{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,13]],"date-time":"2026-01-13T04:14:49Z","timestamp":1768277689495,"version":"3.49.0"},"reference-count":33,"publisher":"Elsevier BV","issue":"2","license":[{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-017"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-037"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-012"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-029"},{"start":{"date-parts":[[2009,2,1]],"date-time":"2009-02-01T00:00:00Z","timestamp":1233446400000},"content-version":"stm-asf","delay-in-days":0,"URL":"https:\/\/doi.org\/10.15223\/policy-004"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Experimental Neurology"],"published-print":{"date-parts":[[2009,2]]},"DOI":"10.1016\/j.expneurol.2008.10.021","type":"journal-article","created":{"date-parts":[[2008,11,15]],"date-time":"2008-11-15T04:10:05Z","timestamp":1226722205000},"page":"349-357","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":67,"title":["Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann\u2013Pick A disease"],"prefix":"10.1016","volume":"215","author":[{"given":"James C.","family":"Dodge","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jennifer","family":"Clarke","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Christopher M.","family":"Treleaven","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Tatyana V.","family":"Taksir","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Denise A.","family":"Griffiths","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wendy","family":"Yang","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan A.","family":"Fidler","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Marco A.","family":"Passini","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kenneth P.","family":"Karey","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Edward H.","family":"Schuchman","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Seng H.","family":"Cheng","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Lamya S.","family":"Shihabuddin","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.expneurol.2008.10.021_bib1","first-page":"132","article-title":"Recombinant human acid alpha-glucosidase enzyme therapy for infantile glycogen storage disease type II: results of a phase I\/II clinical trial","volume":"3","author":"Amalfitano","year":"2001","journal-title":"Genet. Med."},{"key":"10.1016\/j.expneurol.2008.10.021_bib2","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1097\/00006123-199902000-00026","article-title":"A prospective study of long-term intrathecal morphine in the management of chronic nonmalignant pain","volume":"44","author":"Anderson","year":"1999","journal-title":"Neurosurgery"},{"key":"10.1016\/j.expneurol.2008.10.021_bib3","doi-asserted-by":"crossref","first-page":"2","DOI":"10.1006\/exnr.2000.7408","article-title":"Convection-enhanced delivery of AAV vector in parkinsonian monkeys; in vivo detection of gene expression and restoration of dopaminergic function using pro-drug approach","volume":"164","author":"Bankiewicz","year":"2000","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.expneurol.2008.10.021_bib4","doi-asserted-by":"crossref","first-page":"431","DOI":"10.1016\/j.ymthe.2005.03.011","article-title":"AAV8-mediated hepatic expression of acid sphingomyelinase corrects the metabolic defect in the visceral organs of a mouse model of Niemann\u2013Pick disease","volume":"12","author":"Barbon","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib6","doi-asserted-by":"crossref","first-page":"1913","DOI":"10.1073\/pnas.87.5.1913","article-title":"Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease","volume":"87","author":"Barton","year":"1990","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib5","doi-asserted-by":"crossref","first-page":"1464","DOI":"10.1056\/NEJM199105233242104","article-title":"Replacement therapy for inherited enzyme deficiency\u2014macrophage-targeted glucocerebrosidase for Gaucher's disease","volume":"324","author":"Barton","year":"1991","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.expneurol.2008.10.021_bib7","first-page":"424","article-title":"Cerebrospinal fluid infusion of alglucerase in the treatment of acute neuronopathic Gaucher's disease","volume":"38","author":"Bembi","year":"1995","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.expneurol.2008.10.021_bib8","doi-asserted-by":"crossref","first-page":"653","DOI":"10.1021\/bi00842a021","article-title":"The filipin