{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:22:14Z","timestamp":1773274934234,"version":"3.50.1"},"reference-count":33,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2018,4,1]],"date-time":"2018-04-01T00:00:00Z","timestamp":1522540800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2018,4,1]],"date-time":"2018-04-01T00:00:00Z","timestamp":1522540800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2018,1,15]],"date-time":"2018-01-15T00:00:00Z","timestamp":1515974400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100013927","name":"National MPS Society","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100013927","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2018,4]]},"DOI":"10.1016\/j.ymthe.2018.01.010","type":"journal-article","created":{"date-parts":[[2018,1,30]],"date-time":"2018-01-30T23:33:42Z","timestamp":1517355222000},"page":"1118-1126","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":16,"title":["Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy"],"prefix":"10.1016","volume":"26","author":[{"given":"Yonghong","family":"Chen","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shujuan","family":"Zheng","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Luis","family":"Tecedor","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Beverly L.","family":"Davidson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"issue":"Suppl 1","key":"10.1016\/j.ymthe.2018.01.010_bib1","doi-asserted-by":"crossref","first-page":"S27","DOI":"10.1016\/j.jpeds.2004.01.052","article-title":"The mucopolysaccharidoses: a heterogeneous group of disorders with variable pediatric presentations","volume":"144","author":"Muenzer","year":"2004","journal-title":"J.\u00a0Pediatr."},{"key":"10.1016\/j.ymthe.2018.01.010_bib2","doi-asserted-by":"crossref","first-page":"65","DOI":"10.1203\/01.PDR.0000129661.40499.12","article-title":"Enzyme-replacement therapy from birth delays the development of behavior and learning problems in mucopolysaccharidosis type IIIA mice","volume":"56","author":"Gliddon","year":"2004","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymthe.2018.01.010_bib3","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.ymgme.2006.10.005","article-title":"Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice","volume":"90","author":"Hemsley","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2018.01.010_bib4","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.expneurol.2014.09.008","article-title":"Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods","volume":"263","author":"Marshall","year":"2015","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2018.01.010_bib5","doi-asserted-by":"crossref","first-page":"3254","DOI":"10.1172\/JCI66778","article-title":"Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy","volume":"123","author":"Haurigot","year":"2013","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2018.01.010_bib6","doi-asserted-by":"crossref","first-page":"1237","DOI":"10.1089\/hum.2012.029","article-title":"Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer","volume":"23","author":"Ruzo","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib7","doi-asserted-by":"crossref","first-page":"16036","DOI":"10.1038\/mtm.2016.36","article-title":"Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery","volume":"3","author":"Fu","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2018.01.010_bib8","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.ymthe.2018.01.010_bib9","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1038\/gt.2015.111","article-title":"Mucopolysaccharidosis IIIB confers enhanced neonatal intracranial transduction by AAV8 but not by 5, 9 or rh10","volume":"23","author":"Gilkes","year":"2016","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib10","doi-asserted-by":"crossref","first-page":"1393","DOI":"10.1038\/mt.2012.100","article-title":"Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease","volume":"20","author":"Chen","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib11","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/mt.2008.9","article-title":"Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Chang","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib12","doi-asserted-by":"crossref","first-page":"313ra180","DOI":"10.1126\/scitranslmed.aac6191","article-title":"AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease","volume":"7","author":"Katz","year":"2015","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2018.01.010_bib13","doi-asserted-by":"crossref","first-page":"9321","DOI":"10.1523\/JNEUROSCI.2936-05.2005","article-title":"Functional correction of CNS phenotypes in a lysosomal storage disease model using adeno-associated virus type 4 vectors","volume":"25","author":"Liu","year":"2005","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.010_bib14","series-title":"The Neuronal Ceroid Lipofuscinoses (Batten Disease)","first-page":"80","article-title":"CLN2","author":"Chang","year":"2011"},{"key":"10.1016\/j.ymthe.2018.01.010_bib15","doi-asserted-by":"crossref","DOI":"10.1074\/mcp.M111.013284","article-title":"A proteome-wide, quantitative survey of in\u00a0vivo ubiquitylation sites reveals widespread regulatory roles","volume":"10","author":"Wagner","year":"2011","journal-title":"Mol. Cell Proteomics"},{"key":"10.1016\/j.ymthe.2018.01.010_bib16","doi-asserted-by":"crossref","first-page":"3428","DOI":"10.1073\/pnas.97.7.3428","article-title":"Recombinant adeno-associated virus type 2, 4, and 5 vectors: transduction of variant cell types and regions in the mammalian central nervous system","volume":"97","author":"Davidson","year":"2000","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2018.01.010_bib17","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1093\/glycob\/9.12.1389","article-title":"A\u00a0mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome)","volume":"9","author":"Bhaumik","year":"1999","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymthe.2018.01.010_bib18","doi-asserted-by":"crossref","first-page":"6565","DOI":"10.1016\/S0021-9258(19)69026-5","article-title":"Secretion of lysosomal enzymes in the cellular slime mold, Dictyostelium discoideum","volume":"256","author":"Dimond","year":"1981","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2018.01.010_bib19","doi-asserted-by":"crossref","first-page":"259","DOI":"10.1016\/0014-4827(87)90304-1","article-title":"Secretion heterogeneity of lysosomal enzymes in Tetrahymena pyriformis","volume":"170","author":"Banno","year":"1987","journal-title":"Exp. Cell Res."