{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,11]],"date-time":"2026-03-11T23:48:27Z","timestamp":1773272907939,"version":"3.50.1"},"reference-count":38,"publisher":"Elsevier BV","issue":"6","license":[{"start":{"date-parts":[[2007,11,1]],"date-time":"2007-11-01T00:00:00Z","timestamp":1193875200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2007,11,1]],"date-time":"2007-11-01T00:00:00Z","timestamp":1193875200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"}],"content-domain":{"domain":["clinicalkey.com","clinicalkey.com.au","clinicalkey.es","clinicalkey.fr","clinicalkey.jp","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Brain Research Bulletin"],"published-print":{"date-parts":[[2007,11]]},"DOI":"10.1016\/j.brainresbull.2007.07.018","type":"journal-article","created":{"date-parts":[[2007,8,7]],"date-time":"2007-08-07T08:34:11Z","timestamp":1186475651000},"page":"429-438","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":33,"title":["Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy"],"prefix":"10.1016","volume":"74","author":[{"given":"Mayra F.","family":"Garcia-Rivera","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Leah E.","family":"Colvin-Wanshura","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Matthew S.","family":"Nelson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zhenhong","family":"Nan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shaukat A.","family":"Khan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tyson B.","family":"Rogers","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Indrani","family":"Maitra","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Walter C.","family":"Low","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Pankaj","family":"Gupta","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.brainresbull.2007.07.018_bib1","doi-asserted-by":"crossref","first-page":"1038","DOI":"10.1038\/2023","article-title":"A newly discovered class of human hematopoietic cells with SCID-repopulating activity","volume":"4","author":"Bhatia","year":"1998","journal-title":"Nat. Med."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib2","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1016\/0003-2697(76)90527-3","article-title":"A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding","volume":"72","author":"Bradford","year":"1976","journal-title":"Anal. Biochem."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib3","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1203\/01.pdr.0000190579.24054.39","article-title":"Cardiac functional and histopathologic findings in humans and mice with mucopolysaccharidosis type I: implications for assessment of therapeutic interventions in hurler syndrome","volume":"59","author":"Braunlin","year":"2006","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib4","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/0003-2697(87)90658-0","article-title":"Microdetermination of proteoglycans and glycosaminglycans in the presence of guanidine hydrochloride","volume":"161","author":"Chandrasekhar","year":"1987","journal-title":"Anal. Biochem."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib5","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":"10.1016\/j.brainresbull.2007.07.018_bib6","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1089\/hum.2005.16.81","article-title":"Gene Therapy for a mucopolysaccharidosis type I murine model with lentiviral-IDUA vector","volume":"16","author":"Di Domenico","year":"2005","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib7","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1002\/stem.5530150826","article-title":"Assay of human stem cells by repopulation of NOD\/SCID mice","volume":"15","author":"Dick","year":"1997","journal-title":"Stem Cells"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib8","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1111\/j.1749-6632.2001.tb03588.x","article-title":"In vivo dynamics of human stem cell repopulation in NOD\/SCID mice","volume":"938","author":"Dick","year":"2001","journal-title":"Ann. N. Y. Acad. Sci."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib9","doi-asserted-by":"crossref","first-page":"365","DOI":"10.1016\/j.neubiorev.2004.06.002","article-title":"Motor and cognitive function evaluation following experimental traumatic brain injury","volume":"28","author":"Fujimoto","year":"2004","journal-title":"Neurosci. Biobehav. Rev."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib10","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/S1096-7192(02)00148-8","article-title":"Immune response to enzyme replacement therapy: single epitope control of antigen distribution from circulation","volume":"77","author":"Glaros","year":"2002","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib11","doi-asserted-by":"crossref","first-page":"392","DOI":"10.1097\/00001665-200405000-00009","article-title":"Craniofacial abnormalities in a murine knock-out model of mucopolysaccharidosis I H: a computed tomography and anatomic study","volume":"15","author":"Graupman","year":"2004","journal-title":"J. Craniofac. Surg."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib12","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":"10.1016\/j.brainresbull.2007.07.018_bib13","first-page":"27","article-title":"The pathology of the feline model of mucopolysaccharidosis I","volume":"112","author":"Haskins","year":"1983","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib14","doi-asserted-by":"crossref","first-page":"1294","DOI":"10.1203\/00006450-197911000-00018","article-title":"Alpha-L-iduronidase deficiency in a cat: a model of mucopolysaccharidosis I","volume":"13","author":"Haskins","year":"1979","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib15","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1182\/blood.V90.1.85.85_85_96","article-title":"Engraftment and development of human CD34+ enriched cells from umbilicl cord blood in NOD\/LtSz-scid\/scid mice","volume":"90","author":"Hogan","year":"1997","journal-title":"Blood"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib16","doi-asserted-by":"crossref","first-page":"233","DOI":"10.1016\/j.ymgme.2005.05.003","article-title":"Cardiac manifestations in the mouse model of mucopolysaccharidosis I","volume":"86","author":"Jordan","year":"2005","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib17","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1006\/mgme.2000.3140","article-title":"Enzyme replacement therapy in feline mucopolysaccharidosis I","volume":"72","author":"Kakkis","year":"2001","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib18","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1016\/S0304-3940(01)02384-9","article-title":"Intracerebral