complex","volume":"7","author":"Bergy","year":"1968","journal-title":"Biochemistry"},{"key":"10.1016\/j.expneurol.2008.10.021_bib9","doi-asserted-by":"crossref","first-page":"2076","DOI":"10.1073\/pnas.91.6.2076","article-title":"Convection-enhanced delivery of macromolecules in the brain","volume":"91","author":"Bobo","year":"1994","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib10","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1073\/pnas.55.2.366","article-title":"The metabolism of sphingomyelin. II. Evidence of an enzymatic deficiency in Niemann\u2013Pick disease","volume":"55","author":"Brady","year":"1966","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib11","doi-asserted-by":"crossref","first-page":"1782","DOI":"10.1038\/sj.mt.6300249","article-title":"Timing of therapeutic intervention determines functional and survival outcomes in a mouse model of late infantile batten disease","volume":"15","author":"Cabrera-Salazar","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib12","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/mt.2008.9","article-title":"Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Chang","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib13","doi-asserted-by":"crossref","first-page":"17822","DOI":"10.1073\/pnas.0509062102","article-title":"Gene transfer of human acid sphingomyelinase corrects neuropathology and motor deficits in a mouse model of Niemann\u2013Pick type A disease","volume":"102","author":"Dodge","year":"2005","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib14","doi-asserted-by":"crossref","first-page":"1056","DOI":"10.1038\/mt.2008.60","article-title":"Delivery of AAV-IGF-1 to the CNS extends survival in ALS mice through modification of aberrant glial cell activity","volume":"16","author":"Dodge","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib15","doi-asserted-by":"crossref","first-page":"574","DOI":"10.1016\/j.jpeds.2004.03.018","article-title":"Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux\u2013Lamy syndrome)","volume":"144","author":"Harmatz","year":"2004","journal-title":"J. Pediatr."},{"key":"10.1016\/j.expneurol.2008.10.021_bib16","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.ymgme.2006.10.005","article-title":"Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice","volume":"90","author":"Hemsley","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.expneurol.2008.10.021_bib17","doi-asserted-by":"crossref","first-page":"288","DOI":"10.1038\/ng0795-288","article-title":"Acid sphingomyelinase deficient mice: a model of types A and B Niemann\u2013Pick disease","volume":"10","author":"Horinouchi","year":"1995","journal-title":"Nat. Genet."},{"key":"10.1016\/j.expneurol.2008.10.021_bib19","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1016\/j.ymthe.2003.07.008","article-title":"Ex vivo gene therapy using bone marrow-derived cells: combined effects of intracerebral and intravenous transplantation in a mouse model of Niemann\u2013Pick disease","volume":"8","author":"Jin","year":"2003","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib18","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1172\/JCI14862","article-title":"Intracerebral transplantation of mesenchymal stem cells into acid sphingomyelinase-deficient mice delays the onset of neurological abnormalities and extends their life span","volume":"109","author":"Jin","year":"2002","journal-title":"J. Clin. Invest."},{"key":"10.1016\/j.expneurol.2008.10.021_bib20","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1016\/j.ymgme.2004.07.003","article-title":"Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I","volume":"83","author":"Kakkis","year":"2004","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.expneurol.2008.10.021_bib21","doi-asserted-by":"crossref","first-page":"182","DOI":"10.1056\/NEJM200101183440304","article-title":"Enzyme-replacement therapy in mucopolysaccharidosis I","volume":"344","author":"Kakkis","year":"2001","journal-title":"N. Engl. J. Med."},{"key":"10.1016\/j.expneurol.2008.10.021_bib22","doi-asserted-by":"crossref","first-page":"2520","DOI":"10.1096\/fj.06-6169com","article-title":"Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy","volume":"21","author":"Lee","year":"2007","journal-title":"FASEB J."},{"key":"10.1016\/j.expneurol.2008.10.021_bib23","doi-asserted-by":"crossref","first-page":"44976","DOI":"10.1074\/jbc.M106455200","article-title":"Acid sphingomyelinase-deficient macrophages have defective cholesterol trafficking and efflux","volume":"276","author":"Leventhal","year":"2001","journal-title":"J. Biol. Chem."