{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,29]],"date-time":"2026-09-29T06:52:10Z","timestamp":1790664730084,"version":"4.1.0"},"reference-count":37,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[1999,11,1]],"date-time":"1999-11-01T00:00:00Z","timestamp":941414400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[1999,11,1]],"date-time":"1999-11-01T00:00:00Z","timestamp":941414400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2003,5,25]],"date-time":"2003-05-25T00:00:00Z","timestamp":1053820800000},"content-version":"vor","delay-in-days":1301,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Experimental Neurology"],"published-print":{"date-parts":[[1999,11]]},"DOI":"10.1006\/exnr.1999.7176","type":"journal-article","created":{"date-parts":[[2002,10,6]],"date-time":"2002-10-06T19:00:46Z","timestamp":1033930846000},"page":"17-27","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":116,"title":["Sustained Production of \u03b2-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice"],"prefix":"10.1006","volume":"160","author":[{"given":"Annette F.","family":"Skorupa","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Krisna J.","family":"Fisher","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James M.","family":"Wilson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael K.","family":"Parente","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John H.","family":"Wolfe","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1006\/exnr.1999.7176_RF1","series-title":"The Metabolic and Molecular Basis of Inherited Disease","author":"Schriver","year":"1990"},{"key":"10.1006\/exnr.1999.7176_RF2","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1097\/00019052-199604000-00013","article-title":"CNS cell transplantation: a novel therapy for storage diseases","volume":"9","author":"Snyder","year":"1996","journal-title":"Curr. Opin. Neurol."},{"key":"10.1006\/exnr.1999.7176_RF3","doi-asserted-by":"crossref","first-page":"1575","DOI":"10.1126\/science.3201248","article-title":"Grafting genetically modified cells to the damaged brain: Restorative effects of NGF expression","volume":"242","author":"Rosenberg","year":"1988","journal-title":"Science"},{"key":"10.1006\/exnr.1999.7176_RF4","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1038\/ng1094-148","article-title":"Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain","volume":"8","author":"Kaplitt","year":"1994","journal-title":"Nature Genet."},{"key":"10.1006\/exnr.1999.7176_RF5","doi-asserted-by":"crossref","first-page":"838","DOI":"10.1126\/science.275.5301.838","article-title":"Dopaminergic neurons protected from degeneration by GDNF gene therapy","volume":"275","author":"Choi-Lundberg","year":"1997","journal-title":"Science"},{"key":"10.1006\/exnr.1999.7176_RF6","doi-asserted-by":"crossref","first-page":"820","DOI":"10.1038\/sj.gt.3300650","article-title":"In vivo expression of therapeutic human genes for dopamine production in the caudates of MPTP-treated monkeys using an AAV vector","volume":"5","author":"During","year":"1998","journal-title":"Gene Ther."},{"key":"10.1006\/exnr.1999.7176_RF7","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1038\/nm0797-771","article-title":"Decreased lysosomal storage in the adult MPS VII mouse brain in the vicinity of grafts of retroviral vector-corrected fibroblasts secreting high levels of \u03b2-glucuronidase","volume":"3","author":"Taylor","year":"1997","journal-title":"Nature Med."},{"key":"10.1006\/exnr.1999.7176_RF8","doi-asserted-by":"crossref","first-page":"2331","DOI":"10.1089\/hum.1998.9.16-2331","article-title":"Extensive \u03b2-Glucuronidase activity in murine central nervous system after adenovirus-mediated gene transfer to brain","volume":"9","author":"Ghodsi","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.1999.7176_RF9","series-title":"Protocols for Gene Transfer in Neuroscience: Towards Gene Therapy of Neurologic Disorders","first-page":"263","article-title":"Murine mucopolysaccharidosis type VII: A model system for somatic gene therapy of the central nervous system","author":"Wolfe","year":"1996"},{"key":"10.1006\/exnr.1999.7176_RF10","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/S0022-3476(73)80162-3","article-title":"Beta-glucuronidase deficiency: Report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis","volume":"82","author":"Sly","year":"1973","journal-title":"J. Pediatr."