transplantation of bone marrow stromal cells in a 1- methyl-4-phenyl-1,2,3,6-tetrahydropyridine mouse model of Parkinson's disease","volume":"316","author":"Li","year":"2001","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib19","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1016\/j.ymthe.2004.08.027","article-title":"Liver-directed neonatal gene therapy prevents cardiac, bone, ear, and eye disease in mucopolysaccharidosis I mice","volume":"11","author":"Liu","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib20","first-page":"1895","article-title":"Genetically corrected autologous stem cells engraft, but host immune responses limit their utility in canine alpha-L-iduronidase deficiency","volume":"93","author":"Lutzko","year":"1999","journal-title":"Blood"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib21","series-title":"Late infantile Progressive Genetic Encephalopathies. Neurology of Hereditary Metabolic Diseases of Children","author":"Lyon","year":"1996"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib22","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1038\/sj.mt.6300112","article-title":"Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation","volume":"15","author":"Ma","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib23","doi-asserted-by":"crossref","first-page":"1259","DOI":"10.1182\/blood-2005-03-1081","article-title":"Human short-term repopulating stem cells are efficiently detected following intrafemoral transplant into NOD\/SCID recipients depleted of CD122+ cells","volume":"106","author":"McKenzie","year":"2005","journal-title":"Blood"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib24","doi-asserted-by":"crossref","first-page":"1809","DOI":"10.1089\/104303402760372918","article-title":"In utero injection of alpha-L-iduronidase carrying retrovirus in canine mucopolysaccharidosis type I: infection of multiple tissues and neonatal gene expression","volume":"13","author":"Meertens","year":"2002","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib25","first-page":"3421","article-title":"The mucopolysaccharidoses","volume":"vol. III","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib26","doi-asserted-by":"crossref","first-page":"3817","DOI":"10.4049\/jimmunol.150.9.3817","article-title":"Strain-dependent leakiness of mice with severe combined immune deficiency","volume":"150","author":"Nonoyama","year":"1993","journal-title":"J. Immunol."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib27","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. U.S.A."},{"key":"10.1016\/j.brainresbull.2007.07.018_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(\u2212\/\u2212) mice, a genetic model of mucopolysaccharidosis type I","volume":"1076","author":"Reolon","year":"2006","journal-title":"Brain Res."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib29","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":"10.1016\/j.brainresbull.2007.07.018_bib30","doi-asserted-by":"crossref","first-page":"3132","DOI":"10.1016\/S0021-9258(17)43271-6","article-title":"Human alpha-L-iduronidase. I. Purification and properties of the high uptake (higher molecular weight) and the low uptake (processed) forms","volume":"259","author":"Schuchman","year":"1984","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib31","first-page":"487","article-title":"Morphologic and biochemical studies of canine mucopolysaccharidosis I","volume":"114","author":"Shull","year":"1984","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib32","doi-asserted-by":"crossref","first-page":"377","DOI":"10.1182\/blood.V88.1.377b.377b","article-title":"Humoral immune response limits gene therapy in canine MPS I","volume":"88","author":"Shull","year":"1996","journal-title":"Blood"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib33","first-page":"244","article-title":"Canine alpha-L-iduronidase deficiency. A model of mucopolysaccharidosis I","volume":"109","author":"Shull","year":"1982","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib34","doi-asserted-by":"crossref","first-page":"180","DOI":"10.4049\/jimmunol.154.1.180","article-title":"Multiple defects in innate and adaptive immunologic function in NOD\/LtSz-scid mice","volume":"154","author":"Shultz","year":"1995","journal-title":"J. Immunol."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib35","doi-asserted-by":"crossref","first-page":"6091","DOI":"10.1073\/pnas.80.19.6091","article-title":"A canine model of human alpha-L-iduronidase deficiency","volume":"80","author":"Spellacy","year":"1983","journal-title":"Proc. Natl. Acad. Sci. U.S.A."},{"key":"10.1016\/j.brainresbull.2007.07.018_bib36","doi-asserted-by":"crossref","first-page":"2406","DOI":"10.1182\/blood.V91.7.2406","article-title":"High level engraftment of NOD\/SCID mice by primitive normal and leukemic hematopoietic cells from patients with chronic myeloid leukemia in chronic phase","volume":"91","author":"Wang","year":"1998","journal-title":"Blood"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib37","first-page":"281","article-title":"The mucopolysaccharidoses","volume":"vol. 66 (22)","author":"Whitley","year":"1996"},{"key":"10.1016\/j.brainresbull.2007.07.018_bib38","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."}],"updated-by":[{"DOI":"10.1016\/j.brainresbull.2008.05.002","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2008,8,15]],"date-time":"2008-08-15T00:00:00Z","timestamp":1218758400000}}],"container-title":["Brain Research Bulletin"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0361923007002225?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0361923007002225?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,6]],"date-time":"2025-10-06T19:59:59Z","timestamp":1759780799000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0361923007002225"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2007,11]]},"references-count":38,"journal-issue":{"issue":"6","published-print":{"date-parts":[[2007,11]]}},"alternative-id":["S0361923007002225"],"URL":"https:\/\/doi.org\/10.1016\/j.brainresbull.2007.07.018","relation":{},"ISSN":["0361-9230"],"issn-type":[{"value":"0361-9230","type":"print"}],"subject":[],"published":{"date-parts":[[2007,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Characterization of an immunodeficient mouse model of mucopolysaccharidosis type I suitable for preclinical testing of human stem cell and gene therapy","name":"articletitle","label":"Article Title"},{"value":"Brain Research Bulletin","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.brainresbull.2007.07.018","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2007 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}