{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,3]],"date-time":"2026-10-03T07:41:00Z","timestamp":1791013260604,"version":"4.1.0"},"reference-count":34,"publisher":"Springer Science and Business Media LLC","issue":"21","license":[{"start":{"date-parts":[[2003,9,8]],"date-time":"2003-09-08T00:00:00Z","timestamp":1062979200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.springer.com\/tdm"},{"start":{"date-parts":[[2003,9,8]],"date-time":"2003-09-08T00:00:00Z","timestamp":1062979200000},"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":[[2003,10,1]]},"DOI":"10.1038\/sj.gt.3302081","type":"journal-article","created":{"date-parts":[[2003,9,8]],"date-time":"2003-09-08T09:52:12Z","timestamp":1063014732000},"page":"1841-1849","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":35,"title":["Widespread distribution of \u03b2-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol"],"prefix":"10.1038","volume":"10","author":[{"given":"C","family":"Bourgoin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C","family":"Emiliani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E J","family":"Kremer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A","family":"Gelot","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B","family":"Tancini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R A","family":"Gravel","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C","family":"Drugan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A","family":"Orlacchio","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"L","family":"Poenaru","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C","family":"Caillaud","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2003,9,8]]},"reference":[{"key":"BF3302081_CR1","first-page":"3827","volume-title":"The Metabolic and Molecular Bases of Inherited Disease","author":"RA Gravel","year":"2001","unstructured":"Gravel RA et al. The GM2 gangliosidoses. In: Scriver CR, Beaudet AL, Valle D, Sly WS (eds). The Metabolic and Molecular Bases of Inherited Disease. McGraw Hill: New York, 2001, pp. 3827\u20133876."},{"key":"BF3302081_CR2","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1093\/hmg\/5.1.1","volume":"5","author":"D Phaneuf","year":"1996","unstructured":"Phaneuf D et al. Dramatically different phenotypes in mouse models of human Tay\u2013Sachs and Sandhoff diseases. Hum Mol Genet 1996; 5: 1\u201314.","journal-title":"Hum Mol Genet"},{"key":"BF3302081_CR3","doi-asserted-by":"publisher","first-page":"170","DOI":"10.1038\/ng1095-170","volume":"11","author":"K Sango","year":"1995","unstructured":"Sango K et al. Mouse models of Tay\u2013Sachs and Sandhoff diseases differ in neurologic phenotype and ganglioside metabolism. Nat Genet 1995; 11: 170\u2013176.","journal-title":"Nat Genet"},{"key":"BF3302081_CR4","doi-asserted-by":"publisher","first-page":"224","DOI":"10.1038\/ng0393-224","volume":"3","author":"S Akli","year":"1993","unstructured":"Akli S et al. Transfer of a foreign gene into the brain using adenovirus vectors. Nat Genet 1993; 3: 224\u2013228.","journal-title":"Nat Genet"},{"key":"BF3302081_CR5","doi-asserted-by":"publisher","first-page":"1287","DOI":"10.1111\/j.1460-9568.1993.tb00914.x","volume":"5","author":"C Caillaud","year":"1993","unstructured":"Caillaud C et al. Adenoviral vector as a gene delivery system into cultured rat neuronal and glial cells. Eur J Neurosci 1993; 5: 1287\u20131291.","journal-title":"Eur J Neurosci"},{"key":"BF3302081_CR6","doi-asserted-by":"publisher","first-page":"219","DOI":"10.1038\/ng0393-219","volume":"3","author":"BL Davidson","year":"1993","unstructured":"Davidson BL et al. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat Genet 1993; 3: 219\u2013223.","journal-title":"Nat Genet"},{"key":"BF3302081_CR7","doi-asserted-by":"publisher","first-page":"988","DOI":"10.1126\/science.8382374","volume":"259","author":"G Le Gal La Salle","year":"1993","unstructured":"Le Gal La Salle G et al. An adenovirus vector for gene transfer into neurons and glia in the brain. Science 1993; 259: 988\u2013990.","journal-title":"Science"},{"key":"BF3302081_CR8","doi-asserted-by":"publisher","first-page":"2231","DOI":"10.1089\/hum.1998.9.16-2331","volume":"9","author":"A Ghodsi","year":"1998","unstructured":"Ghodsi A et al. Extensive beta-glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to the brain. Hum Gene Ther 1998; 9: 2231\u20132240.","journal-title":"Hum Gene Ther"},{"key":"BF3302081_CR9","doi-asserted-by":"crossref","first-page":"3424","DOI":"10.1128\/JVI.73.4.3424-3429.1999","volume":"73","author":"C Stein","year":"1999","unstructured":"Stein C, Ghodsi A, Derksen T, Davidson BL . Systemic and central nervous system correction of lysosomal storage in mucopolysaccharidosis type VII mice. J Virol 1999; 73: 3424\u20133429.","journal-title":"J Virol"},{"key":"BF3302081_CR10","doi-asserted-by":"publisher","first-page":"34","DOI":"10.1038\/sj.gt.3301843","volume":"10","author":"RE Haskell","year":"2003","unstructured":"Haskell RE et al. Viral mediated delivery of the late-infantile neuronal ceroid lipofuscinosis gene, TPP-I to the mouse central nervous system. Gene Therapy 2003; 10: 34\u201342.","journal-title":"Gene Therapy"},{"key":"BF3302081_CR11","first-page":"769","volume":"3","author":"S Akli","year":"1996","unstructured":"Akli S et al. Restoration of hexosaminidase A activity in human Tay\u2013Sachs fibroblasts via adenoviral vector-mediated gene transfer. Gene Therapy 1996; 3: 769\u2013774.","journal-title":"Gene Therapy"},{"key":"BF3302081_CR12","doi-asserted-by":"publisher","first-page":"831","DOI":"10.1093\/hmg\/8.5.831","volume":"8","author":"JE Guidotti","year":"1999","unstructured":"Guidotti JE et al. Adenoviral gene therapy of the Tay\u2013Sachs disease in hexosaminidase A-deficient knock-out mice. Hum Mol Genet 1999; 8: 831\u2013838.","journal-title":"Hum Mol Genet"},{"key":"BF3302081_CR13","doi-asserted-by":"publisher","first-page":"225","DOI":"10.1006\/mthe.2001.0246","volume":"3","author":"MY Mastakov","year":"2001","unstructured":"Mastakov MY et al. Combined injection of rAAV with mannitol enhances gene expression in the rat brain. Mol Ther 2001; 3: 225\u2013232.","journal-title":"Mol Ther"},{"key":"BF3302081_CR14","doi-asserted-by":"publisher","first-page":"801","DOI":"10.1042\/bj2440801","volume":"244","author":"T Beccari","year":"1987","unstructured":"Beccari T et al. Intermediate forms of human beta-N-acetylhexosaminidase lack activity towards 4-methylumbelliferyl beta-N-acetylglucosaminide 6-sulphate. Biochem J 1987; 244: 801\u2013804.","journal-title":"Biochem J"},{"key":"BF3302081_CR15","doi-asserted-by":"publisher","first-page":"111","DOI":"10.1042\/bj2670111","volume":"267","author":"C Emiliani","year":"1990","unstructured":"Emiliani C et al. An enzyme with properties similar to those of beta-N-acetylhexosaminidase S is expressed in the promyelocytic cell line HL-60. Biochem J 1990; 267: 111\u2013117.","journal-title":"Biochem J"},{"key":"BF3302081_CR16","doi-asserted-by":"publisher","first-page":"2283","DOI":"10.1096\/fj.01-0321fje","volume":"15","author":"C Soudais","year":"2001","unstructured":"Soudais C, Laplace-Builhe C, Kissa K, Kremer EJ . Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo. FASEB J 2001; 15: 2283\u20132285.","journal-title":"FASEB J"},{"key":"BF3302081_CR17","doi-asserted-by":"publisher","first-page":"9829","DOI":"10.1073\/pnas.92.21.9829","volume":"92","author":"G Nilaver","year":"1995","unstructured":"Nilaver G et al. Delivery of herpesvirus and adenovirus to nude rat intracerebral tumors after osmotic blood\u2013brain barrier disruption. Proc Natl Acad Sci USA 1995; 92: 9829\u20139833.