},{"key":"10.1006\/exnr.1999.7176_RF11","doi-asserted-by":"crossref","first-page":"1258","DOI":"10.1172\/JCI114010","article-title":"Murine mucopolysaccharidosis type VII. Characterization of a mouse with beta-glucuronidase deficiency","volume":"83","author":"Birkenmeier","year":"1989","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.1999.7176_RF12","doi-asserted-by":"crossref","first-page":"3081","DOI":"10.1182\/blood.V78.11.3081.3081","article-title":"Increased lifespan and correction of metabolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation","volume":"78","author":"Birkenmeier","year":"1991","journal-title":"Blood"},{"key":"10.1006\/exnr.1999.7176_RF13","doi-asserted-by":"crossref","first-page":"749","DOI":"10.1038\/360749a0","article-title":"Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer","volume":"360","author":"Wolfe","year":"1992","journal-title":"Nature"},{"key":"10.1006\/exnr.1999.7176_RF14","doi-asserted-by":"crossref","first-page":"154","DOI":"10.1038\/ng0693-154","article-title":"Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts","volume":"4","author":"Moullier","year":"1993","journal-title":"Nature Genet."},{"key":"10.1006\/exnr.1999.7176_RF15","doi-asserted-by":"crossref","first-page":"1642","DOI":"10.1038\/sj.gt.3300775","article-title":"Treatment of lysosomal storage disease in MPS VII mice using a recombinant adeno-associated virus","volume":"5","author":"Watson","year":"1998","journal-title":"Gene Ther."},{"key":"10.1006\/exnr.1999.7176_RF16","doi-asserted-by":"crossref","first-page":"379","DOI":"10.1038\/ng0892-379","article-title":"Herpesvirus vector gene transfer and expression of beta-glucuronidase in the central nervous system of MPS VII mice","volume":"1","author":"Wolfe","year":"1992","journal-title":"Nature Genet."},{"key":"10.1006\/exnr.1999.7176_RF17","doi-asserted-by":"crossref","first-page":"1287","DOI":"10.1073\/pnas.94.4.1287","article-title":"Adenovirus-mediated gene-transfer and expression of human beta-glucuronidase gene in the liver, spleen, and central nervous system in mucopolysaccharidosis type-VII mice","volume":"94","author":"Ohashi","year":"1997","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1006\/exnr.1999.7176_RF18","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1016\/0006-8993(95)01488-8","article-title":"Differential and persistent expression of CNS gene transfer by an adeno-associated virus (AAV) vector","volume":"713","author":"McCown","year":"1996","journal-title":"Brain Res."},{"key":"10.1006\/exnr.1999.7176_RF19","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1006\/exnr.1997.6736","article-title":"Neuron-specific transduction in the rat septohippocampal or nigrostriatal pathway by recombinant adeno-associated virus vector","volume":"42","author":"Klein","year":"1998","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.1999.7176_RF20","doi-asserted-by":"crossref","first-page":"1181","DOI":"10.1089\/hum.1998.9.8-1181","article-title":"Selective and rapid uptake of adeno-associated virus type 2 in brain","volume":"9","author":"Bartlett","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.1999.7176_RF21","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1038\/nm0397-306","article-title":"Recombinant adeno-associated virus for muscle-directed gene therapy","volume":"3","author":"Fisher","year":"1997","journal-title":"Nature Med."},{"key":"10.1006\/exnr.1999.7176_RF22","first-page":"1575","article-title":"Variant clinical course of MPS VII in two groups of mice carrying the same mutation","volume":"78","author":"Casal","year":"1998","journal-title":"Lab. Invest."},{"key":"10.1006\/exnr.1999.7176_RF23","doi-asserted-by":"crossref","first-page":"367","DOI":"10.1038\/374367a0","article-title":"Neural progenitor cell engraftment corrects lysosomal storage throughout the MPS VII mouse brain","volume":"374","author":"Snyder","year":"1995","journal-title":"Nature"},{"key":"10.1006\/exnr.1999.7176_RF24","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1128\/JVI.73.1.161-169.1999","article-title":"Formation of adeno-associated virus circular genomes is differentially regulated by adenovirus E4 ORF6 and E2a gene expression","volume":"73","author":"Duan","year":"1999","journal-title":"J. Virol."},{"key":"10.1006\/exnr.1999.7176_RF25","series-title":"GUS Protocols","first-page":"189","article-title":"\u03b2-glucuronidase (GUS) assay in animal tissue","author":"Kyle","year":"1992"},{"key":"10.1006\/exnr.1999.7176_RF26","doi-asserted-by":"crossref","first-page":"2324","DOI":"10.1172\/JCI117237","article-title":"Enzyme replacement therapy for murine mucopolysaccharidosis type VII","volume":"93","author":"Sands","year":"1994","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.1999.7176_RF27","doi-asserted-by":"crossref","first-page":"1596","DOI":"10.1172\/JCI119322","article-title":"Murine mucopolysaccharidosis type VII: long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow","volume":"99","author":"Sands","year":"1997","journal-title":"J. Clin. Invest."