{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T23:34:49Z","timestamp":1788305689927,"version":"build-2803163510"},"reference-count":45,"publisher":"Elsevier BV","issue":"1-2","license":[{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2024,9,1]],"date-time":"2024-09-01T00:00:00Z","timestamp":1725148800000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2024,9]]},"DOI":"10.1016\/j.ymgme.2024.108533","type":"journal-article","created":{"date-parts":[[2024,7,9]],"date-time":"2024-07-09T15:56:25Z","timestamp":1720540585000},"page":"108533","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Generation of a novel immunodeficient mouse model of Mucopolysaccharidosis type IIIA to test human stem cell-based therapies"],"prefix":"10.1016","volume":"143","author":[{"given":"Oriana","family":"Mandolfo","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Helen","family":"Parker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Asma'u","family":"Usman","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuko Ishikawa","family":"Learmonth","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rebecca J.","family":"Holley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrew","family":"MacDonald","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tristan","family":"McKay","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Bigger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"2","key":"10.1016\/j.ymgme.2024.108533_bb0005","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. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2024.108533_bb0010","author":"Disorders NOfR"},{"issue":"6","key":"10.1016\/j.ymgme.2024.108533_bb0015","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."},{"issue":"Suppl. 5","key":"10.1016\/j.ymgme.2024.108533_bb0020","doi-asserted-by":"crossref","first-page":"v4","DOI":"10.1093\/rheumatology\/ker394","article-title":"Overview of the mucopolysaccharidoses","volume":"50","author":"Muenzer","year":"2011","journal-title":"Rheumatology (Oxford)"},{"issue":"7","key":"10.1016\/j.ymgme.2024.108533_bb0025","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":"10.1016\/j.ymgme.2024.108533_bb0030","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.ymgme.2017.08.009","article-title":"Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations - neurological signs and symptoms","volume":"122S","author":"Shapiro","year":"2017","journal-title":"Mol. Genet. Metab."},{"issue":"9","key":"10.1016\/j.ymgme.2024.108533_bb0035","first-page":"1954","article-title":"Gene-modified neural progenitor cells for the treatment of neuropathic lysosomal storage diseases","volume":"18","author":"Mandolfo","year":"2023","journal-title":"Neural Regen. Res."},{"issue":"12","key":"10.1016\/j.ymgme.2024.108533_bb0040","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"},{"issue":"10","key":"10.1016\/j.ymgme.2024.108533_bb0045","doi-asserted-by":"crossref","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"},{"issue":"4","key":"10.1016\/j.ymgme.2024.108533_bb0050","doi-asserted-by":"crossref","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"},{"issue":"3","key":"10.1016\/j.ymgme.2024.108533_bb0055","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.201911185","article-title":"Haematopoietic stem cell gene therapy with IL1Ra rescues cognitive loss in mucopolysaccharidosis IIIA","volume":"12","author":"Parker","year":"2020","journal-title":"EMBO Mol. Med."},{"issue":"10","key":"10.1016\/j.ymgme.2024.108533_bb0060","doi-asserted-by":"crossref","first-page":"1938","DOI":"10.1038\/mt.2013.141","article-title":"Myeloid\/microglial driven autologous hematopoietic stem cell gene therapy corrects a Neuronopathic lysosomal disease","volume":"21","author":"Sergijenko","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2024.108533_bb0065","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/j.omtm.2019.04.001","article-title":"Pre-clinical safety and efficacy of lentiviral vector-mediated ex vivo stem cell gene therapy for the treatment of Mucopolysaccharidosis IIIA","volume":"13","author":"Ellison","year":"2019","journal-title":"Mol. Ther. - Methods Clin. Develop."},{"key":"10.1016\/j.ymgme.2024.108533_bb0070","author":"ISSCR"},{"issue":"6","key":"10.1016\/j.ymgme.2024.108533_bb0075","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.brainresbull.2007.07.018","article-title":"Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy","volume":"74","author":"Garcia-Rivera","year":"2007","journal-title":"Brain Res. Bull."