{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,20]],"date-time":"2026-05-20T06:30:10Z","timestamp":1779258610453,"version":"3.51.4"},"reference-count":30,"publisher":"Springer Science and Business Media LLC","issue":"17","license":[{"start":{"date-parts":[[2003,8,1]],"date-time":"2003-08-01T00:00:00Z","timestamp":1059696000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2003,8,1]],"date-time":"2003-08-01T00:00:00Z","timestamp":1059696000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Gene Ther"],"published-print":{"date-parts":[[2003,8,1]]},"DOI":"10.1038\/sj.gt.3302033","type":"journal-article","created":{"date-parts":[[2003,8,4]],"date-time":"2003-08-04T15:15:27Z","timestamp":1060010127000},"page":"1487-1493","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":34,"title":["Attenuation of ganglioside GM1 accumulation in the brain of GM1 gangliosidosis mice by neonatal intravenous gene transfer"],"prefix":"10.1038","volume":"10","author":[{"given":"N","family":"Takaura","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T","family":"Yagi","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"M","family":"Maeda","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"E","family":"Nanba","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A","family":"Oshima","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Y","family":"Suzuki","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"T","family":"Yamano","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"A","family":"Tanaka","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"297","published-online":{"date-parts":[[2003,8,4]]},"reference":[{"key":"BF3302033_CR1","doi-asserted-by":"crossref","first-page":"3081","DOI":"10.1182\/blood.V78.11.3081.3081","volume":"78","author":"EH Birkenmeier","year":"1991","unstructured":"Birkenmeier EH et al. Increased life span and correction of metabolic defects in murine MPSVII after syngeneic bone marrow transplantation. Blood 1991; 78: 3081\u20133092.","journal-title":"Blood"},{"key":"BF3302033_CR2","doi-asserted-by":"publisher","first-page":"1287","DOI":"10.1073\/pnas.94.4.1287","volume":"94","author":"T Ohashi","year":"1997","unstructured":"Ohashi T et al. Adenovirus-mediated gene transfer and expression of human \u03b2-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type VII mice. Proc Natl Acad Sci USA 1997; 94: 1287\u20131292.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3302033_CR3","doi-asserted-by":"publisher","first-page":"462","DOI":"10.1096\/fasebj.1.6.3315809","volume":"1","author":"S Konfeld","year":"1987","unstructured":"Konfeld S . Trafficking of lysosomal enzyme. FASEB J 1987; 1: 462\u2013468.","journal-title":"FASEB J"},{"key":"BF3302033_CR4","doi-asserted-by":"publisher","first-page":"1596","DOI":"10.1172\/JCI119322","volume":"99","author":"MS Sands","year":"1997","unstructured":"Sands MS et al. Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation. J Clin Invest 1997; 99: 1596\u20131605.","journal-title":"J Clin Invest"},{"key":"BF3302033_CR5","doi-asserted-by":"publisher","first-page":"182","DOI":"10.1056\/NEJM200101183440304","volume":"344","author":"ED Kakkis","year":"2001","unstructured":"Kakkis ED, Muenzer J, Tiller GE, Waber L . Enzyme replacement therapy in mucopolysacharidosis I. N Engl J Med 2001; 344: 182\u2013188.","journal-title":"N Engl J Med"},{"key":"BF3302033_CR6","doi-asserted-by":"publisher","first-page":"1398","DOI":"10.1016\/S0140-6736(95)92597-X","volume":"345","author":"PM Hoogerbrugge","year":"1995","unstructured":"Hoogerbrugge PM et al. Allogenic bone marrow transplantation for lysosomal storage diseases. Lancet 1995; 345: 1398\u20131402.","journal-title":"Lancet"},{"key":"BF3302033_CR7","first-page":"302","volume":"58","author":"K Suzuki","year":"1998","unstructured":"Suzuki K et al. The Twitcher mouse: central nervous system pathology after bone marrow transplantation. Lab Invest 1998; 58: 302\u2013309.","journal-title":"Lab Invest"},{"key":"BF3302033_CR8","doi-asserted-by":"publisher","first-page":"1250","DOI":"10.1038\/sj.gt.3301232","volume":"7","author":"U Matzner","year":"2000","unstructured":"Matzner U et al. Long-term expression and transfer of arylsulfatase A into brain of arylsulfatase A-deficient mice transplanted with bone marrow expressing the arylsulfatase A cDNA from a retroviral vector. Gene Therapy 2000; 7: 1250\u20131257.","journal-title":"Gene Therapy"},{"key":"BF3302033_CR9","doi-asserted-by":"crossref","first-page":"408","DOI":"10.1182\/blood.V82.2.408.408","volume":"82","author":"GM Pastores","year":"1993","unstructured":"Pastores