{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T14:31:54Z","timestamp":1791210714951,"version":"4.1.0"},"reference-count":48,"publisher":"Elsevier BV","issue":"8","license":[{"start":{"date-parts":[[2012,8,1]],"date-time":"2012-08-01T00:00:00Z","timestamp":1343779200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":1528,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2012,8]]},"DOI":"10.1038\/mt.2012.82","type":"journal-article","created":{"date-parts":[[2012,5,1]],"date-time":"2012-05-01T08:25:36Z","timestamp":1335860736000},"page":"1610-1621","source":"Crossref","is-referenced-by-count":93,"title":["Hematopoietic Stem Cell and Gene Therapy Corrects Primary Neuropathology and Behavior in Mucopolysaccharidosis IIIA Mice"],"prefix":"10.1016","volume":"20","author":[{"given":"Alexander","family":"Langford-Smith","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona L","family":"Wilkinson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kia J","family":"Langford-Smith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca J","family":"Holley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana","family":"Sergijenko","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Steven J","family":"Howe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"William R","family":"Bennett","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon A","family":"Jones","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"JE","family":"Wraith","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine LR","family":"Merry","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert F","family":"Wynn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian W","family":"Bigger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/mt.2012.82_bib1","doi-asserted-by":"crossref","first-page":"240","DOI":"10.1007\/s10545-008-0838-5","article-title":"Sanfilippo syndrome: A mini-review","volume":"31","author":"Valstar","year":"2008","journal-title":"J Inher Metabol Dis"},{"key":"10.1038\/mt.2012.82_bib2","doi-asserted-by":"crossref","first-page":"58","DOI":"10.1002\/ajmg.a.33779","article-title":"Incidence and natural history of mucopolysaccharidosis type III in France and comparison with United Kingdom and Greece","volume":"155A","author":"H\u00e9ron","year":"2011","journal-title":"Am J Med Genet A"},{"key":"10.1038\/mt.2012.82_bib3","doi-asserted-by":"crossref","first-page":"e1255","DOI":"10.1542\/peds.2007-0282","article-title":"Scoring evaluation of the natural course of mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A)","volume":"120","author":"Meyer","year":"2007","journal-title":"Pediatrics"},{"key":"10.1038\/mt.2012.82_bib4","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":"10.1038\/mt.2012.82_bib5","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1002\/cne.20355","article-title":"Differential subcellular localization of cholesterol, gangliosides, and glycosaminoglycans in murine models of mucopolysaccharide storage disorders","volume":"480","author":"McGlynn","year":"2004","journal-title":"J Comp Neurol"},{"key":"10.1038\/mt.2012.82_bib6","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":"10.1038\/mt.2012.82_bib7","doi-asserted-by":"crossref","first-page":"18","DOI":"10.1016\/j.ymgme.2011.01.015","article-title":"Neuroinflammatory and oxidative stress phenomena in MPS IIIA mouse model: the positive effect of long-term aspirin treatment","volume":"103","author":"Arfi","year":"2011","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2012.82_bib8","doi-asserted-by":"crossref","first-page":"273","DOI":"10.1016\/j.ymgme.2004.05.005","article-title":"Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA","volume":"82","author":"Savas","year":"2004","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2012.82_bib9","doi-asserted-by":"crossref","DOI":"10.3324\/haematol.2011.058644","article-title":"Hematopoietic stem cell transplantation ameliorates the high incidence of neutralizing allo-antibodies observed in MPSI-Hurler after pharmacological enzyme replacement therapy","author":"Saif","year":"2012","journal-title":"Haematologica"},{"key":"10.1038\/mt.2012.82_bib10","doi-asserted-by":"crossref","first-page":"618","DOI":"10.1016\/j.bbmt.2009.01.020","article-title":"Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome","volume":"15","author":"Boelens","year":"2009","journal-title":"Biol Blood Marrow Transplant"},{"key":"10.1038\/mt.2012.82_bib11","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1016\/j.jpeds.2008.07.004","article-title":"Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients","volume":"154","author":"Wynn","year":"2009","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2012.82_bib12","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1016\/0963-6897(95)00021-O","article-title":"Microglia: the effector cell for reconstitution of the central nervous system following bone marrow transplantation for lysosomal and peroxisomal storage diseases","volume":"4","author":"Krivit","year":"1995","journal-title":"Cell Transplant"},{"key":"10.1038\/mt.2012.82_bib13","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1007\/BF00710053","article-title":"Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation","volume":"18","author":"Shapiro","year":"1995","journal-title":"J Inherit Metab Dis"},{"key":"10.1038\/mt.2012.82_bib14","doi-asserted-by":"crossref","first-page":"849","DOI":"10.1023\/A:1005526628598","article-title":"Bone