},{"issue":"9","key":"10.1016\/j.ymgme.2024.108533_bb0080","doi-asserted-by":"crossref","first-page":"3175","DOI":"10.1182\/blood-2001-12-0207","article-title":"NOD\/SCID\/gamma(c)(null) mouse: an excellent recipient mouse model for engraftment of human cells","volume":"100","author":"Ito","year":"2002","journal-title":"Blood"},{"issue":"5","key":"10.1016\/j.ymgme.2024.108533_bb0085","doi-asserted-by":"crossref","first-page":"1370","DOI":"10.1634\/stemcells.2005-0340","article-title":"Hematopoietic engraftment of human embryonic stem cell-derived cells is regulated by recipient innate immunity","volume":"24","author":"Tian","year":"2006","journal-title":"Stem Cells"},{"issue":"8","key":"10.1016\/j.ymgme.2024.108533_bb0090","doi-asserted-by":"crossref","first-page":"1999","DOI":"10.1016\/j.stemcr.2021.06.008","article-title":"Improved hematopoietic stem cell transplantation upon inhibition of natural killer cell-derived interferon-gamma","volume":"16","author":"Lobo De Figueiredo-Pontes","year":"2021","journal-title":"Stem Cell Rep."},{"key":"10.1016\/j.ymgme.2024.108533_bb0095","article-title":"Humanized NOD\/SCID\/IL2r\u03b3null (hu-NSG) mouse model for HIV replication and latency studies","volume":"143","author":"Xia","year":"2019","journal-title":"J. Vis. Exp."},{"key":"10.1016\/j.ymgme.2024.108533_bb0100","doi-asserted-by":"crossref","first-page":"14068","DOI":"10.1038\/mtm.2014.68","article-title":"A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I","volume":"2","author":"Mendez","year":"2015","journal-title":"Mol. Ther. - Methods Clin. Develop."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0105","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1111\/j.1600-065X.2006.00461.x","article-title":"Bone marrow versus thymic pathways of natural killer cell development","volume":"214","author":"Di Santo","year":"2006","journal-title":"Immunol. Rev."},{"issue":"5","key":"10.1016\/j.ymgme.2024.108533_bb0110","doi-asserted-by":"crossref","first-page":"835","DOI":"10.1016\/j.stemcr.2015.02.022","article-title":"Ex vivo gene therapy using patient iPSC-derived NSCs reverses pathology in the brain of a homologous mouse model","volume":"4","author":"Griffin","year":"2015","journal-title":"Stem Cell Reports."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0115","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1097\/MOT.0b013e32834254cf","article-title":"Natural killer cells in rejection and tolerance of solid organ allografts","volume":"16","author":"Benichou","year":"2011","journal-title":"Curr. Opin. Organ Transplant."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0120","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-023-45178-0","article-title":"An immune deficient mouse model for mucopolysaccharidosis IIIA (Sanfilippo syndrome)","volume":"13","author":"Pollock","year":"2023","journal-title":"Sci. Rep."},{"issue":"10","key":"10.1016\/j.ymgme.2024.108533_bb0125","doi-asserted-by":"crossref","first-page":"6477","DOI":"10.4049\/jimmunol.174.10.6477","article-title":"Human lymphoid and myeloid cell development in NOD\/LtSz-<i>scid IL2R<\/i>\u03b3<i>null<\/i> mice engrafted with mobilized human Hemopoietic stem cells","volume":"174","author":"Shultz","year":"2005","journal-title":"J. Immunol."},{"issue":"11","key":"10.1016\/j.ymgme.2024.108533_bb0130","doi-asserted-by":"crossref","first-page":"786","DOI":"10.1038\/nri3311","article-title":"Humanized mice for immune system investigation: progress, promise and challenges","volume":"12","author":"Shultz","year":"2012","journal-title":"Nat. Rev. Immunol."},{"issue":"3","key":"10.1016\/j.ymgme.2024.108533_bb0135","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1093\/cvr\/cvv147","article-title":"Monocyte trafficking across the vessel wall","volume":"107","author":"Gerhardt","year":"2015","journal-title":"Cardiovasc. Res."},{"key":"10.1016\/j.ymgme.2024.108533_bb0140","doi-asserted-by":"crossref","first-page":"6303","DOI":"10.1021\/bi002670n","article-title":"Structural and functional analysis of the RANTES-GlycosaminoglycansInteractions","volume":"40","author":"Lo\u0131c Martin","year":"2001","journal-title":"Biochemistry"},{"issue":"4","key":"10.1016\/j.ymgme.2024.108533_bb0145","doi-asserted-by":"crossref","first-page":"1999","DOI":"10.3390\/ijms23041999","article-title":"Innate immunity in Mucopolysaccharide diseases","volume":"23","author":"Mandolfo","year":"2022","journal-title":"Int. J. Mol. Sci."},{"issue":"2","key":"10.1016\/j.ymgme.2024.108533_bb0150","doi-asserted-by":"crossref","first-page":"396","DOI":"10.3390\/jcm9020396","article-title":"Brain pathology in Mucopolysaccharidoses (MPS) patients with neurological forms","volume":"9","author":"Viana","year":"2020","journal-title":"J. Clin. Med."},{"issue":"4","key":"10.1016\/j.ymgme.2024.108533_bb0155","doi-asserted-by":"crossref","first-page":"1902","DOI":"10.1073\/pnas.252784899","article-title":"Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB","volume":"100","author":"Ohmi","year":"2003","journal-title":"Proc. Natl. Acad. Sci."},{"issue":"7","key":"10.1016\/j.ymgme.2024.108533_bb0160","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.201708730","article-title":"Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms","volume":"10","author":"Gleitz","year":"2018","journal-title":"EMBO Mol. Med."