Transplantation of human umbilical cord blood cells benefits an animal model of Sanfilippo syndrome type B

Stem Cells and Development

Volumn 14, Issue 4, 2005, Pages 384-394

  Garbuzova Davis, Svitlana ^a   Willing, Alison E ^a   Desjarlais, Tammy ^a   Sanberg, Cyndy Davis ^b   Sanberg, Paul R ^a  

^a UNIVERSITY OF SOUTH FLORIDA   (United States)

^b University Technology Center II   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLGLUCOSAMINIDASE; GLYCOSAMINOGLYCAN; HEPARAN SULFATE;

ANIMAL CELL; ANTIGEN EXPRESSION; ARTICLE; ASTROCYTE; BRAIN VENTRICLE; CELL MIGRATION; CELL SURVIVAL; CELL TRANSPLANTATION; CEREBELLUM; CORD BLOOD STEM CELL TRANSPLANTATION; CYTOARCHITECTURE; ENZYME DEFICIENCY; ENZYME REPLACEMENT; FEMALE; GENE; HIPPOCAMPUS; HISTOPATHOLOGY; HUMAN; HUMAN CELL; IN VIVO STUDY; LONG TERM EXPOSURE; MALE; MONONUCLEAR CELL; MOUSE; NAGLU GENE; NONHUMAN; PRIORITY JOURNAL; SANFILIPPO SYNDROME; SANFILIPPO SYNDROME TYPE B; TREATMENT OUTCOME; UMBILICAL CORD BLOOD;

ANIMALS; CELL DIFFERENTIATION; CELL MOVEMENT; CELL SURVIVAL; CELL TRANSPLANTATION; CEREBELLUM; DISEASE MODELS, ANIMAL; FETAL BLOOD; GLYCOSAMINOGLYCANS; HEPARITIN SULFATE; HIPPOCAMPUS; HUMANS; IMMUNOHISTOCHEMISTRY; LEUKOCYTES, MONONUCLEAR; LIVER; MICE; MUCOPOLYSACCHARIDOSIS III; NEURONS; UMBILICAL VEINS;

ANIMALIA;

EID: 24144485784     PISSN: 15473287     EISSN: None     Source Type: Journal    
DOI: 10.1089/scd.2005.14.384     Document Type: Article

References (44)