},{"key":"10.1016\/j.expneurol.2008.10.021_bib24","doi-asserted-by":"crossref","first-page":"542","DOI":"10.1002\/ana.20444","article-title":"Convection perfusion of glucocerebrosidase for neuronopathic Gaucher's disease","volume":"57","author":"Lonser","year":"2005","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.expneurol.2008.10.021_bib25","doi-asserted-by":"crossref","first-page":"1988","DOI":"10.1096\/fj.00-0014com","article-title":"Infusion of recombinant human acid sphingomyelinase into Niemann\u2013Pick disease mice leads to visceral, but not neurological, correction of the pathophysiology","volume":"14","author":"Miranda","year":"2000","journal-title":"FASEB J."},{"key":"10.1016\/j.expneurol.2008.10.021_bib27","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1016\/j.ymthe.2005.01.011","article-title":"AAV vector-mediated correction of brain pathology in a mouse model of Niemann\u2013Pick A disease","volume":"11","author":"Passini","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.expneurol.2008.10.021_bib26","doi-asserted-by":"crossref","first-page":"9505","DOI":"10.1073\/pnas.0703509104","article-title":"Combination brain and systemic injections of AAV provide maximal functional and survival benefits in the Niemann\u2013Pick mouse","volume":"104","author":"Passini","year":"2007","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib28","doi-asserted-by":"crossref","first-page":"2743","DOI":"10.1001\/jama.285.21.2743","article-title":"Enzyme replacement therapy in Fabry disease: a randomized controlled trial","volume":"285","author":"Schiffmann","year":"2001","journal-title":"JAMA"},{"key":"10.1016\/j.expneurol.2008.10.021_bib29","series-title":"The Metabolic and Molecular Basis of Inherited Disease","first-page":"3589","article-title":"Niemann\u2013Pick disease types A and B: acid sphingomyelinase deficiencies","author":"Schuchman","year":"2001"},{"key":"10.1016\/j.expneurol.2008.10.021_bib30","doi-asserted-by":"crossref","first-page":"10642","DOI":"10.1523\/JNEUROSCI.3584-04.2004","article-title":"Intracerebral transplantation of adult mouse neural progenitor cells into the Niemann\u2013Pick-A mouse leads to a marked decrease in lysosomal storage pathology","volume":"24","author":"Shihabuddin","year":"2004","journal-title":"J. Neurosci."},{"key":"10.1016\/j.expneurol.2008.10.021_bib31","doi-asserted-by":"crossref","first-page":"5760","DOI":"10.1073\/pnas.102175599","article-title":"Brain-directed gene therapy for lysosomal storage disease: going well beyond the blood\u2013brain barrier","volume":"99","author":"Sly","year":"2002","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.expneurol.2008.10.021_bib32","doi-asserted-by":"crossref","first-page":"258","DOI":"10.1016\/j.expneurol.2007.06.017","article-title":"Intraparenchymal injections of acid sphingomyelinase results in regional correction of lysosomal storage pathology in the Niemann\u2013Pick A mouse","volume":"207","author":"Yang","year":"2007","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.expneurol.2008.10.021_bib33","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1038\/sj.mt.6300066","article-title":"Correction of the biochemical and functional deficits in Fabry mice following AAV8-mediated hepatic expression of alpha-galactosidase A","volume":"15","author":"Ziegler","year":"2007","journal-title":"Mol. Ther."}],"container-title":["Experimental Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488608004172?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0014488608004172?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,16]],"date-time":"2025-09-16T05:36:24Z","timestamp":1758000984000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0014488608004172"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2009,2]]},"references-count":33,"journal-issue":{"issue":"2","published-print":{"date-parts":[[2009,2]]}},"alternative-id":["S0014488608004172"],"URL":"https:\/\/doi.org\/10.1016\/j.expneurol.2008.10.021","relation":{},"ISSN":["0014-4886"],"issn-type":[{"value":"0014-4886","type":"print"}],"subject":[],"published":{"date-parts":[[2009,2]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann\u2013Pick A disease","name":"articletitle","label":"Article Title"},{"value":"Experimental Neurology","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.expneurol.2008.10.021","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2008 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}