{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,18]],"date-time":"2026-06-18T11:01:40Z","timestamp":1781780500840,"version":"3.54.5"},"reference-count":65,"publisher":"Elsevier BV","issue":"12","license":[{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,12,1]],"date-time":"2022-12-01T00:00:00Z","timestamp":1669852800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,10,19]],"date-time":"2022-10-19T00:00:00Z","timestamp":1666137600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":["jbc.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Journal of Biological Chemistry"],"published-print":{"date-parts":[[2022,12]]},"DOI":"10.1016\/j.jbc.2022.102625","type":"journal-article","created":{"date-parts":[[2022,10,26]],"date-time":"2022-10-26T01:58:54Z","timestamp":1666749534000},"page":"102625","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":2,"title":["Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction"],"prefix":"10.1016","volume":"298","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7097-5757","authenticated-orcid":false,"given":"Jenna","family":"Magat","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samantha","family":"Jones","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Baridon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vishal","family":"Agrawal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hio","family":"Wong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"Giaramita","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8691-7562","authenticated-orcid":false,"given":"Linley","family":"Mangini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3103-5762","authenticated-orcid":false,"given":"Britta","family":"Handyside","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Catherine","family":"Vitelli","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monica","family":"Parker","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Natasha","family":"Yeung","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yu","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erno","family":"Pungor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ilya","family":"Slabodkin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivia","family":"Gorostiza","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Allora","family":"Aguilera","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melanie J.","family":"Lo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saida","family":"Alcozie","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Terri M.","family":"Christianson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascale M.N.","family":"Tiger","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jon","family":"Vincelette","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3818-4146","authenticated-orcid":false,"given":"Sylvia","family":"Fong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Geuncheol","family":"Gil","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chuck","family":"Hague","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roger","family":"Lawrence","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6364-8187","authenticated-orcid":false,"given":"Daniel J.","family":"Wendt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan H.","family":"Lebowitz","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Stuart","family":"Bunting","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sherry","family":"Bullens","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7811-4564","authenticated-orcid":false,"given":"Brett E.","family":"Crawford","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sushmita M.","family":"Roy","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Josh C.","family":"Woloszynek","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.jbc.2022.102625_bib1","series-title":"The Online Metabolic and Molecular Bases of Inherited Disease","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2019"},{"key":"10.1016\/j.jbc.2022.102625_bib2","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1016\/j.ymgme.2016.08.002","article-title":"A\u00a0prospective one-year natural history study of mucopolysaccharidosis types IIIA and IIIB: implications for clinical trial design","volume":"119","author":"Truxal","year":"2016","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib3","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1016\/j.jpeds.2015.11.079","article-title":"A\u00a0prospective natural history study of mucopolysaccharidosis type IIIA","volume":"170","author":"Shapiro","year":"2016","journal-title":"J.\u00a0Pediatr."},{"key":"10.1016\/j.jbc.2022.102625_bib4","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1002\/ana.22092","article-title":"Mucopolysaccharidosis type IIIA: clinical spectrum and genotype-phenotype correlations","volume":"68","author":"Valstar","year":"2010","journal-title":"Ann. Neurol."