},{"key":"10.1016\/j.ymthe.2018.01.010_bib20","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.2018.01.010_bib21","series-title":"Extensive release. Excessive storage. In Lysosomes","first-page":"319","author":"Holtzman","year":"1989"},{"key":"10.1016\/j.ymthe.2018.01.010_bib22","doi-asserted-by":"crossref","first-page":"538","DOI":"10.1016\/j.ymthe.2005.11.008","article-title":"CNS-directed AAV2-mediated gene therapy ameliorates functional deficits in a murine model of infantile neuronal ceroid lipofuscinosis","volume":"13","author":"Griffey","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib23","doi-asserted-by":"crossref","first-page":"3430","DOI":"10.1002\/j.1460-2075.1994.tb06648.x","article-title":"Differential sorting of lysosomal enzymes in mannose 6-phosphate\u00a0receptor-deficient fibroblasts","volume":"13","author":"Ludwig","year":"1994","journal-title":"EMBO J."},{"key":"10.1016\/j.ymthe.2018.01.010_bib24","doi-asserted-by":"crossref","first-page":"640","DOI":"10.1038\/90242","article-title":"The HIV Tat protein transduction domain improves the biodistribution of beta-glucuronidase expressed from recombinant viral vectors","volume":"19","author":"Xia","year":"2001","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2018.01.010_bib25","doi-asserted-by":"crossref","first-page":"345","DOI":"10.1016\/j.ymthe.2005.02.031","article-title":"Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice","volume":"12","author":"Orii","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib26","doi-asserted-by":"crossref","first-page":"212ra161","DOI":"10.1126\/scitranslmed.3007000","article-title":"Gene transfer of human\u00a0Apoe isoforms results in differential modulation of amyloid deposition and neurotoxicity in mouse brain","volume":"5","author":"Hudry","year":"2013","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2018.01.010_bib27","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1089\/hum.2016.172","article-title":"Impact of AAV capsid-specific T-cell responses on design and outcome of clinical gene transfer trials with recombinant adeno-associated\u00a0viral vectors: an evolving controversy","volume":"28","author":"Ertl","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib28","doi-asserted-by":"crossref","first-page":"1935","DOI":"10.1089\/10430340260355347","article-title":"Insect cells as a factory to produce adeno-associated virus type 2 vectors","volume":"13","author":"Urabe","year":"2002","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib29","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1007\/BF01799255","article-title":"A fluorimetric enzyme assay for the diagnosis of Sanfilippo disease type A (MPS IIIA)","volume":"19","author":"Karpova","year":"1996","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.ymthe.2018.01.010_bib30","doi-asserted-by":"crossref","first-page":"6559","DOI":"10.1074\/jbc.M507336200","article-title":"Determination of the substrate specificity of tripeptidyl-peptidase I using combinatorial peptide libraries and development of improved fluorogenic substrates","volume":"281","author":"Tian","year":"2006","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2018.01.010_bib31","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1038\/gt.2009.157","article-title":"High AAV vector purity results in serotype- and tissue-independent enhancement of transduction efficiency","volume":"17","author":"Ayuso","year":"2010","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.010_bib32","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/glycob\/11.1.99","article-title":"A\u00a0novel missense mutation in lysosomal sulfamidase is the basis of MPS III A in a spontaneous mouse mutant","volume":"11","author":"Bhattacharyya","year":"2001","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymthe.2018.01.010_bib33","doi-asserted-by":"crossref","first-page":"848","DOI":"10.1038\/nprot.2006.116","article-title":"Morris water maze: procedures for assessing spatial and related forms of learning and memory","volume":"1","author":"Vorhees","year":"2006","journal-title":"Nat. Protoc."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618300170?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618300170?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T17:52:20Z","timestamp":1761501140000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001618300170"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,4]]},"references-count":33,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2018,4]]}},"alternative-id":["S1525001618300170"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.01.010","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2018,4]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Overcoming Limitations Inherent in Sulfamidase to Improve Mucopolysaccharidosis IIIA Gene Therapy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.01.010","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"}]}}