{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,27]],"date-time":"2026-10-27T03:55:10Z","timestamp":1793073310740,"version":"4.5.0"},"reference-count":25,"publisher":"Oxford University Press (OUP)","issue":"7","license":[{"start":{"date-parts":[[2011,7,1]],"date-time":"2011-07-01T00:00:00Z","timestamp":1309478400000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/academic.oup.com\/journals\/pages\/open_access\/funder_policies\/chorus\/standard_publication_model"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":[],"published-print":{"date-parts":[[2011,7,1]]},"abstract":"<jats:sec>\n                    <jats:title>BACKGROUND<\/jats:title>\n                    <jats:p>New therapies for the treatment of mucopolysaccharidoses that target the brain, including intrathecal enzyme replacement, are being explored. Quantitative analysis of the glycosaminoglycans (GAGs) that accumulate in these disorders is required to assess the disease burden and monitor the effect of therapy in affected patients. Because current methods lack the required limit of quantification and specificity to analyze GAGs in small volumes of cerebrospinal fluid (CSF), we developed a method based on ultra-performance liquid chromatography\u2013tandem mass spectrometry (UPLC-MS\/MS).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>METHODS<\/jats:title>\n                    <jats:p>Samples of CSF (25 \u03bcL) were evaporated to dryness and subjected to methanolysis. The GAGs were degraded to uronic acid-N-acetylhexosamine dimers and mixed with internal standards derived from deuteriomethanolysis of GAG standards. Specific dimers derived from heparan, dermatan and chondroitin sulfates (HS, DS and CS) were separated by UPLC and analyzed by electrospray ionization MS\/MS using selected reaction monitoring for each targeted GAG product and its corresponding internal standard.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>RESULTS<\/jats:title>\n                    <jats:p>CSF from control pediatric subjects (n = 22) contained &amp;lt;0.38 mg\/L HS, 0.26 mg\/L DS, and 2.8 mg\/L CS, whereas CSF from patients with Hurler syndrome (n = 7) contained concentrations of DS and HS that were at least 6-fold greater than the upper control limits. These concentrations were reduced by 17.5% to 82.5% after allogeneic transplantation and treatment with intrathecal and intravenous enzyme replacement therapy.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>CONCLUSIONS<\/jats:title>\n                    <jats:p>The method described here has potential value in monitoring patients with mucopolysaccharidoses receiving treatment targeted to the brain.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1373\/clinchem.2010.161141","type":"journal-article","created":{"date-parts":[[2011,5,17]],"date-time":"2011-05-17T00:23:50Z","timestamp":1305591830000},"page":"1005-1012","source":"Crossref","is-referenced-by-count":94,"title":["Analysis of Glycosaminoglycans in Cerebrospinal Fluid from Patients with Mucopolysaccharidoses by Isotope-Dilution Ultra-Performance Liquid Chromatography\u2013Tandem Mass Spectrometry"],"prefix":"10.1093","volume":"57","author":[{"given":"Haoyue","family":"Zhang","sequence":"first","affiliation":[{"name":"Duke University Medical Center, Pediatrics, Medical Genetics Division, Durham, NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah P","family":"Young","sequence":"first","affiliation":[{"name":"Duke University Medical Center, Pediatrics, Medical Genetics Division, Durham, NC"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christiane","family":"Auray-Blais","sequence":"first","affiliation":[{"name":"Services of Genetics, Department of Pediatrics, Faculty of Medicine and Health Sciences, Universit\u00e9 de Sherbrooke, Sherbrooke, Quebec, Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul J","family":"Orchard","sequence":"first","affiliation":[{"name":"Division of Pediatric Blood and Marrow Transplantation, Institute of Human Genetics, University of Minnesota, Minneapolis, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jakub","family":"Tolar","sequence":"first","affiliation":[{"name":"Division of Pediatric Blood and Marrow Transplantation, Institute of Human Genetics, University of Minnesota, Minneapolis, MN"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David S","family":"Millington","sequence":"first","affiliation":[{"name":"Duke University Medical Center, Pediatrics, Medical Genetics Division, Durham, NC"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"286","published-online":{"date-parts":[[2011,7,1]]},"reference":[{"key":"2020022704542004300_B1","doi-asserted-by":"crossref","first-page":"965","DOI":"10.1002\/ajmg.a.32812","article-title":"Mucopolysaccharidosis type I in 21 Czech and Slovak patients: mutation analysis suggests a functional importance of C-terminus of the IDUA protein","volume":"149A","author":"Vazna","year":"2009","journal-title":"Am J Med 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