},{"key":"10.1016\/j.jbc.2022.102625_bib5","doi-asserted-by":"crossref","first-page":"165539","DOI":"10.1016\/j.bbadis.2019.165539","article-title":"Heparan sulfate proteoglycans: the sweet side of development turns sour in mucopolysaccharidoses","volume":"1865","author":"De Pasquale","year":"2019","journal-title":"Biochim. Biophys. Acta Mol. Basis Dis."},{"key":"10.1016\/j.jbc.2022.102625_bib6","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.jbc.2022.102625_bib7","doi-asserted-by":"crossref","first-page":"740","DOI":"10.1111\/j.1601-183X.2008.00413.x","article-title":"Effect of high dose, repeated intra-cerebrospinal fluid injection of sulphamidase on neuropathology in mucopolysaccharidosis type IIIA mice","volume":"7","author":"Hemsley","year":"2008","journal-title":"Genes Brain Behav."},{"key":"10.1016\/j.jbc.2022.102625_bib8","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1111\/j.1460-9568.2009.06666.x","article-title":"Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease","volume":"29","author":"Hemsley","year":"2009","journal-title":"Eur. J. Neurosci."},{"key":"10.1016\/j.jbc.2022.102625_bib9","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.ymgme.2015.03.002","article-title":"Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice","volume":"115","author":"Beard","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib10","first-page":"59","article-title":"Continual low-dose infusion of sulfamidase is superior to intermittent high-dose delivery in ameliorating neuropathology in the MPS IIIA mouse brain","volume":"29","author":"Beard","year":"2016","journal-title":"JIMD Rep."},{"key":"10.1016\/j.jbc.2022.102625_bib11","doi-asserted-by":"crossref","first-page":"409","DOI":"10.1111\/jnc.13533","article-title":"Low-dose, continuous enzyme replacement therapy ameliorates brain pathology in the neurodegenerative lysosomal disorder mucopolysaccharidosis type IIIA","volume":"137","author":"King","year":"2016","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.jbc.2022.102625_bib12","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.expneurol.2014.09.008","article-title":"Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods","volume":"263","author":"Marshall","year":"2015","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.jbc.2022.102625_bib13","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1007\/s10545-014-9790-8","article-title":"Evaluation of enzyme dose and dose-frequency in ameliorating substrate accumulation in MPS IIIA Huntaway dog brain","volume":"38","author":"King","year":"2015","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.jbc.2022.102625_bib14","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1007\/s10545-016-9994-1","article-title":"Slow, continuous enzyme replacement via spinal CSF in dogs with the pediatric-onset neurodegenerative disease, MPS IIIA","volume":"40","author":"King","year":"2017","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.jbc.2022.102625_bib15","doi-asserted-by":"crossref","first-page":"2693","DOI":"10.1093\/hmg\/ddm223","article-title":"Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes","volume":"16","author":"Fraldi","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.jbc.2022.102625_bib16","doi-asserted-by":"crossref","first-page":"1237","DOI":"10.1089\/hum.2012.029","article-title":"Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer","volume":"23","author":"Ruzo","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jbc.2022.102625_bib17","doi-asserted-by":"crossref","first-page":"3254","DOI":"10.1172\/JCI66778","article-title":"Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy","volume":"123","author":"Haurigot","year":"2013","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.jbc.2022.102625_bib18","doi-asserted-by":"crossref","first-page":"638","DOI":"10.1038\/mt.2015.9","article-title":"Broad functional correction of molecular impairments by systemic delivery of scAAVrh74-hSGSH gene delivery in MPS IIIA mice","volume":"23","author":"Duncan","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.jbc.2022.102625_bib19","doi-asserted-by":"crossref","first-page":"16036","DOI":"10.1038\/mtm.2016.36","article-title":"Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery","volume":"3","author":"Fu","year":"2016","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.jbc.2022.102625_bib20","doi-asserted-by":"crossref","first-page":"363","DOI":"10.1089\/hum.2015.170","article-title":"A\u00a0preclinical study evaluating AAVrh10-based gene therapy for Sanfilippo syndrome","volume":"27","author":"Winner","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jbc.2022.102625_bib21","doi-asserted-by":"crossref","first-page":"203","DOI":"10.1007\/s11011-016-9895-x","article-title":"Glycosaminoglycan levels and structure in a mucopolysaccharidosis IIIA mice and the effect of a highly secreted sulfamidase engineered to cross the blood-brain barrier","volume":"32","author":"Maccari","year":"2017","journal-title":"Metab. Brain Dis."