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3302081_CR18","doi-asserted-by":"publisher","first-page":"217","DOI":"10.1023\/A:1007049806660","volume":"20","author":"SI Rapoport","year":"2000","unstructured":"Rapoport SI . Osmotic opening of the blood\u2013brain barrier: principles, mechanism, and therapeutic applications. Cell Mol Neurobiol 2000; 20: 217\u2013230.","journal-title":"Cell Mol Neurobiol"},{"key":"BF3302081_CR19","doi-asserted-by":"publisher","first-page":"1809","DOI":"10.1517\/13543784.10.10.1809","volume":"10","author":"SI Rapoport","year":"2001","unstructured":"Rapoport SI . Advances in osmotic opening of the blood\u2013brain barrier to enhance CNS chemotherapy. Expert Opin Investig Drugs 2001; 10: 1809\u20131818.","journal-title":"Expert Opin Investig Drugs"},{"key":"BF3302081_CR20","doi-asserted-by":"publisher","first-page":"1","DOI":"10.1172\/JCI112262","volume":"77","author":"S Kornfeld","year":"1986","unstructured":"Kornfeld S . Trafficking of lysosomal enzymes in normal and disease states. J Clin Invest 1986; 77: 1\u20136.","journal-title":"J Clin Invest"},{"key":"BF3302081_CR21","doi-asserted-by":"publisher","first-page":"77","DOI":"10.1016\/S0165-0270(96)00128-8","volume":"71","author":"E Peltekian","year":"1997","unstructured":"Peltekian E . Adenovirus-mediated gene transfer to the brain: methodological assessment. J Neurosci Methods 1997; 71: 77\u201384.","journal-title":"J Neurosci Methods"},{"key":"BF3302081_CR22","doi-asserted-by":"publisher","first-page":"36","DOI":"10.1006\/mthe.2000.0224","volume":"3","author":"CE Thomas","year":"2001","unstructured":"Thomas CE et al. Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol Ther 2001; 3: 36\u201346.","journal-title":"Mol Ther"},{"key":"BF3302081_CR23","doi-asserted-by":"publisher","first-page":"93","DOI":"10.1016\/S0006-8993(02)02953-0","volume":"948","author":"NR Bhat","year":"2002","unstructured":"Bhat NR, Fan F . Adenovirus infection induces microglial activation: involvement of mitogen-activated protein kinase pathways. Brain Res 2002; 948: 93\u2013101.","journal-title":"Brain Res"},{"key":"BF3302081_CR24","doi-asserted-by":"publisher","first-page":"2111","DOI":"10.1097\/00001756-199707070-00005","volume":"8","author":"HD Durham","year":"1997","unstructured":"Durham HD et al. The immunosuppressant FK506 prolongs transgene expression in brain following adenovirus-mediated gene transfer. NeuroReport 1997; 8: 2111\u20132115.","journal-title":"NeuroReport"},{"key":"BF3302081_CR25","doi-asserted-by":"publisher","first-page":"839","DOI":"10.1089\/104303401750148829","volume":"12","author":"CE Thomas","year":"2001","unstructured":"Thomas CE et al. Preexisting antiadenoviral immunity is not a barrier to efficient and stable transduction of the brain, mediated by novel high-capacity adenovirus vectors. Hum Gene Ther 2001; 12: 839\u2013846.","journal-title":"Hum Gene Ther"},{"key":"BF3302081_CR26","doi-asserted-by":"publisher","first-page":"63","DOI":"10.1006\/mthe.1999.0005","volume":"1","author":"A Bosch","year":"2000","unstructured":"Bosch A, Perret E, Desmaris N, Heard JM . Long-term and significant correction of brain lesions in adult mucopoly-saccharidosis type VII mice using recombinant AAV vectors. Mol Ther 2000; 1: 63\u201370.","journal-title":"Mol Ther"},{"key":"BF3302081_CR27","doi-asserted-by":"publisher","first-page":"1139","DOI":"10.1089\/10430340050015194","volume":"11","author":"A Bosch","year":"2000","unstructured":"Bosch A et al. Reversal of pathology in the entire brain