{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,5]],"date-time":"2026-10-05T14:31:54Z","timestamp":1791210714185,"version":"4.1.0"},"reference-count":43,"publisher":"Elsevier BV","issue":"4","license":[{"start":{"date-parts":[[2004,8,1]],"date-time":"2004-08-01T00:00:00Z","timestamp":1091318400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2004,8]]},"DOI":"10.1016\/j.ymgme.2004.05.005","type":"journal-article","created":{"date-parts":[[2004,8,5]],"date-time":"2004-08-05T15:26:14Z","timestamp":1091719574000},"page":"273-285","source":"Crossref","is-referenced-by-count":59,"title":["Intracerebral injection of sulfamidase delays neuropathology in murine MPS-IIIA"],"prefix":"10.1016","volume":"82","author":[{"given":"Peter S","family":"Savas","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kim M","family":"Hemsley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"John J","family":"Hopwood","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2004.05.005_BIB1","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","article-title":"Prevalence of lysosomal storage disorders","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB2","first-page":"381","article-title":"The mucopolysaccharidoses. Diagnosis, molecular genetics and treatment","volume":"7","author":"Hopwood","year":"1990","journal-title":"Mol. Biol. Med."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB3","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1093\/glycob\/9.12.1389","article-title":"A mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome)","volume":"9","author":"Bhaumik","year":"1999","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB4","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/glycob\/11.1.99","article-title":"A novel missense mutation in lysosomal sulfamidase is the basis of MPS III A in a spontaneous mouse mutant","volume":"11","author":"Bhattacharyya","year":"2001","journal-title":"Glycobiology"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB5","doi-asserted-by":"crossref","first-page":"282","DOI":"10.1006\/mgme.1998.2761","article-title":"Glycosaminoglycan accumulation and excretion in the mucopolysaccharidoses: characterization and basis of a diagnostic test for MPS","volume":"65","author":"Byers","year":"1998","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB6","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1016\/0003-2697(82)90674-1","article-title":"High-resolution electrophoresis of urinary glycosaminoglycans: an improved screening test for the mucopolysaccharidoses","volume":"119","author":"Hopwood","year":"1982","journal-title":"Anal. Biochem."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB7","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1203\/00006450-199807000-00012","article-title":"Sulfamidase deficiency in a family of Dachshunds: a canine model of mucopolysaccharidosis IIIA (Sanfilippo A)","volume":"44","author":"Fischer","year":"1998","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB8","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1080\/00480169.2000.36181","article-title":"Mucopolysaccharidosis IIIA (Sanfilippo syndrome) in a New Zealand Huntaway dog with ataxia","volume":"49","author":"Jolly","year":"2000","journal-title":"N. Z. Vet. J."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB9","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1097\/00005072-199802000-00006","article-title":"Caprine mucopolysaccharidosis-IIID: clinical, biochemical, morphological and immunohistochemical characteristics","volume":"57","author":"Jones","year":"1998","journal-title":"J. Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB10","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1203\/01.PDR.0000129661.40499.12","article-title":"Enzyme replacement therapy in a murine model of mucopolysaccharidosis type IIIA","volume":"56","author":"Gliddon","year":"2004","journal-title":"Pediatr. Res."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB11","series-title":"Metabolic Basis of Inherited Disease","first-page":"2465","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"1995"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB12","doi-asserted-by":"crossref","first-page":"539","DOI":"10.1067\/mpd.2001.112171","article-title":"The efficacy of enzyme replacement therapy in patients with chronic neuronopathic Gaucher's disease","volume":"138","author":"Altarescu","year":"2001","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB13","doi-asserted-by":"crossref","first-page":"733","DOI":"10.2165\/00003495-200262050-00002","article-title":"New prospects for the treatment of lysosomal storage diseases","volume":"62","author":"Schiffmann","year":"2002","journal-title":"Drugs"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB14","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1007\/BF00710053","article-title":"Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation","volume":"18","author":"Shapiro","year":"1995","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB15","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1006\/bcmd.2001.0453","article-title":"In utero fetal liver cell transplantation without toxic irradiation alleviates lysosomal storage in mice with mucopolysaccharidosis type VII","volume":"27","author":"Barker","year":"2001","journal-title":"Blood Cells Mol. Dis."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB16","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.ymgme.2004.05.005_BIB17","doi-asserted-by":"crossref","first-page":"15428","DOI":"10.1073\/pnas.192582899","article-title":"Chemical chaperones increase the cellular activity of N370S \u03b2-glucosidase: a therapeutic strategy for Gaucher disease","volume":"99","author":"Sawkar","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB18","doi-asserted-by":"crossref","first-page":"13102","DOI":"10.1073\/pnas.192353499","article-title":"Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs","volume":"99","author":"Ponder","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB19","doi-asserted-by":"crossref","first-page":"6216","DOI":"10.1073\/pnas.082011999","article-title":"Functional correction of established central nervous system deficits in an animal model of lysosomal storage disease with feline immunodeficiency virus-based vectors","volume":"99","author":"Brooks","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"},{"issue":"Part 1","key":"10.1016\/j.ymgme.2004.05.005_BIB20","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1042\/bj3290145","article-title":"Recombinant human sulphamidase: expression, amplification, purification and characterization","volume":"329","author":"Bielicki","year":"1998","journal-title":"Biochem. J."