{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,4,30]],"date-time":"2026-04-30T13:31:43Z","timestamp":1777555903843,"version":"3.51.4"},"reference-count":42,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2015,1,1]],"date-time":"2015-01-01T00:00:00Z","timestamp":1420070400000},"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":645,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/3.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2015]]},"DOI":"10.1038\/mtm.2014.68","type":"journal-article","created":{"date-parts":[[2015,2,10]],"date-time":"2015-02-10T17:28:21Z","timestamp":1423589301000},"page":"14068","source":"Crossref","is-referenced-by-count":18,"special_numbering":"C","title":["A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I"],"prefix":"10.1016","volume":"2","author":[{"given":"Daniel C","family":"Mendez","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Alexander E","family":"Stover","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Anthony D","family":"Rangel","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"David J","family":"Brick","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Hubert E","family":"Nethercott","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marissa A","family":"Torres","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Omar","family":"Khalid","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Andrew MS","family":"Wong","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan D","family":"Cooper","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"James V","family":"Jester","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Edwin S","family":"Monuki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Cian","family":"McGuire","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Steven Q","family":"Le","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shih-hsin","family":"Kan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patricia I","family":"Dickson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Philip H","family":"Schwartz","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1038\/mtm.2014.68_bib1","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1002\/humu.1380060403","article-title":"Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications","volume":"6","author":"Scott","year":"1995","journal-title":"Hum Mutat"},{"key":"10.1038\/mtm.2014.68_bib2","first-page":"250","article-title":"Neuropsychological development in children with Hurler syndrome following hematopoietic stem cell transplantation","volume":"2","author":"Peters","year":"1998","journal-title":"Pediatr Transplant"},{"key":"10.1038\/mtm.2014.68_bib3","doi-asserted-by":"crossref","first-page":"471","DOI":"10.1002\/ajmg.1320200308","article-title":"The clinical spectrum of alpha-L-iduronidase deficiency","volume":"20","author":"Roubicek","year":"1985","journal-title":"Am J Med Genet"},{"key":"10.1038\/mtm.2014.68_bib4","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1007\/s00281-004-0166-2","article-title":"Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases","volume":"26","author":"Krivit","year":"2004","journal-title":"Springer Semin Immunopathol"},{"key":"10.1038\/mtm.2014.68_bib5","doi-asserted-by":"crossref","first-page":"1","DOI":"10.2165\/00063030-200519010-00001","article-title":"Laronidase treatment of mucopolysaccharidosis I","volume":"19","author":"Wraith","year":"2005","journal-title":"BioDrugs"},{"key":"10.1038\/mtm.2014.68_bib6","doi-asserted-by":"crossref","first-page":"356","DOI":"10.1111\/j.1365-2141.2009.07974.x","article-title":"Cord blood and bone marrow transplantation in inherited metabolic diseases: scientific basis, current status and future directions","volume":"148","author":"Prasad","year":"2010","journal-title":"Br J Haematol"},{"key":"10.1038\/mtm.2014.68_bib7","doi-asserted-by":"crossref","first-page":"946","DOI":"10.2174\/138920111795542642","article-title":"Intrathecal enzyme replacement therapy for mucopolysaccharidosis I: translating success in animal models to patients","volume":"12","author":"Dickson","year":"2011","journal-title":"Curr Pharm Biotechnol"},{"key":"10.1038\/mtm.2014.68_bib8","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1016\/j.clim.2009.01.003","article-title":"Optimizing outcomes of hematopoietic stem cell transplantation for severe combined immunodeficiency","volume":"131","author":"Cuvelier","year":"2009","journal-title":"Clin Immunol"},{"key":"10.1038\/mtm.2014.68_bib9","doi-asserted-by":"crossref","first-page":"1526","DOI":"10.1038\/bmt.2010.338","article-title":"Successful reduced-intensity SCT from unrelated cord blood in three patients with X-linked SCID","volume":"46","author":"Iguchi","year":"2011","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mtm.2014.68_bib10","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1111\/j.1399-3046.2011.01606.x","article-title":"A single-center