1. Neufeld EF and J Muenzer. (1995). The mucopolysaccharidoses. In: The Metabolic and Molecular Bases of Inherited Disease. C Scriver, A Beaudet, W Sly and D Valle, eds. McGraw-Hill, New York, pp 2465-2494.
2. Zhao HG, HH Li, A Schmidtchen and EF Neufeld. (1996). The molecular basis of Sanfilippo syndrome type B. Proc Natl Acad Sci USA 93:6101-6105.
3. Weber B, L Blanch, PR Clements, HS Scott and JJ Hopwood. (1996). Cloning and expression of the gene involved in Sanfilippo B syndrome (mucopolysaccharidosis III B). Hum Mol Genet 5:771-777.
4. Zhao HG, EL Aronovich and CB Whitley. (1998). Genotype-phenotype correspondence in Sanfilippo syndrome type B. Am J Hum Genet 62:53-63.
5. Weber B, XH Guo, WJ Kleijer, JJ van de Kamp, BJ Poorthuis and JJ Hopwood. (1996). Sanfilippo type B syndrome (mucopolysaccharidosis type III B): allelic heterogeneity corresponds to the wide spectrum of clinical phenotypes. Eur J Hum Genet 7:34-44.
6. Yogalingam G and JJ Hopwood. (2001). Molecular genetics of mucopolysaccharidosis type III A and III B: diagnostic, clinical, and biological implications. Hum Mutat 18:264-281.
7. Li HH, WH Yu, N Rozengurt, HZ Zhao, KM Lyons, S Anagnostaras, MS Fanselow, K Suzuki, MT Vanier and EF Neufeld. (1999). Mouse model of Sanfilippo syndrome type B produced by targeted disruption of the gene encoding α-N-acetylglucosaminidase. Proc Natl Acad Sci USA 96:14505-14510.
8. Shapiro EG, LA Lockman, M Balthazor and W Krivit. (1995). Neuropsychological outcomes of several storage diseases with and without bone marrow transplantation. J Inherit Metab Dis 18:413-429.
9. Peters C, EG Shapiro, J Anderson, PJ Henslee-Downey, MR Klemperer, MJ Cowan, EF Saunders, PA de Alarcon, C Twist, JB Nachman, GA Hale, RE Harris, MK Rozans, J Kurtzberg, GH Grayson, TE Williams, C Lenarsky, JE Wagner and W Krivit. (1998). Hurler syndrome: II. Outcome of HLA-genotypically identical sibling and HLA-haploidentical related donor bone marrow transplantation in fifty-four children. The Storage Disease Collaborative Study Group. Blood 91:2601-2608.
10. Hoogerbrugge PM, OF Brouwer, P Bordigoni, O Ringden, P Kapaun, JJ Ortega, A O'Meara, G Cornu, G Souillet and D Frappaz. (1995). Allogeneic bone marrow transplantation for lysosomal storage diseases. The European Group for Bone Marrow Transplantation. Lancet 345:1398-1402.
11. Skopura AF, KJ Fisher, JM Wilson, MK Parente and JH Wolfe. (1999). Sustained production of beta-glucuronidase from localized sites after AAV vector gene transfer results in widespread distribution of enzyme and reversal of lysosomal storage lesion in a large volume of brain in mucopolysaccharidosis VII mice. Exp Neurol 160:17-27.
12. Zheng Y, N Rozengurt, S Ryazantsev, DB Kohn, N Satake and EF Neufeld. (2003). Treatment of the mouse model of mucopolysaccharidosis 1 with retrovirally transduced bone marrow. Mol Gen Metab 79:233-244.
13. Zheng Y, S Ryazantsev, K Ohmi, H-Z Zhao, N Rozengurt, DB Kohn and EF Neufeld. (2004). Retrovirally tranduced bone marrow has a therapeutic effect on brain in the mouse model of mucopolysaccharidosis IIIB. Mol Gen Metab 82:286-295.
14. Gluckman E. (2000). Current status of umbilical cord blood hematopoietic stem cell transplantation. Exp Hematol 28:1197-1205.
15. Thomson BG, KA Robertson, D Gowan, D Heilman, HE Broxmeyer, D Emanuel, P Kotylo, Z Brahmi and FO Smith. (2000). Analysis of engraftment, graft-versus-host disease, and immune recovery following unrelated donor cord blood transplantation. Blood 96:2703-2711.
16. Madrigal JA, SB Cohen, E Gluckman and DJ Charron. (1997). Does cord blood transplantation result in lower graft-versus-host disease? It takes more than two to tango. Hum Immunol 56:1-5.
17. Nayar B, GM Raju and D Deka. (2002). Hematopoietic stem/progenitor cell harvesting from umbilical cord blood. Int J Gynaecol Obstet 79:31-32.
18. Todaro AM, C Pafumi, G Pernicone, S Munda, MR Pilastro, A Russo, M Farina and A Messina. (2000). Haematopoietic progenitors from umbilical cord blood. Blood Purif 18:144-147.
19. Broxmeyer HE, GW Douglas, G Hangoc, S Cooper, J Bard, D English, M Arny, L Thomas and EA Boyse. (1989). Human umbilical cord blood as a potential source of transplantable hematopoietic stem/progenitor cells. Proc Natl Acad Sci USA 86:3828-3832.
20. Mikami T, M Eguchi, H Kurosawa, Y Sato, K Sugita, H Suzumura, N Tadokoro, H Watanabe and N Inaba. (2002). Ultrastructural and cytochemical characterization of human cord blood cells. Med Electron Microsc 35:96-101.
21. Sirchia G and P Rebulla. (1999). Placental/umbilical cord blood transplantation. Haematologica 84:738-747.
22. Frassoni F, M Podesta, R Maccario, G Giorgiani, G Rossi, M Zecca, A Bacigalupo, G Piaggio and F Locatelli. (2003). Cord blood transplantation provides better reconstitution of hematopoietic reservoir compared with bone marrow transplantation. Blood 102:1138-1141.