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB21","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/0009-8981(82)90168-1","article-title":"Diagnosis of Sanfilippo type A syndrome by estimation of sulfamidase activity using a radiolabelled tetrasaccharide substrate","volume":"123","author":"Hopwood","year":"1982","journal-title":"Clin. Chim. Acta"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB22","series-title":"The Mouse Brain in Stereotaxic Co-ordinates","author":"Paxinos","year":"2001"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB23","doi-asserted-by":"crossref","first-page":"762","DOI":"10.1002\/ana.10373","article-title":"Selective neurodegeneration in murine mucopolysaccharidosis VII is progressive and reversible","volume":"52","author":"Heuer","year":"2002","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB24","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1002\/jnr.10278","article-title":"Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo syndrome type B","volume":"69","author":"Li","year":"2002","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB25","doi-asserted-by":"crossref","first-page":"6437","DOI":"10.1523\/JNEUROSCI.22-15-06437.2002","article-title":"Distribution of a lysosomal enzyme in the adult brain by axonal transport and by cells of the rostral migratory stream","volume":"22","author":"Passini","year":"2002","journal-title":"J. Neurosci."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB26","doi-asserted-by":"crossref","first-page":"3302","DOI":"10.1523\/JNEUROSCI.23-08-03302.2003","article-title":"Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice","volume":"23","author":"Hennig","year":"2003","journal-title":"J. Neurosci."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB27","doi-asserted-by":"crossref","first-page":"351","DOI":"10.1006\/mthe.2001.0274","article-title":"Intracranial injection of recombinant adeno-associated virus improves cognitive function in a murine model of mucopolysaccharidosis type VII","volume":"3","author":"Frisella","year":"2001","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB28","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/0022-510X(91)90208-O","article-title":"Neuroaxonal dystrophy in neuronal storage disorders: evidence for major GABAergic neuron involvement","volume":"104","author":"Walkley","year":"1991","journal-title":"J. Neurol. Sci."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB29","doi-asserted-by":"crossref","first-page":"29","DOI":"10.1007\/s004010050857","article-title":"Pathological study of mice with total deficiency of sphingolipid activator proteins (SAP knockout mice)","volume":"96","author":"Oya","year":"1998","journal-title":"Acta Neuropathol. (Berl.)"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB30","doi-asserted-by":"crossref","first-page":"175","DOI":"10.1007\/BF00227765","article-title":"Cerebellar degeneration in the Niemann\u2013Pick type C mouse","volume":"85","author":"Higashi","year":"1993","journal-title":"Acta Neuropathol. (Berl.)"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB31","first-page":"87","article-title":"Ubiquitin immunoreactive structures in normal human brains, distribution and developmental aspects","volume":"63","author":"Dickson","year":"1990","journal-title":"Lab. Invest."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB32","doi-asserted-by":"crossref","first-page":"60","DOI":"10.1016\/S0165-0173(01)00067-4","article-title":"Applications of the Morris water maze in the study of learning and memory","volume":"36","author":"D'Hooge","year":"2001","journal-title":"Brain Res. Brain Res. Rev."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB33","doi-asserted-by":"crossref","first-page":"107","DOI":"10.1016\/S0166-4328(02)00274-7","article-title":"Testing the importance of the caudal retrosplenial cortex for spatial memory in rats","volume":"140","author":"Vann","year":"2003","journal-title":"Behav. Brain Res."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB34","doi-asserted-by":"crossref","first-page":"1155","DOI":"10.1523\/JNEUROSCI.22-03-01155.2002","article-title":"Impaired spatial performance in rats with retrosplenial lesions: importance of the spatial problem and the rat strain in identifying lesion effects in a swimming pool","volume":"22","author":"Harker","year":"2002","journal-title":"J. Neurosci."},{"issue":"Part 3","key":"10.1016\/j.ymgme.2004.05.005_BIB35","first-page":"185","article-title":"Developmental and degenerative patterns associated with cognitive, behavioural and motor difficulties in the Sanfilippo syndrome: an epidemiological study","volume":"27","author":"Nidiffer","year":"1983","journal-title":"J. Ment. Defic. Res."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB36","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/S0278-5846(00)00150-0","article-title":"Brain structures and neurotransmitters regulating aggression in cats: implications for human aggression","volume":"25","author":"Gregg","year":"2001","journal-title":"Prog. Neuropsychopharmacol. Biol. Psychiatry"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB37","series-title":"Principles of Neural Science","first-page":"782","article-title":"The control of gaze","author":"Goldberg","year":"2000"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB38","series-title":"Principles of Neural Science","first-page":"430","article-title":"The bodily senses","author":"Gardner","year":"2000"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB39","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1016\/S1357-2725(02)00384-9","article-title":"Neurodegeneration: linking ubiquitin\/proteasome pathway impairment with inflammation","volume":"35","author":"Li","year":"2003","journal-title":"Int. J. Biochem. Cell Biol."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB40","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. USA"},{"key":"10.1016\/j.ymgme.2004.05.005_BIB41","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1093\/hmg\/11.11.1343","article-title":"Molecular pathophysiology in Tay\u2013Sachs and Sandhoff diseases as revealed by gene expression profiling","volume":"11","author":"Myerowitz","year":"2002","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB42","first-page":"251","article-title":"Neuronal ubiquitin and neurofilament expression in different lysosomal storage disorders","volume":"11","author":"Zhan","year":"1992","journal-title":"Clin. Neuropath."},{"key":"10.1016\/j.ymgme.2004.05.005_BIB43","doi-asserted-by":"crossref","first-page":"89","DOI":"10.1016\/S0006-8993(87)80025-2","article-title":"Ectopic axon hillock-associated neurite growth is maintained in metabolically reversed swainsonine-induced neuronal storage disease","volume":"410","author":"Walkley","year":"1987","journal-title":"Brain Res."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719204001350?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719204001350?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2019,2,5]],"date-time":"2019-02-05T03:23:09Z","timestamp":1549336989000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719204001350"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2004,8]]},"references-count":43,"journal-issue":{"issue":"4","published-print":{"date-parts":[[2004,8]]}},"alternative-id":["S1096719204001350"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2004.05.005","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2004,8]]}}}