},{"key":"10.1016\/j.jbc.2022.102625_bib22","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1089\/hum.2013.238","article-title":"Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I\/II trial","volume":"25","author":"Tardieu","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.jbc.2022.102625_bib23","first-page":"100510","article-title":"Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice","volume":"21","author":"Gustaavsson","year":"2019","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.jbc.2022.102625_bib24","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A\u00a0highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.jbc.2022.102625_bib25","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.ymgme.2013.07.016","article-title":"Glycan-based biomarkers for mucopolysaccharidoses","volume":"111","author":"Lawrence","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib26","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1016\/j.ymgme.2016.05.006","article-title":"A\u00a0phase 1\/2 study of intrathecal heparan-N-sulfatase in patients with mucopolysaccharidosis IIIA","volume":"118","author":"Jones","year":"2016","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib27","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/j.ymgme.2018.10.006","article-title":"Intrathecal heparan-N-sulfatase in patients with Sanfilippo syndrome type A: a phase IIB randomized trial","volume":"126","author":"Wijburg","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib28","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1016\/j.omtm.2017.05.009","article-title":"Clearance of heparan sulfate and attenuation of CNS pathology by intracerebroventricular BMN 250 in Sanfilippo type B mice","volume":"6","author":"Aoyagi-Scharber","year":"2017","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.jbc.2022.102625_bib29","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1146\/annurev.bi.61.070192.001515","article-title":"Structure and function of the mannose 6-phosphate\/insulin-like growth factor II receptors","volume":"61","author":"Kornfeld","year":"1992","journal-title":"Annu. Rev. Biochem."},{"key":"10.1016\/j.jbc.2022.102625_bib30","doi-asserted-by":"crossref","first-page":"239","DOI":"10.1016\/j.ymgme.2004.07.016","article-title":"Purification and characterization of recombinant murine sulfamidase","volume":"83","author":"Gliddon","year":"2004","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib31","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.jbc.2022.102625_bib32","doi-asserted-by":"crossref","first-page":"1251","DOI":"10.1006\/bbrc.2001.4265","article-title":"Heparan N-sulfatase: in\u00a0vitro mutagenesis of potential N-glycosylation sites","volume":"280","author":"Natale","year":"2001","journal-title":"Biochem. Biophys. Res. Commun."},{"key":"10.1016\/j.jbc.2022.102625_bib33","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1016\/j.ymthe.2018.01.010","article-title":"Overcoming limitations inherent in sulfamidase to improve mucopolysaccharidosis IIIA gene therapy","volume":"26","author":"Chen","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.jbc.2022.102625_bib34","doi-asserted-by":"crossref","first-page":"47","DOI":"10.3390\/ijms18010047","article-title":"Subcellular trafficking of mammalian lysosomal proteins: an extended view","volume":"18","author":"Staudt","year":"2017","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.jbc.2022.102625_bib35","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/S0074-7696(01)09011-8","article-title":"The roles of receptor-associated protein (RAP) as a molecular chaperone for members of the LDL receptor family","volume":"209","author":"Bu","year":"2001","journal-title":"Int. Rev. Cytol."},{"key":"10.1016\/j.jbc.2022.102625_bib36","doi-asserted-by":"crossref","first-page":"719","DOI":"10.1242\/jcs.107.3.719","article-title":"Genetic deficiency in low density lipoprotein receptor-related protein confers cellular resistance to Pseudomonas exotoxin A","volume":"107","author":"Willnow","year":"1994","journal-title":"J.\u00a0Cell Sci."},{"key":"10.1016\/j.jbc.2022.102625_bib59","doi-asserted-by":"crossref","first-page":"306","DOI":"10.1016\/j.cmpb.2010.01.007","article-title":"PKSolver: an add-in program for pharmacokinetic and pharmacodynamics data analysis in Microsoft excel","volume":"99","author":"Zhang","year":"2010","journal-title":"Comput. Methods Programs Biomed."