},{"issue":"4","key":"10.1016\/j.ymgme.2024.108533_bb0165","doi-asserted-by":"crossref","first-page":"978","DOI":"10.1002\/jnr.21912","article-title":"Innate and adaptive immune activation in the brain of MPS IIIB mouse model","volume":"87","author":"Dirosario","year":"2009","journal-title":"J. Neurosci. Res."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0170","doi-asserted-by":"crossref","first-page":"39","DOI":"10.1186\/1742-2094-7-39","article-title":"Mucopolysaccharidosis IIIB, a lysosomal storage disease, triggers a pathogenic CNS autoimmune response","volume":"7","author":"Killedar","year":"2010","journal-title":"J. Neuroinflammation"},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0175","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s10545-013-9613-3","article-title":"Mucopolysaccharide diseases: a complex interplay between neuroinflammation, microglial activation and adaptive immunity","volume":"37","author":"Archer","year":"2014","journal-title":"J. Inherit. Metab. Dis."},{"issue":"5","key":"10.1016\/j.ymgme.2024.108533_bb0180","doi-asserted-by":"crossref","first-page":"639","DOI":"10.1111\/jnc.14632","article-title":"The role of innate immunity in mucopolysaccharide diseases","volume":"148","author":"Parker","year":"2019","journal-title":"J. Neurochem."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0185","article-title":"Human genome-edited hematopoietic stem cells phenotypically correct Mucopolysaccharidosis type I. Nature","volume":"10","author":"Gomez-Ospina","year":"2019","journal-title":"Communications"},{"issue":"2","key":"10.1016\/j.ymgme.2024.108533_bb0190","doi-asserted-by":"crossref","first-page":"352","DOI":"10.5966\/sctm.2015-0414","article-title":"Generation of human induced pluripotent stem cell-derived Bona fide neural stem cells for ex vivo gene therapy of metachromatic Leukodystrophy","volume":"6","author":"Meneghini","year":"2017","journal-title":"Stem Cells Transl. Med."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0195","doi-asserted-by":"crossref","DOI":"10.1186\/s13041-016-0191-9","article-title":"Age-related changes in behavior in C57BL\/6J mice from young adulthood to middle age","volume":"9","author":"Shoji","year":"2016","journal-title":"Mol. Brain"},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0200","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.brainres.2006.05.079","article-title":"Characterization of a C57BL\/6 congenic mouse strain of mucopolysaccharidosis type IIIA","volume":"1104","author":"Crawley","year":"2006","journal-title":"Brain Res."},{"issue":"2","key":"10.1016\/j.ymgme.2024.108533_bb0205","doi-asserted-by":"crossref","first-page":"262","DOI":"10.3390\/biomedicines11020262","article-title":"Temporal appearance of enhanced innate anxiety in Alzheimer model mice","volume":"11","author":"Szab\u00f3","year":"2023","journal-title":"Biomedicines"},{"issue":"8","key":"10.1016\/j.ymgme.2024.108533_bb0210","doi-asserted-by":"crossref","first-page":"1610","DOI":"10.1038\/mt.2012.82","article-title":"Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in Mucopolysaccharidosis IIIA mice","volume":"20","author":"Langford-Smith","year":"2012","journal-title":"Mol. Ther."},{"issue":"3","key":"10.1016\/j.ymgme.2024.108533_bb0215","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."},{"issue":"1","key":"10.1016\/j.ymgme.2024.108533_bb0220","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/brain\/awx311","article-title":"Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy","volume":"141","author":"Holley","year":"2018","journal-title":"Brain"},{"issue":"12","key":"10.1016\/j.ymgme.2024.108533_bb0225","doi-asserted-by":"crossref","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"}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719224004177?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719224004177?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,3,20]],"date-time":"2025-03-20T02:19:46Z","timestamp":1742437186000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719224004177"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,9]]},"references-count":45,"journal-issue":{"issue":"1-2","published-print":{"date-parts":[[2024,9]]}},"alternative-id":["S1096719224004177"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2024.108533","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2024,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Generation of a novel immunodeficient mouse model of Mucopolysaccharidosis type IIIA to test human stem cell-based therapies","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2024.108533","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2024 Published by Elsevier Inc.","name":"copyright","label":"Copyright"}],"article-number":"108533"}}