marrow transplantation in mucopolysaccharidosis type IIIA: a comparison of an early treated patient with his untreated sibling","volume":"22","author":"Sivakumur","year":"1999","journal-title":"J Inherit Metab Dis"},{"key":"10.1038\/mt.2012.82_bib15","doi-asserted-by":"crossref","first-page":"2979","DOI":"10.1182\/blood-2008-03-140830","article-title":"Unrelated donor umbilical cord blood transplantation for inherited metabolic disorders in 159 pediatric patients from a single center: influence of cellular composition of the graft on transplantation outcomes","volume":"112","author":"Prasad","year":"2008","journal-title":"Blood"},{"key":"10.1038\/mt.2012.82_bib16","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1016\/j.jpeds.2008.11.005","article-title":"Improved metabolic correction in patients with lysosomal storage disease treated with hematopoietic stem cell transplant compared with enzyme replacement therapy","volume":"154","author":"Wynn","year":"2009","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2012.82_bib17","doi-asserted-by":"crossref","first-page":"873","DOI":"10.1038\/mt.2010.17","article-title":"Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB","volume":"18","author":"Heldermon","year":"2010","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2012.82_bib18","doi-asserted-by":"crossref","first-page":"10229","DOI":"10.1523\/JNEUROSCI.3558-04.2004","article-title":"Improved behavior and neuropathology in the mouse model of Sanfilippo type IIIB disease after adeno-associated virus-mediated gene transfer in the striatum","volume":"24","author":"Cressant","year":"2004","journal-title":"J Neurosci"},{"key":"10.1038\/mt.2012.82_bib19","doi-asserted-by":"crossref","first-page":"1528","DOI":"10.1038\/sj.gt.3302011","article-title":"Recombinant AAV serotype 1 transduction efficiency and tropism in the murine brain","volume":"10","author":"Wang","year":"2003","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2012.82_bib20","doi-asserted-by":"crossref","first-page":"195","DOI":"10.1089\/hum.2006.178","article-title":"Comparative analysis of adeno-associated viral vector serotypes 1, 2, 5, 7, and 8 in mouse brain","volume":"18","author":"Taymans","year":"2007","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2012.82_bib21","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1089\/hum.2008.022","article-title":"Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA","volume":"19","author":"Worgall","year":"2008","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2012.82_bib22","doi-asserted-by":"crossref","first-page":"5130","DOI":"10.1182\/blood-2010-04-278234","article-title":"Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model","volume":"116","author":"Visigalli","year":"2010","journal-title":"Blood"},{"key":"10.1038\/mt.2012.82_bib23","doi-asserted-by":"crossref","first-page":"3070","DOI":"10.1172\/JCI28873","article-title":"Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice","volume":"116","author":"Biffi","year":"2006","journal-title":"J Clin Invest"},{"key":"10.1038\/mt.2012.82_bib24","doi-asserted-by":"crossref","first-page":"58ra84","DOI":"10.1126\/scitranslmed.3001522","article-title":"Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy","volume":"2","author":"Gentner","year":"2010","journal-title":"Sci Transl Med"},{"key":"10.1038\/mt.2012.82_bib25","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"10.1038\/mt.2012.82_bib26","doi-asserted-by":"crossref","first-page":"19958","DOI":"10.1073\/pnas.0908528106","article-title":"Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome","volume":"106","author":"Wang","year":"2009","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/mt.2012.82_bib27","doi-asserted-by":"crossref","first-page":"445","DOI":"10.1016\/j.expneurol.2010.07.024","article-title":"Allogeneic stem cell transplantation does not improve neurological deficits in mucopolysaccharidosis type IIIA mice","volume":"225","author":"Lau","year":"2010","journal-title":"Exp Neurol"},{"key":"10.1038\/mt.2012.82_bib28","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1016\/j.ymgme.2004.06.004","article-title":"Retrovirally transduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB","volume":"82","author":"Zheng","year":"2004","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2012.82_bib29","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1111\/j.1601-183X.2011.00706.x","article-title":"Hyperactive behaviour in the mouse model of mucopolysaccharidosis IIIB in the open field and home cage environments","volume":"10","author":"Langford-Smith","year":"2011","journal-title":"Genes Brain Behav"},{"key":"10.1038\/mt.2012.82_bib30","doi-asserted-by":"crossref","first-page":"e25717","DOI":"10.1371\/journal.pone.0025717","article-title":"Female mucopolysaccharidosis IIIA mice exhibit hyperactivity and a reduced sense of danger in the open field test","volume":"6","author":"Langford-Smith","year":"2011","journal-title":"PLoS ONE"},{"key":"10.1038\/mt.2012.82_bib31","doi-asserted-by":"crossref","first-page":"537","DOI":"10.1016\/j.ymthe.2005.01.022","article-title":"Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells","volume":"12","author":"Siapati","year":"2005","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2012.82_bib32","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1038\/sj.gt.3302638","article-title":"Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX","volume":"13","author":"Bigger","year":"2006","journal-title":"Gene