},{"key":"10.1016\/j.jbc.2022.102625_bib37","doi-asserted-by":"crossref","first-page":"676621","DOI":"10.3389\/fimmu.2021.676621","article-title":"Immune response in neurological pathology: emerging role of central and peripheral immune crosstalk","volume":"12","author":"Passaro","year":"2021","journal-title":"Front. Immunol."},{"key":"10.1016\/j.jbc.2022.102625_bib38","doi-asserted-by":"crossref","first-page":"121","DOI":"10.1016\/j.ceb.2015.02.004","article-title":"Glial fibrillary acidic protein (GFAP) and the astrocyte intermediate filament system in diseases of the central nervous system","volume":"32","author":"Hol","year":"2015","journal-title":"Curr. Opin. Cell Biol."},{"key":"10.1016\/j.jbc.2022.102625_bib39","doi-asserted-by":"crossref","first-page":"713031","DOI":"10.3389\/fnmol.2021.713031","article-title":"Tweaking progranulin expression: therapeutic avenues and opportunities","volume":"14","author":"Terryn","year":"2021","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.jbc.2022.102625_bib40","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1186\/s12974-020-02024-8","article-title":"The good, the bad, and the opportunities of the complement system in neurodegenerative disease","volume":"17","author":"Schartz","year":"2020","journal-title":"J.\u00a0Neuroinflammation"},{"key":"10.1016\/j.jbc.2022.102625_bib41","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0155878","article-title":"Low density lipoprotein-receptor related protein 1 is differentially expressed by neuronal and glial populations in the developing and mature mouse central nervous system","volume":"11","author":"Auderset","year":"2016","journal-title":"PLoS One"},{"key":"10.1016\/j.jbc.2022.102625_bib42","doi-asserted-by":"crossref","first-page":"18430","DOI":"10.1074\/jbc.M116.744904","article-title":"High affinity binding of the receptor-associated protein D1D2 domains with the low density lipoprotein receptor-related protein (LRP1) involves bivalent complex formation","volume":"291","author":"Prasad","year":"2016","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.jbc.2022.102625_bib43","doi-asserted-by":"crossref","first-page":"1380","DOI":"10.1038\/82199","article-title":"Uptake of HIV-1 Tat protein mediated by low-density lipoprotein receptor-related protein disrupts the neuronal metabolic balance of the receptor ligands","volume":"6","author":"Liu","year":"2000","journal-title":"Nat. Med."},{"key":"10.1016\/j.jbc.2022.102625_bib44","doi-asserted-by":"crossref","first-page":"42488","DOI":"10.1074\/jbc.M206510200","article-title":"Structural recognition by recombinant human heparanase that plays critical roles in tumor metastasis","volume":"277","author":"Okada","year":"2002","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.jbc.2022.102625_bib45","first-page":"710","article-title":"The enzymatic degradation of heparan sulfate","volume":"24","author":"Griffin","year":"2017","journal-title":"Protein Pet. Lett."},{"key":"10.1016\/j.jbc.2022.102625_bib46","doi-asserted-by":"crossref","first-page":"285","DOI":"10.4155\/bio.15.243","article-title":"A\u00a0novel LC-MS\/MS assay for heparan sulfate screening in the cerebrospinal fluid of mucopolysaccharidosis IIIA patients","volume":"8","author":"Naimy","year":"2016","journal-title":"Bioanalysis"},{"key":"10.1016\/j.jbc.2022.102625_bib47","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/j.ymgme.2018.07.013","article-title":"Evaluation of cerebrospinal fluid heparan sulfate as a biomarker of neuropathology in a murine model of mucopolysaccharidosis type II using high-sensitivity LC\/MS\/MS","volume":"125","author":"Tanaka","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib48","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 transplantation","volume":"99","author":"Sands","year":"1997","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.jbc.2022.102625_bib49","doi-asserted-by":"crossref","first-page":"414","DOI":"10.1111\/j.1365-2990.2010.01158.x","article-title":"Intracisternal enzyme replacement therapy in lysosomal storage diseases: routes of absorption into brain","volume":"37","author":"Jolly","year":"2011","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"10.1016\/j.jbc.2022.102625_bib50","doi-asserted-by":"crossref","first-page":"14870","DOI":"10.1073\/pnas.1416660111","article-title":"Delivery of an enzyme-IGFII fusion protein to the mouse brain is a therapeutic for mucopolysaccharidosis type IIIB","volume":"111","author":"Kan","year":"2014","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.jbc.2022.102625_bib51","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":"10.1016\/j.jbc.2022.102625_bib52","doi-asserted-by":"crossref","first-page":"S42","DOI":"10.1016\/j.ymgme.2017.12.091","article-title":"Pharmacology of BMN 250 administered via intracerebroventricular infusion once every 2 weeks for twenty-six weeks or longer in a canine model of mucopolysaccharidosis