GM, Sibille AR, Grabowski GA . Enzyme therapy in Gaucher disease type I: dosage efficacy and adverse and effects in 33 patients treated for 6 to 24 months. Blood 1993; 82: 408\u2013416.","journal-title":"Blood"},{"key":"BF3302033_CR10","doi-asserted-by":"publisher","first-page":"1","DOI":"10.5414\/CNP57001","volume":"57","author":"RJ Desnick","year":"2002","unstructured":"Desnick RJ, Banikazemi M, Wasserstein M . Enzyme replacement therapy for Fabry disease, an inherited nephropathy. Clin Nephrol 2002; 57: 1\u20138.","journal-title":"Clin Nephrol"},{"key":"BF3302033_CR11","doi-asserted-by":"publisher","first-page":"30","DOI":"10.1111\/j.1442-200X.1994.tb03125.x","volume":"36","author":"M Imaizumi","year":"1994","unstructured":"Imaizumi M et al. Long term effects of bone marrow transplantation for inborn errors of metabolism: a study of four patients with lysosomal storage disease. Acta Pediat Jpn 1994; 36: 30\u201336.","journal-title":"Acta Pediat Jpn"},{"key":"BF3302033_CR12","doi-asserted-by":"publisher","first-page":"1175","DOI":"10.1038\/sj.gt.3300931","volume":"6","author":"SS Elliger","year":"1999","unstructured":"Elliger SS et al. Elimination of lysosomal storage in brains of MPS VII mice treated by intrathecal administration of an adeno-associated virus vector. Gene Therapy 1999; 6: 1175\u20131178.","journal-title":"Gene Therapy"},{"key":"BF3302033_CR13","doi-asserted-by":"publisher","first-page":"351","DOI":"10.1006\/mthe.2001.0274","volume":"3","author":"WA Frisella","year":"2001","unstructured":"Frisella WA et al. Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII. Mol Ther 2001; 3: 351\u2013358.","journal-title":"Mol Ther"},{"key":"BF3302033_CR14","doi-asserted-by":"publisher","first-page":"1081","DOI":"10.1038\/sj.gt.3301495","volume":"8","author":"JS Shen","year":"2001","unstructured":"Shen JS, Watabe K, Ohashi T, Eto Y . Intraventricular administration of recombinant adenovirus to neonatal Twitcher mouse leads to clinicopatholgical improvements. Gene Therapy 2001; 8: 1081\u20131087.","journal-title":"Gene Therapy"},{"key":"BF3302033_CR15","doi-asserted-by":"crossref","first-page":"2033","DOI":"10.1182\/blood.V86.5.2033.bloodjournal8652033","volume":"86","author":"MS Sands","year":"1995","unstructured":"Sands MS et al. Syngenic bone marrow transplantation reduces hearing loss associated with murine mucopolysaccharidosis type VII. Blood 1995; 86: 2033\u20132040.","journal-title":"Blood"},{"key":"BF3302033_CR16","doi-asserted-by":"publisher","first-page":"1394","DOI":"10.1172\/JCI1773","volume":"101","author":"LH O'Connor","year":"1998","unstructured":"O'Connor LH et al. Enzyme replacement therapy for murine mucopolysaccharidosis type VII leads to improvements in behavior and auditory function. J Clin Invest 1998; 101: 1394\u20131400.","journal-title":"J Clin Invest"},{"key":"BF3302033_CR17","doi-asserted-by":"publisher","first-page":"838","DOI":"10.1203\/00006450-199906000-00010","volume":"45","author":"C Volger","year":"1999","unstructured":"Volger C et al. Enzyme replacement therapy in murine mucopolysaccharidosis type VII: neuronal and glial response to \u03b2-glucuronidase requires early inhibition of enzyme replacement therapy. Ped Res 1999; 45: 838\u2013844.","journal-title":"Ped Res"},{"key":"BF3302033_CR18","doi-asserted-by":"publisher","first-page":"11288","DOI":"10.1073\/pnas.96.22.12760","volume":"96","author":"TM Day","year":"1999","unstructured":"Day TM et al. Neonatal gene transfer leads to widespread correction of pathology in a murine model of lysosomal storage disease. Proc Natl Acad Sci USA 1999; 96: 11288\u201311294.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3302033_CR19","doi-asserted-by":"publisher","first-page":"1291","DOI":"10.1038\/sj.gt.3301420","volume":"8","author":"TM Day","year":"2001","unstructured":"Day TM et al. Prevention of systemic clinical disease in MPS VII mice following AAV-mediated neonatal gene transfer. Gene Therapy 2001; 8: 1291\u20131298.","journal-title":"Gene Therapy"},{"key":"BF3302033_CR20","first-page":"3775","volume-title":"The Metabolic and Molecular Bases of Inherited Disease","author":"Y Suzuki","year":"2001","unstructured":"Suzuki Y, Oshima A, Nanba E . \u03b2-Galactosidase deficiency (\u03b2-galactosidosis): GM1 gangliosidosis and Morquio B disease. In: Scriver CR, Beaudet AL, Sly WS, Valle D (eds) The Metabolic and Molecular Bases of Inherited Disease, 8th edn. McGraw-Hill: New York, 2001, pp 3775\u20133803."