Ther"},{"key":"10.1038\/mt.2012.82_bib33","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 a-l-iduronidase","volume":"286","author":"Holley","year":"2011","journal-title":"J Biol Chem"},{"key":"10.1038\/mt.2012.82_bib34","doi-asserted-by":"crossref","first-page":"e2296","DOI":"10.1371\/journal.pone.0002296","article-title":"Early neurodegeneration progresses independently of microglial activation by heparan sulfate in the brain of mucopolysaccharidosis IIIB mice","volume":"3","author":"Ausseil","year":"2008","journal-title":"PLoS ONE"},{"key":"10.1038\/mt.2012.82_bib35","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1111\/j.1601-183X.2008.00413.x","article-title":"Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice","volume":"7","author":"Hemsley","year":"2008","journal-title":"Genes, Brain and Behavior"},{"key":"10.1038\/mt.2012.82_bib36","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1016\/j.nbd.2011.04.014","article-title":"Enzyme replacement reduces neuropathology in MPS IIIA dogs","volume":"43","author":"Crawley","year":"2011","journal-title":"Neurobiol Dis"},{"key":"10.1038\/mt.2012.82_bib37","doi-asserted-by":"crossref","first-page":"e14192","DOI":"10.1371\/journal.pone.0014192","article-title":"Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic disease","volume":"5","author":"Malinowska","year":"2010","journal-title":"PLoS ONE"},{"key":"10.1038\/mt.2012.82_bib38","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1016\/j.jpeds.2008.07.004","article-title":"Use of enzyme replacement therapy (Laronidase) before hematopoietic stem cell transplantation for mucopolysaccharidosis I: experience in 18 patients","volume":"154","author":"Wynn","year":"2009","journal-title":"J Pediatr"},{"key":"10.1038\/mt.2012.82_bib39","doi-asserted-by":"crossref","first-page":"1919","DOI":"10.1038\/mt.2009.179","article-title":"Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors","volume":"17","author":"Modlich","year":"2009","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2012.82_bib40","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1089\/10430340252898984","article-title":"High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of imunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter","volume":"13","author":"Demaison","year":"2002","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2012.82_bib41","doi-asserted-by":"crossref","first-page":"1788","DOI":"10.1101\/gr.143000","article-title":"DNA cloning using in vitro site-specific recombination","volume":"10","author":"Hartley","year":"2000","journal-title":"Genome Res"},{"key":"10.1038\/mt.2012.82_bib42","doi-asserted-by":"crossref","first-page":"495","DOI":"10.1038\/nprot.2009.22","article-title":"Production, concentration and titration of pseudotyped HIV-1-based lentiviral vectors","volume":"4","author":"Kutner","year":"2009","journal-title":"Nat Protoc"},{"key":"10.1038\/mt.2012.82_bib43","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1089\/104303403764539387","article-title":"Real-time quantitative reverse transcriptase-polymerase chain reaction as a method for determining lentiviral vector titers and measuring transgene expression","volume":"14","author":"Liz\u00e9e","year":"2003","journal-title":"Hum Gene Ther"},{"key":"10.1038\/mt.2012.82_bib44","doi-asserted-by":"crossref","first-page":"763","DOI":"10.1016\/j.ymthe.2005.07.358","article-title":"Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice","volume":"12","author":"Themis","year":"2005","journal-title":"Mol Ther"},{"key":"10.1038\/mt.2012.82_bib45","doi-asserted-by":"crossref","first-page":"269","DOI":"10.1016\/j.ymgme.2009.10.175","article-title":"Evaluation of heparin cofactor II-thrombin complex as a biomarker on blood spots from mucopolysaccharidosis I, IIIA and IIIB mice","volume":"99","author":"Langford-Smith","year":"2010","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2012.82_bib46","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 Inherit Metab Dis"},{"key":"10.1038\/mt.2012.82_bib47","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1016\/j.ymgme.2009.06.013","article-title":"Genistein reduces lysosomal storage in peripheral tissues of mucopolysaccharide IIIB mice","volume":"98","author":"Malinowska","year":"2009","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mt.2012.82_bib48","doi-asserted-by":"crossref","first-page":"212","DOI":"10.1016\/j.bbr.2010.01.045","article-title":"Circadian rhythm and suprachiasmatic nucleus alterations in the mouse model of mucopolysaccharidosis IIIB","volume":"209","author":"Canal","year":"2010","journal-title":"Behav Brain Res"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616325965?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616325965?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2019,6,27]],"date-time":"2019-06-27T23:55:26Z","timestamp":1561679726000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616325965"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2012,8]]},"references-count":48,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2012,8]]}},"alternative-id":["S1525001616325965"],"URL":"https:\/\/doi.org\/10.1038\/mt.2012.82","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2012,8]]}}}