{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,6]],"date-time":"2026-01-06T13:37:46Z","timestamp":1767706666089},"reference-count":38,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2009,3,20]],"date-time":"2009-03-20T00:00:00Z","timestamp":1237507200000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["J of Comparative Neurology"],"published-print":{"date-parts":[[2009,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:p>Sanfilippo syndrome type B (MPS III B) is caused by a deficiency of \u03b1\u2010N\u2010acetylglucosaminidase enzyme (<jats:italic>Naglu<\/jats:italic>), leading to accumulation of heparan sulfate (HS), a glycosaminoglycan (GAG), within lysosomes and to eventual progressive cerebral and systemic multiple organ abnormalities. Treatment of MPS patients is mainly supportive and enzyme replacement cell therapy shows promise for treating this disease. One new approach for potential treatment of MPS III B is human umbilical cord blood (hUCB) cell transplantation. Recently, we demonstrated that administration of hUCB cells into the cerebral ventricle of presymptomatic <jats:italic>Naglu<\/jats:italic> mice had a beneficial effect, probably due to enzyme delivery into the enzyme\u2010deficient mutant mice. However, administration of these cells into the systemic circulation of mutant mice could be more advantageous and may lead to new strategies of enzyme replacement for Sanfilippo. The aim of this study was to determine the effect of intravenous administration of hUCB cells into a mouse model of Sanfilippo Syndrome type B. The major findings in our study were that hUCB cell administration improved behavioral outcomes (decreased hyper\/stereotypical activity and improved cognitive function). Cells widely distribute within and outside the CNS and intraparenchymally migrate. Administered cells have an antiinflammatory effect (Th2\u2010associated cytokines) in the brain and reduce heparan sulfate accumulation in the liver and spleen. Our results demonstrate the advantages of intravenously administering hUCB cells into a mouse model of Sanfilippo Syndrome type B, the advantages probably a result of <jats:italic>Naglu<\/jats:italic> delivery to enzyme\u2010deficient organs. J. Comp. Neurol. 515:93\u2013101, 2009. \u00a9 2009 Wiley\u2010Liss, Inc.<\/jats:p>","DOI":"10.1002\/cne.21949","type":"journal-article","created":{"date-parts":[[2009,3,20]],"date-time":"2009-03-20T17:00:36Z","timestamp":1237568436000},"page":"93-101","source":"Crossref","is-referenced-by-count":11,"title":["Intravenous administration of human umbilical cord blood cells in an animal model of MPS III B"],"prefix":"10.1002","volume":"515","author":[{"given":"Svitlana","family":"Garbuzova\u2010Davis","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephen K.","family":"Klasko","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul 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