type IIIB","volume":"123","author":"Ellinwood","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib53","first-page":"S168","article-title":"ICV-administered BMN 250 is well tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo Syndrome Type B","volume":"41","author":"Lin","year":"2018","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.jbc.2022.102625_bib54","doi-asserted-by":"crossref","first-page":"S40","DOI":"10.1016\/j.ymgme.2018.12.083","article-title":"ICV-administered tralesinidase alfa (BMN 250 NAGLU-IGF2) is well-tolerated and reduces heparan sulfate accumulation in the CNS of subjects with Sanfilippo syndrome type B (MPS IIIB)","volume":"126","author":"Cleary","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.jbc.2022.102625_bib55","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1093\/glycob\/9.12.1389","article-title":"A\u00a0mouse model for mucopolysaccharidosis type III A (Sanfilippo syndrome)","volume":"9","author":"Bhaumik","year":"1999","journal-title":"Glycobiology"},{"key":"10.1016\/j.jbc.2022.102625_bib56","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.brainres.2006.05.079","article-title":"Characterization of a C57BL\/6 congenic mouse strain of mucopolysaccharidosis type IIIA","volume":"1104","author":"Crawley","year":"2006","journal-title":"Brain Res."},{"key":"10.1016\/j.jbc.2022.102625_bib57","doi-asserted-by":"crossref","first-page":"1389","DOI":"10.1002\/elps.201000037","article-title":"Improved sample preparation method for glycan analysis of glycoproteins by CE-LIF and CE-MS","volume":"31","author":"Szabo","year":"2010","journal-title":"Electrophoresis"},{"key":"10.1016\/j.jbc.2022.102625_bib58","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/glycob\/11.1.99","article-title":"A\u00a0novel 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.jbc.2022.102625_bib65","doi-asserted-by":"crossref","first-page":"676","DOI":"10.1038\/nmeth.2019","article-title":"Fiji: an open-source platform for biological-image analysis","author":"Schindelin","year":"2012","journal-title":"Nat. Methods"},{"key":"10.1016\/j.jbc.2022.102625_bib60","doi-asserted-by":"crossref","first-page":"1111","DOI":"10.1002\/pmic.201100463","article-title":"Using iRT, a normalized retention time for more targeted measurement of peptides","volume":"12","author":"Escher","year":"2012","journal-title":"Proteomics"},{"key":"10.1016\/j.jbc.2022.102625_bib61","doi-asserted-by":"crossref","first-page":"430","DOI":"10.1038\/nmeth.1584","article-title":"mProphet: automated data processing and statistical validation for large-scale SRM experiments","volume":"8","author":"Reiter","year":"2011","journal-title":"Nat. Methods"},{"key":"10.1016\/j.jbc.2022.102625_bib62","doi-asserted-by":"crossref","first-page":"33674","DOI":"10.1074\/jbc.M804288200","article-title":"Evolutionary differences in glycosaminoglycan fine structure detected by quantitative glycan reductive isotope labeling","volume":"283","author":"Lawrence","year":"2008","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.jbc.2022.102625_bib63","doi-asserted-by":"crossref","first-page":"197","DOI":"10.1038\/nchembio.766","article-title":"Disease-specific non-reducing end carbohydrate biomarkers for mucopolysaccharidoses","volume":"8","author":"Lawrence","year":"2012","journal-title":"Nat. Chem. Biol."},{"key":"10.1016\/j.jbc.2022.102625_bib64","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1159\/000121839","article-title":"Brain weight-body weight scaling in breeds of dogs and cats","volume":"16","author":"Bronson","year":"1979","journal-title":"Brain Behav. Evol."}],"container-title":["Journal of Biological Chemistry"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0021925822010687?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0021925822010687?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,9,19]],"date-time":"2025-09-19T00:25:54Z","timestamp":1758241554000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0021925822010687"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,12]]},"references-count":65,"journal-issue":{"issue":"12","published-print":{"date-parts":[[2022,12]]}},"alternative-id":["S0021925822010687"],"URL":"https:\/\/doi.org\/10.1016\/j.jbc.2022.102625","relation":{},"ISSN":["0021-9258"],"issn-type":[{"value":"0021-9258","type":"print"}],"subject":[],"published":{"date-parts":[[2022,12]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction","name":"articletitle","label":"Article Title"},{"value":"Journal of Biological Chemistry","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.jbc.2022.102625","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 THE AUTHORS. Published by Elsevier Inc on behalf of American Society for Biochemistry and Molecular Biology.","name":"copyright","label":"Copyright"}],"article-number":"102625"}}