study of hematopoietic stem cell transplantation for primary immune deficiencies (PIDD)","volume":"16","author":"Dinardo","year":"2012","journal-title":"Pediatr Transplant"},{"key":"10.1038\/mtm.2014.68_bib11","doi-asserted-by":"crossref","first-page":"355","DOI":"10.1007\/s12185-013-1408-7","article-title":"B-cell function after unrelated umbilical cord blood transplantation using a minimal-intensity conditioning regimen in patients with X-SCID","volume":"98","author":"Kumaki","year":"2013","journal-title":"Int J Hematol"},{"key":"10.1038\/mtm.2014.68_bib12","doi-asserted-by":"crossref","first-page":"406","DOI":"10.1016\/j.ymgme.2009.07.015","article-title":"Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II","volume":"98","author":"Wang","year":"2009","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mtm.2014.68_bib13","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"},{"key":"10.1038\/mtm.2014.68_bib14","first-page":"25","article-title":"Humanized SCID mouse models for biomedical research","volume":"324","author":"Pearson","year":"2008","journal-title":"Curr Top Microbiol Immunol"},{"key":"10.1038\/mtm.2014.68_bib15","doi-asserted-by":"crossref","first-page":"743","DOI":"10.1038\/nbt.1554","article-title":"Variation in the safety of induced pluripotent stem cell lines","volume":"27","author":"Miura","year":"2009","journal-title":"Nat Biotechnol"},{"key":"10.1038\/mtm.2014.68_bib16","first-page":"Unit 4A.2","article-title":"Generation and characterization of human induced pluripotent stem cells","volume":"Chapter 4","author":"Ohnuki","year":"2009","journal-title":"Curr Protoc Stem Cell Biol"},{"key":"10.1038\/mtm.2014.68_bib17","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1007\/978-1-61779-201-4_17","article-title":"The teratoma assay: an in vivo assessment of pluripotency","volume":"767","author":"Wesselschmidt","year":"2011","journal-title":"Methods Mol Biol"},{"key":"10.1038\/mtm.2014.68_bib18","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1093\/hmg\/6.4.503","article-title":"Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene","volume":"6","author":"Clarke","year":"1997","journal-title":"Hum Mol Genet"},{"key":"10.1038\/mtm.2014.68_bib19","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.ymthe.2004.03.011","article-title":"Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene","volume":"9","author":"Hartung","year":"2004","journal-title":"Mol Ther"},{"key":"10.1038\/mtm.2014.68_bib20","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/S1096-7192(03)00116-1","article-title":"Treatment of the mouse model of mucopolysaccharidosis I with retrovirally transduced bone marrow","volume":"79","author":"Zheng","year":"2003","journal-title":"Mol Genet Metab"},{"key":"10.1038\/mtm.2014.68_bib21","doi-asserted-by":"crossref","first-page":"329","DOI":"10.1620\/tjem.132.329","article-title":"An altered hexosaminidase A in the liver affected by Hurler and Hunter syndromes","volume":"132","author":"Minami","year":"1980","journal-title":"Tohoku J Exp Med"},{"key":"10.1038\/mtm.2014.68_bib22","doi-asserted-by":"crossref","first-page":"88","DOI":"10.2174\/157488808784223041","article-title":"Stem cell therapies for the lysosomal storage diseases - the quintessential neurodegenerative diseases","volume":"3","author":"Schwartz","year":"2008","journal-title":"Curr Stem Cell Res Ther"},{"key":"10.1038\/mtm.2014.68_bib23","doi-asserted-by":"crossref","first-page":"153","DOI":"10.1586\/14737175.6.2.153","article-title":"The potential of stem cell therapies for neurological diseases","volume":"6","author":"Schwartz","year":"2006","journal-title":"Expert Rev Neurother"},{"key":"10.1038\/mtm.2014.68_bib24","doi-asserted-by":"crossref","first-page":"290","DOI":"10.1097\/00004703-200608000-00002","article-title":"Long-term outcomes of adaptive functions for children with mucopolysaccharidosis I (Hurler syndrome) treated with hematopoietic stem cell transplantation","volume":"27","author":"Bjoraker","year":"2006","journal-title":"J Dev Behav Pediatr"},{"key":"10.1038\/mtm.2014.68_bib25","doi-asserted-by":"crossref","first-page":"1005","DOI":"10.1038\/bmt.2008.20","article-title":"Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation","volume":"41","author":"Polgreen","year":"2008","journal-title":"Bone Marrow Transplant"},{"key":"10.1038\/mtm.2014.68_bib26","doi-asserted-by":"crossref","first-page":"997","DOI":"10.3181\/0802-RM-70","article-title":"Expanded CD34+ human umbilical cord blood cells generate multiple lymphohematopoietic lineages in NOD-scid IL2rgamma(null) mice","volume":"233","author":"Giassi","year":"2008","journal-title":"Exp Biol Med (Maywood)"},{"key":"10.1038\/mtm.2014.68_bib27","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1111\/j.1399-0004.1998.tb02745.x","article-title":"Murine MPS I: insights into the pathogenesis of Hurler syndrome","volume":"53","author":"Russell","year":"1998","journal-title":"Clin