of mucopolysaccharidosis type VII mice after lentivirus-mediated gene transfer. Hum Gene Ther 2000; 11: 1139\u20131150.","journal-title":"Hum Gene Ther"},{"key":"BF3302081_CR28","doi-asserted-by":"crossref","first-page":"5919","DOI":"10.1128\/JVI.72.7.5919-5926.1998","volume":"72","author":"P Wu","year":"1998","unstructured":"Wu P, Phillips MI, Bui J, Terwilliger EF . Adeno-associated virus vector-mediated transgene integration into neurons and other nondividing cells targets. J Virol 1998; 72: 5919\u20135926.","journal-title":"J Virol"},{"key":"BF3302081_CR29","doi-asserted-by":"publisher","first-page":"5411","DOI":"10.1128\/JVI.76.11.5411-5421.2002","volume":"76","author":"NJ Philpott","year":"2002","unstructured":"Philpott NJ et al. Efficient integration of recombinant adeno-associated virus DNA vector requires a p5-rep sequence in cis. J Virol 2002; 76: 5411\u20135421.","journal-title":"J Virol"},{"key":"BF3302081_CR30","doi-asserted-by":"publisher","first-page":"31","DOI":"10.1093\/oxfordjournals.bmb.a072951","volume":"51","author":"EJ Kremer","year":"1995","unstructured":"Kremer EJ, Perricaudet M . Adenovirus and adeno-associated virus mediated gene transfer. Br Med Bull 1995; 51: 31\u201344.","journal-title":"Br Med Bull"},{"key":"BF3302081_CR31","doi-asserted-by":"publisher","first-page":"4455","DOI":"10.1073\/pnas.90.10.4455","volume":"90","author":"L Cheng","year":"1993","unstructured":"Cheng L, Ziegelhoffer PR, Yang NS . In vivo promoter activity and transgene expression in mammalian somatic tissues evaluated by using particle bombardment. Proc Natl Acad Sci USA 1993; 90: 4455\u20134459.","journal-title":"Proc Natl Acad Sci USA"},{"key":"BF3302081_CR32","volume-title":"The Mouse Brain in Stereotaxic Coordinates","author":"KBJ Franklin","year":"1997","unstructured":"Franklin KBJ, Paxinos G . The Mouse Brain in Stereotaxic Coordinates. Academic Press: San Diego, 1997."},{"key":"BF3302081_CR33","doi-asserted-by":"publisher","first-page":"73","DOI":"10.1016\/0009-8981(84)90215-8","volume":"143","author":"J Bayleran","year":"1984","unstructured":"Bayleran J, Hechtman P, Saray W . Synthesis of 4-methyl-umbelliferyl-beta-D-N-acetylglucosamine-6-sulfate and its use in classification of GM2 gangliosidosis genotypes. Clin Chim Acta 1984; 143: 73\u201389.","journal-title":"Clin Chim Acta"},{"key":"BF3302081_CR34","first-page":"434","volume":"19","author":"HD Lacorazza","year":"1995","unstructured":"Lacorazza HD, Jendoubi M . In situ assessment of beta-hexosaminidase activity. Biotechniques 1995; 19: 434\u2013440.","journal-title":"Biotechniques"}],"container-title":["Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/www.nature.com\/articles\/3302081.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3302081","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/www.nature.com\/articles\/3302081.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2022,3,30]],"date-time":"2022-03-30T02:42:58Z","timestamp":1648608178000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.nature.com\/articles\/3302081"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2003,9,8]]},"references-count":34,"journal-issue":{"issue":"21","published-print":{"date-parts":[[2003,10,1]]}},"alternative-id":["BF3302081"],"URL":"https:\/\/doi.org\/10.1038\/sj.gt.3302081","relation":{},"ISSN":["0969-7128","1476-5462"],"issn-type":[{"value":"0969-7128","type":"print"},{"value":"1476-5462","type":"electronic"}],"subject":[],"published":{"date-parts":[[2003,9,8]]},"assertion":[{"value":"7 March 2003","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"14 April 2003","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"8 September 2003","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}}]}}