"1253","DOI":"10.1038\/nprot.2008.95","article-title":"Primary culture and phenotyping of murine chondrocytes.","volume":"3","author":"M Gosset","year":"2008","journal-title":"Nat Protoc"},{"key":"ref30","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1016\/0014-5793(89)81651-5","article-title":"Expression of mRNAs for collagens and other matrix components in dedifferentiating and redifferentiating human chondrocytes in culture.","volume":"258","author":"K Elima","year":"1989","journal-title":"FEBS Lett"},{"key":"ref31","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1002\/jor.1100180606","article-title":"Enhanced proliferation and differentiation of human articular chondrocytes when seeded at low cell densities in alginate in vitro.","volume":"18","author":"TA Gagne","year":"2000","journal-title":"J Orthop Res"},{"key":"ref32","doi-asserted-by":"crossref","first-page":"381","DOI":"10.1016\/S0945-053X(99)00033-5","article-title":"Modulation of keratan sulfate synthesis on lumican by the action of cytokines on human articular chondrocytes.","volume":"18","author":"LI Melching","year":"1999","journal-title":"Matrix Biol"},{"key":"ref33","first-page":"308","article-title":"Keratocyte phenotype is enhanced in the absence of attachment to the substratum.","volume":"14","author":"ML Funderburgh","year":"2008","journal-title":"Mol Vis"},{"key":"ref34","doi-asserted-by":"crossref","first-page":"1003","DOI":"10.1016\/j.berh.2006.06.003","article-title":"Update on the biology of the chondrocyte and new approaches to treating cartilage diseases.","volume":"20","author":"MB Goldring","year":"2006","journal-title":"Best Pract Res Clin Rheumatol"},{"key":"ref35","doi-asserted-by":"crossref","first-page":"1747","DOI":"10.1074\/jbc.270.4.1747","article-title":"A COL2A1 mutation in achondrogenesis type II results in the replacement of type II collagen by type I and III collagens in cartilage.","volume":"270","author":"D Chan","year":"1995","journal-title":"J Biol Chem"},{"key":"ref36","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1096\/fasebj.11.4.9068611","article-title":"Heritable diseases of the skeleton. Part II: Molecular insights into skeletal development-matrix components and their homeostasis.","volume":"11","author":"S Mundlos","year":"1997","journal-title":"FASEB J"},{"key":"ref37","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1016\/j.ymgme.2007.01.004","article-title":"Characterization and pharmacokinetic study of recombinant human N-acetylgalactosamine-6-sulfate sulfatase.","volume":"91","author":"S Tomatsu","year":"2007","journal-title":"Mol Genet Metab"},{"key":"ref38","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1093\/hmg\/ddm353","article-title":"Enzyme replacement therapy in a murine model of Morquio A syndrome.","volume":"17","author":"S Tomatsu","year":"2008","journal-title":"Hum Mol Genet"},{"key":"ref39","article-title":"Enhancement of Drug Delivery: Enzyme-replacement Therapy for Murine Morquio A Syndrome.","author":"S Tomatsu","journal-title":"Mol Ther"},{"key":"ref40","doi-asserted-by":"crossref","first-page":"746","DOI":"10.1136\/adc.65.7.746","article-title":"Echocardiographic abnormalities in type IV mucopolysaccharidosis.","volume":"65","author":"RM John","year":"1990","journal-title":"Arch Dis Child"},{"key":"ref41","doi-asserted-by":"crossref","first-page":"207","DOI":"10.1016\/0009-8981(79)90090-1","article-title":"Identification of keratan sulfate in liver affected by Morquio syndrome.","volume":"93","author":"R Minami","year":"1979","journal-title":"Clin Chim Acta"},{"key":"ref42","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1620\/tjem.139.321","article-title":"Characterization of keratan sulfate isolated from liver affected by Morquio syndrome.","volume":"139","author":"R Minami","year":"1983","journal-title":"Tohoku J Exp Med"},{"key":"ref43","doi-asserted-by":"crossref","first-page":"165","DOI":"10.1007\/s10545-007-0529-7","article-title":"International Morquio A Registry: clinical manifestation and natural course of Morquio A disease.","volume":"30","author":"AM Montano","year":"2007","journal-title":"J Inherit Metab Dis"},{"key":"ref44","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1007\/s00401-007-0296-4","article-title":"Immune system irregularities in lysosomal storage disorders.","volume":"115","author":"JA Castaneda","year":"2008","journal-title":"Acta Neuropathol"},{"key":"ref45","doi-asserted-by":"crossref","first-page":"4645","DOI":"10.1182\/blood.V86.12.4645.bloodjournal86124645","article-title":"Impaired microbicidal capacity of mononuclear phagocytes from patients with type I Gaucher disease: partial correction by enzyme replacement therapy.","volume":"86","author":"L Marodi","year":"1995","journal-title":"Blood"},{"key":"ref46","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1016\/0009-8981(90)90339-T","article-title":"A fluorimetric enzyme assay for the diagnosis of Morquio disease type A (MPS IV A).","volume":"187","author":"OP van Diggelen","year":"1990","journal-title":"Clin Chim Acta"}],"container-title":["PLoS ONE"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/dx.plos.org\/10.1371\/journal.pone.0012194","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2021,11,6]],"date-time":"2021-11-06T00:24:45Z","timestamp":1636158285000},"score":1,"resource":{"primary":{"URL":"https:\/\/dx.plos.org\/10.1371\/journal.pone.0012194"}},"subtitle":[],"editor":[{"given":"Raphael","family":"Schiffmann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"editor"}]}],"short-title":[],"issued":{"date-parts":[[2010,8,16]]},"references-count":46,"journal-issue":{"issue":"8","published-online":{"date-parts":[[2010,8,16]]}},"URL":"https:\/\/doi.org\/10.1371\/journal.pone.0012194","relation":{},"ISSN":["1932-6203"],"issn-type":[{"value":"1932-6203","type":"electronic"}],"subject":[],"published":{"date-parts":[[2010,8,16]]}}}