Genet"},{"key":"10.1038\/mtm.2014.68_bib28","doi-asserted-by":"crossref","first-page":"225","DOI":"10.1016\/j.brainres.2006.01.008","article-title":"Long-term memory for aversive training is impaired in Idua(-\/-) mice, a genetic model of mucopolysaccharidosis type I","volume":"1076","author":"Reolon","year":"2006","journal-title":"Brain Res"},{"key":"10.1038\/mtm.2014.68_bib29","doi-asserted-by":"crossref","first-page":"433","DOI":"10.1007\/978-1-61779-201-4_32","article-title":"In vivo evaluation of putative hematopoietic stem cells derived from human pluripotent stem cells","volume":"767","author":"Hexum","year":"2011","journal-title":"Methods Mol Biol"},{"key":"10.1038\/mtm.2014.68_bib30","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1038\/nm1548","article-title":"Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease","volume":"13","author":"Lee","year":"2007","journal-title":"Nat Med"},{"key":"10.1038\/mtm.2014.68_bib31","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1097\/GIM.0b013e3182174703","article-title":"The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease","volume":"13","author":"Banugaria","year":"2011","journal-title":"Genet Med"},{"key":"10.1038\/mtm.2014.68_bib32","doi-asserted-by":"crossref","first-page":"764","DOI":"10.1523\/JNEUROSCI.5052-10.2011","article-title":"HDAC3 is a critical negative regulator of long-term memory formation","volume":"31","author":"McQuown","year":"2011","journal-title":"J Neurosci"},{"key":"10.1038\/mtm.2014.68_bib33","doi-asserted-by":"crossref","first-page":"552","DOI":"10.1038\/nn.3359","article-title":"The neuron-specific chromatin regulatory subunit BAF53b is necessary for synaptic plasticity and memory","volume":"16","author":"Vogel-Ciernia","year":"2013","journal-title":"Nat Neurosci"},{"key":"10.1038\/mtm.2014.68_bib34","doi-asserted-by":"crossref","first-page":"156","DOI":"10.1006\/bmme.1996.0044","article-title":"Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I","volume":"58","author":"Kakkis","year":"1996","journal-title":"Biochem Mol Med"},{"key":"10.1038\/mtm.2014.68_bib35","first-page":"159","article-title":"Quantitation of proteoglycans in biological fluids using Alcian blue","volume":"171","author":"Karlsson","year":"2001","journal-title":"Methods Mol Biol"},{"key":"10.1038\/mtm.2014.68_bib36","first-page":"Unit17.9","article-title":"Direct chemical analysis of glycoconjugates for carbohydrates","volume":"Chapter 17","author":"Manzi","year":"2001","journal-title":"Curr Protoc Mol Biol"},{"key":"10.1038\/mtm.2014.68_bib37","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1016\/j.nbd.2004.02.010","article-title":"Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Bible","year":"2004","journal-title":"Neurobiol Dis"},{"key":"10.1038\/mtm.2014.68_bib38","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1016\/j.nbd.2006.09.001","article-title":"Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis","volume":"25","author":"Kielar","year":"2007","journal-title":"Neurobiol Dis"},{"key":"10.1038\/mtm.2014.68_bib39","doi-asserted-by":"crossref","first-page":"231","DOI":"10.1016\/j.brainres.2004.07.030","article-title":"Late onset neurodegeneration in the Cln3-\/- mouse model of juvenile neuronal ceroid lipofuscinosis is preceded by low level glial activation","volume":"1023","author":"Pontikis","year":"2004","journal-title":"Brain Res"},{"key":"10.1038\/mtm.2014.68_bib40","doi-asserted-by":"crossref","first-page":"1228","DOI":"10.1097\/NEN.0b013e3181ffe742","article-title":"Distinct neuropathologic phenotypes after disrupting the chloride transport proteins ClC-6 or ClC-7\/Ostm1","volume":"69","author":"Pressey","year":"2010","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/mtm.2014.68_bib41","doi-asserted-by":"crossref","first-page":"1247","DOI":"10.1002\/jnr.23245","article-title":"Process-based expansion and neural differentiation of human pluripotent stem cells for transplantation and disease modeling","volume":"91","author":"Stover","year":"2013","journal-title":"J Neurosci Res"},{"key":"10.1038\/mtm.2014.68_bib42","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1002\/jnr.10854","article-title":"Isolation and characterization of neural progenitor cells from post-mortem human cortex","volume":"74","author":"Schwartz","year":"2003","journal-title":"J Neurosci Res"}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050116300122?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050116300122?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,9,25]],"date-time":"2018-09-25T05:25:45Z","timestamp":1537853145000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050116300122"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2015]]},"references-count":42,"alternative-id":["S2329050116300122"],"URL":"https:\/\/doi.org\/10.1038\/mtm.2014.68","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2015]]},"article-number":"14068"}}