| Intravenous administration of human umbilical cord blood cells in an animal model of MPS III B. | |
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MedLine Citation:
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PMID: 19399896 Owner: NLM Status: MEDLINE |
Abstract/OtherAbstract:
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Sanfilippo syndrome type B (MPS III B) is caused by a deficiency of alpha-N-acetylglucosaminidase enzyme (Naglu), 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 Naglu mice had a beneficial effect, probably due to enzyme delivery into the enzyme-deficient 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-associated 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 Naglu delivery to enzyme-deficient organs. |
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Authors:
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Svitlana Garbuzova-Davis; Stephen K Klasko; Paul R Sanberg |
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Publication Detail:
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Type: Journal Article; Research Support, Non-U.S. Gov't |
Journal Detail:
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Title: The Journal of comparative neurology Volume: 515 ISSN: 1096-9861 ISO Abbreviation: J. Comp. Neurol. Publication Date: 2009 Jul |
Date Detail:
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Created Date: 2009-05-04 Completed Date: 2009-07-28 Revised Date: - |
Medline Journal Info:
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Nlm Unique ID: 0406041 Medline TA: J Comp Neurol Country: United States |
Other Details:
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Languages: eng Pagination: 93-101 Citation Subset: IM |
Copyright Information:
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Copyright 2009 Wiley-Liss, Inc. |
Affiliation:
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Center of Excellence for Aging & Brain Repair, College of Medicine, University of South Florida, Tampa, Florida 33612, USA. sgarbuzo@health.usf.edu |
Export Citation:
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| MeSH Terms | |
Descriptor/Qualifier:
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Acetylglucosaminidase
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genetics* Animals Brain / enzymology*, physiopathology Cells, Cultured Cognition Disorders / enzymology, physiopathology, therapy Cord Blood Stem Cell Transplantation / methods* Cytokines / metabolism, secretion Disease Models, Animal Female Humans Hyperkinesis / enzymology, physiopathology, therapy Inflammation / enzymology, immunology, therapy Injections, Intravenous / methods Male Mice Mice, Inbred C57BL Mice, Knockout Mucopolysaccharidosis III / enzymology, physiopathology, therapy* Th2 Cells / immunology Treatment Outcome |
| Chemical | |
Reg. No./Substance:
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0/Cytokines; EC 3.2.1.50/alpha-N-acetyl-D-glucosaminidase; EC 3.2.1.52/Acetylglucosaminidase |
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