},{"key":"BF3302033_CR21","doi-asserted-by":"publisher","first-page":"271","DOI":"10.1016\/0165-3806(87)90107-6","volume":"32","author":"PA Stewart","year":"1987","unstructured":"Stewart PA, Hayakawa EM . Interendothelial junctional change underlie the developmental \u2018tightening\u2019 of the blood\u2013brain barrier. Dev Brain Res 1987; 32: 271\u2013281.","journal-title":"Dev Brain Res"},{"key":"BF3302033_CR22","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1016\/S0387-7604(96)00077-0","volume":"19","author":"J Matsuda","year":"1997","unstructured":"Matsuda J et al. Neurological manifestations of knockout mice with \u03b2-galactosidase deficiency. Brain Dev 1997; 19: 19\u201320.","journal-title":"Brain Dev"},{"key":"BF3302033_CR23","doi-asserted-by":"publisher","first-page":"729","DOI":"10.1023\/A:1018573518127","volume":"14","author":"J Matsuda","year":"1997","unstructured":"Matsuda J et al. \u03b2-Galactosidase-deficient mouse as an animal model for GM1-gangliosidosis. Glycoconjugate J 1997; 14: 729\u2013736.","journal-title":"Glycoconjugate J"},{"key":"BF3302033_CR24","doi-asserted-by":"publisher","first-page":"2509","DOI":"10.1073\/pnas.95.5.2509","volume":"95","author":"TC He","year":"1998","unstructured":"He TC et al. A simplified system for generating recombinant adenoviruses. Proc Natl Acad Sci USA 1998; 95: 2509\u20132514.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3302033_CR25","doi-asserted-by":"publisher","first-page":"141","DOI":"10.1016\/S0006-291X(05)81033-2","volume":"173","author":"E Nanba","year":"1990","unstructured":"Nanba E, Suzuki K . Molecular cloning of mouse acid \u03b2-galactosidase cDNA: sequence, expression of catalytic activity and comparison with human enzyme. Biochem Biophys Res Comm 1990; 173: 141\u2013148.","journal-title":"Biochem Biophys Res Comm"},{"key":"BF3302033_CR26","doi-asserted-by":"publisher","first-page":"727","DOI":"10.1016\/0076-6879(87)38063-2","volume":"138","author":"K Suzuki","year":"1987","unstructured":"Suzuki K . Enzymatic diagnosis of sphingolipidosis. Methods Enzymol 1987; 138: 727\u2013762.","journal-title":"Methods Enzymol"},{"key":"BF3302033_CR27","doi-asserted-by":"publisher","first-page":"255","DOI":"10.1016\/0003-2697(76)90527-3","volume":"72","author":"MM Bradford","year":"1976","unstructured":"Bradford MM . A rapid and sensitive method for the quantitation of microgram quantities of protein utilizing the principle of protein-dye binding. Anal Biochem 1976; 72: 255\u2013260.","journal-title":"Anal Biochem"},{"key":"BF3302033_CR28","doi-asserted-by":"publisher","first-page":"711","DOI":"10.1093\/hmg\/5.6.711","volume":"5","author":"N Fujita","year":"1996","unstructured":"Fujita N et al. Targeted disruption of the mouse sphingolipid activator protein gene: a complex phenotype, including severe leukodystrophy and wide-spread storage of multiple sphingolipids. Hum Mol Genet 1996; 5: 711\u2013725.","journal-title":"Hum Mol Genet"},{"key":"BF3302033_CR29","doi-asserted-by":"publisher","first-page":"205","DOI":"10.1093\/hmg\/6.2.205","volume":"6","author":"CN Hahn","year":"1997","unstructured":"Hahn CN et al. Generalized CNS disease and massive GM1-ganglioside accumulation in mice defective in lysosomal acid \u03b2-galactosidase. Hum Mol Genet 1997; 6: 205\u2013211.","journal-title":"Hum Mol Genet"},{"key":"BF3302033_CR30","doi-asserted-by":"publisher","first-page":"604","DOI":"10.1016\/0006-3002(57)90254-8","volume":"24","author":"L Svennerholm","year":"1957","unstructured":"Svennerholm L . Estimation of sialic acids. II. A colorimetric resorcinol-hydrochloric acid method. Biochem Biophys Acta 1957; 24: 604\u2013611.","journal-title":"Biochem Biophys Acta"}],"container-title":["Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/3302033.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3302033","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3302033.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,3,30]],"date-time":"2022-03-30T02:36:16Z","timestamp":1648607776000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/3302033"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2003,8,1]]},"references-count":30,"journal-issue":{"issue":"17","published-print":{"date-parts":[[2003,8,1]]}},"alternative-id":["BF3302033"],"URL":"https:\/\/doi.org\/10.1038\/sj.gt.3302033","relation":{},"ISSN":["0969-7128","1476-5462"],"issn-type":[{"value":"0969-7128","type":"print"},{"value":"1476-5462","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,8,1]]},"assertion":[{"value":"22 November 2002","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"18 February 2003","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 August 2003","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}}]}}