23. Rubinstein P, C Carrier, A Scaradavou, J Kurtzberg, J Adamson, AR Migliaccio, RL Berkowitz, M Cabbad, NL Dobrila, PE Taylor, RE Rosenfield and CE Stevens. (l998). Outcomes among 562 recipients of placental-blood transplants from unrelated donors. N Engl J Med 339:1565-1577.
24. Wagner JE, JN Barker, TE DeFor, KS Baker, BR Blazar, C Eide, A Goldman, J Kersey, W Krivit, ML MacMillan, PJ Orchard, C Peters, DJ Weisdorf, NK Ramsay and SM Davies. (2002). Transplantation of unrelated donor umbilical cord blood in 102 patients with malignant and non-malignant diseases: influence of CD34 cell dose and HLA disparity on treatment-related mortality and survival. Blood 100:1611-1618.
25. Gluckman E, V Rocha and S Chevret. (2001). Results of unrelated umbilical cord blood hematopoietic stem cell transplant. Transfus Clin Biol 8:146-154.
26. Sanchez-Ramos JR, S Song, SG Kamath, T Zigova, A Willing, F Cardozo-Pelaez, T Stedeford, M Chopp and PR Sanberg. (2001). Expression of neural markers in human umbilical cord blood. Exp Neurol 171:109-115.
27. Ha Y, JU Choi, DH Yoon, DS Yeon, JJ Lee, HO Kim and YE Cho. (2001). Neural phenotype expression of cultured human cord blood cells in vitro. NeuroReport 12:3523-3527.
28. Bicknese AR, HS Goodwin, CO Quinn, VC Henderson, SN Chien and DA Wall. (2002). Human umbilical cord blood cells can be induced to express markers for neurons and glia. Cell Transplant 11:261-264.
29. Chen J, PR Sanberg, Y Li, L Wang, M Lu, AE Willing, J Sanchez-Ramos and M Chopp. (2001). Intravenous administration of human umbilical cord blood reduces behavioral deficits after stroke in rats. Stroke 32:2682-2688.
30. Vendrame M, J Cassady, J Newcomb, T Butler, K Pennypacker, T Zigova, C Davis Sanberg, PR Sanberg and AE Willing. (2004). Infusion of human umbilical cord blood cells in a rat model of stroke dose-dependently rescues behavioral deficits and reduces infarct volume. Stroke 35:2390-2395.
31. Lu D, PR Sanberg, A Mahmood, Y Li, L Wang, J Sanchez-Ramos and M Chopp. (2002). Intravenous administration of human umbilical cord blood reduces neurological deficit in the rat after traumatic brain injury. Cell Transplant 11:275-281.
32. Saporta S, JJ Kim, AE Willing, ES Fu, CD Hart and PR Sanberg. (2003). Intravenous administration of human umbilical cord blood cells in rats with spinal cord injury. J Hematother Stem Cell Res 12:271-278.
33. Garbuzova-Davis S, AE Willing, T Zigova, S Saporta, EB Justen, JC Lane, JE Hudson, N Chen, CD Davis and PR Sanberg. (2003). Intravenous administration of human umbilical cord blood cells in a mouse model of ALS: distribution, migration, and differentiation. J Hematother Stem Cell Res 12:255-270.
34. Sanberg PR, AE Willing, C Davis Sanberg, Z Kazi and CV Borlongan. (2004). Differential functional recovery and neurostructural repair of the stroke brain produced by volume-reduced and mononuclear fractioned human umbilical cord blood. Program No. 617 A. Abstract Viewer/Itinerary Planner. Society for Neuroscience, Washington, DC.
35. Henning RJ, H Abu-Ali, JU Balis, MB Morgan, AE Willing and PR Sanberg. (2004). Human umbilical cord blood mononuclear cells for treatment of acute myocardial infarction. Cell Transplant 13:729-739.
36. Staba SL, ML Escolar, M Poe, Y Kim, PL Martin, P Szabolcs, J Allison-Thacker, S Wood, DA Wenger, P Rubinstein, JJ Hopwood, W Krivit and J Kurtzberg. (2004). Cord-blood transplants from unrelated donors in patients with Hurler's syndrome. N Engl J Med 350:1960-1969.
37. Yu WH, KW Zhao, S Ryazantsev, N Rozengurt and EF Neufeld. (2000). Short-term enzyme replacement in the murine model of Sanfilippo syndrome type B. Mol Genet Metab 71:573-580.
38. Zhao KW and EF Neufeld. (2000). Purification and characterization of recombinant human α-N-acetylglucosaminidase secreted by Chinese Hamster Ovary cells. Protein Expr Purif 19:202-211.
39. Li HH, HZ Zhao, EF Neufield, Y Cai and F Gomez-Pinilla. (2003). Attenuated plasticity in neurons and astrocytes in the mouse model of Sanfilippo Syndrome type B. J Neurosci Res 69:30-38.
40. Ohmi K, DS Greenberg, KS Rajavel, S Ryazantsev, HH Li and EF Neufeld. (2003). Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB. Proc Natl Acad Sci USA 100:1902-1907.
41. Pranke P, RR Failace, WF Allebrandt, G Steibel, F Schmidt and NB Nardi. (2001). Hematologic and immunophenotypic characterization of human umbilical cord blood. Acta Haematol 105:71-76.
42. Rainsford E and DJ Reen. (2002). Interleukin 10, produced in abundance by human newborn T cell, may be the regulator of increased tolerance associated with cord blood stem cell transplantation. Br J Haematol 116:702-709.
43. Ransom B, T Behar and M Nedergaard. (2003). New roles for astrocytes (star at last). Trends Neurosci 26:520-522.
44. Chen Y and RA Swanson. (2003). Astrocytes and brain injury. J Cereb Blood Flow Metab 23:137-149.