},{"key":"10.1006\/exnr.1999.7176_RF28","doi-asserted-by":"crossref","first-page":"562","DOI":"10.1007\/s004010050562","article-title":"Neuropathology of murine mucopolysaccharidosis type VII","volume":"92","author":"Levy","year":"1996","journal-title":"Acta Neuropathol."},{"key":"10.1006\/exnr.1999.7176_RF29","doi-asserted-by":"crossref","first-page":"2079","DOI":"10.1046\/j.1471-4159.1997.68052079.x","article-title":"Glycosaminoglycan storage in neonatal murine mucopolysaccharidosis type VII neuroglial cells and correction by \u03b2-glucuronidase gene transfer","volume":"68","author":"Taylor","year":"1997","journal-title":"J. Neurochem."},{"key":"10.1006\/exnr.1999.7176_RF30","unstructured":"Casal, M. L, and, J. H. Wolfe, Mucopolysaccharidosis VII in the developing mouse fetus. Submitted."},{"key":"10.1006\/exnr.1999.7176_RF31","doi-asserted-by":"crossref","first-page":"837","DOI":"10.1203\/00006450-199312000-00028","article-title":"Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse","volume":"34","author":"Vogler","year":"1993","journal-title":"Pediatr. Res."},{"key":"10.1006\/exnr.1999.7176_RF32","doi-asserted-by":"crossref","first-page":"5","DOI":"10.1016\/0304-3940(94)91015-4","article-title":"Direct plasmid mediated transfection of adult murine brain cells in vivo using cationic liposomes","volume":"167","author":"Roessler","year":"1994","journal-title":"Neurosci. Lett."},{"key":"10.1006\/exnr.1999.7176_RF33","doi-asserted-by":"crossref","first-page":"606","DOI":"10.1006\/excr.1994.1298","article-title":"Cross-correction of beta-glucuronidase deficiency by retroviral vector-mediated gene transfer","volume":"214","author":"Taylor","year":"1994","journal-title":"Exp. Cell Res."},{"key":"10.1006\/exnr.1999.7176_RF34","first-page":"417","article-title":"Increased susceptibility to the pathogenic effects of wild-type and recombinant herpesviruses in MPS VII mice compared to normal siblings","volume":"2","author":"Wolfe","year":"1996","journal-title":"J. Neuro. Virol."},{"key":"10.1006\/exnr.1999.7176_RF35","first-page":"84","article-title":"Neonatal intramuscular injection with recombinant adeno-associated virus results in prolonged \u03b2-glucuronidase expression in situ and correction of liver pathology in mucopolysaccharidosis type VII mice","volume":"10","author":"Daly","year":"1998","journal-title":"Hum. Gene Ther."},{"key":"10.1006\/exnr.1999.7176_RF36","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1006\/exnr.1994.1192","article-title":"Specific patterns of defective HSV-1 gene transfer in the adult central nervous system: Implications for gene targeting","volume":"130","author":"Wood","year":"1994","journal-title":"Exp. Neurol."},{"key":"10.1006\/exnr.1999.7176_RF37","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/0006-8993(94)91919-4","article-title":"Adenoviral vectors as functional retrograde neuronal tracers","volume":"648","author":"Ridoux","year":"1994","journal-title":"Brain Res."}],"container-title":["Experimental Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488699971765?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488699971765?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,16]],"date-time":"2025-09-16T05:29:00Z","timestamp":1758000540000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014488699971765"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[1999,11]]},"references-count":37,"journal-issue":{"issue":"1","published-print":{"date-parts":[[1999,11]]}},"alternative-id":["S0014488699971765"],"URL":"https:\/\/doi.org\/10.1006\/exnr.1999.7176","relation":{},"ISSN":["0014-4886"],"issn-type":[{"value":"0014-4886","type":"print"}],"subject":[],"published":{"date-parts":[[1999,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Sustained Production of \u03b2-Glucuronidase from Localized Sites after AAV Vector Gene Transfer Results in Widespread Distribution of Enzyme and Reversal of Lysosomal Storage Lesions in a Large Volume of Brain in Mucopolysaccharidosis VII Mice","name":"articletitle","label":"Article Title"},{"value":"Experimental Neurology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1006\/exnr.1999.7176","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"converted-article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 1999 Academic Press. All rights reserved.","name":"copyright","label":"Copyright"}]}}