A genetically myeloablated MPS VII model detects the expansion and curative properties of as few as 100 enriched murine stem cells

Experimental Hematology

Volumn 27, Issue 11, 1999, Pages 1691-1704

  Soper, Brian W ^a   Duffy, Ted M ^a   Vogler, Carole A ^b   Barker, Jane E ^a  

^a JACKSON LABORATORY   (United States)

^b SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

Author keywords

glucuronidase; Bone marrow transplantation; Hematopoietic stem cells; In situ; MPS VII

Indexed keywords

BETA GLUCURONIDASE; STEM CELL FACTOR RECEPTOR;

ANIMAL CELL; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOCHEMISTRY; BONE MARROW CELL; DONOR; HEMATOPOIETIC STEM CELL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HISTOCHEMISTRY; HISTOPATHOLOGY; LIFESPAN; LYMPHOID CELL; MOUSE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PRIORITY JOURNAL;

ANIMALS; CELL COUNT; GRAFT SURVIVAL; HEMATOPOIETIC STEM CELL TRANSPLANTATION; MICE; MICE, MUTANT STRAINS; MUCOPOLYSACCHARIDOSIS VII;

ANIMALIA; MURINAE; SIGANUS;

EID: 0032699530     PISSN: 0301472X     EISSN: None     Source Type: Journal    
DOI: 10.1016/S0301-472X(99)00098-3     Document Type: Article

References (44)

1. Harrison D.E. Lifesparing ability (in lethally irradiated mice) of W-W mouse marrow with no macroscopic colonies. Radiat Res. 52:1972;553.
2. Harrison D.E. Competitive repopulation. a new assay for long-term stem cell functional capacity Blood. 55:1980;77.
3. Emerson S.G. Ex vivo expansion of hematopoietic precursors, progenitors, and stem cells. the next generation of cellular therapeutics Blood. 87:1996;3082.
4. McCulloch E.A. Stem cell renewal and determination during clonal expansion in normal and leukaemic haemopoisis. Cell Prolif. 26:1993;399.
5. Micklem H.S., Ford C.E., Evans E.P., Ogden D.A., Papworth D.S. Competitive in vivo proliferation of foetal and adult haematopoietic cells in lethally irradiated mice. J Cell Physiol. 79:1972;293.
6. Harrison D.E., Jordan C.T., Zhong R.K., Astle C.M. Primitive hemopoietic stem cells. direct assay of most productive populations by competitive repopulation with simple binomial, correlation and covariance calculations Exp Hematol. 21:1993;206.
7. Szilvassy S.J., Humphries R.K., Lansdorp P.M., Eaves A.C., Eaves C.J. Quantitative assay for totipotent reconstituting hematopoietic stem cells by a competitive repopulation strategy. Proc Natl Acad Sci U S A. 87:1990;8736.
8. Sands M.S., Barker J.E., Vogler C., Levy B., Gwynn B., Galvin N., Sly W.S., Birkenmeier E.H. Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab Invest. 68:1993;676.
9. Gerritsen E.J., Vossen J.M., Fasth A., Friedrich W., Morgan G., Padmos A., Vellodi A., Porras O., O'Meara A., Porta F.et al. Bone marrow transplantation for autosomal recessive osteopetrosis. A report from the Working Party on Inborn Errors of the European Bone Marrow Transplantation Group. J Pediatr. 125:1994;896.
10. Zanjani E.D., Pallavacini M.G., Ascensao J.L., Flake A.W., Harrison M.R., Tavassoli M. Human-ovine xenogenic transplantation of stem cells in utero. Bone Marrow Transplant. 1:1992;86.
11. Carrier E., Lee T.H., Busch M.P., Cowan M.J. Recruitment of engrafted donor cells postnatally into the blood with cytokines after in utero transplantation in mice. Transplantation. 64:1997;627.
12. Geissler E.N., McFarland E.C., Russell E.S. Analysis of pleiotropism at the dominant white-spotting (W) locus of the house mouse. a description of ten new W alleles Genetics. 97:1981;337.
13. 41 and W EMBO J. 9:1990;1805.
14. Sly W.S., Quinton B.A., McAlister W.H., Rimoin D.L. Beta glucuronidase deficiency. report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis J Pediat. 82:1973;249.
15. Chang P.L., Lambert D.T., Pisa M.A. Behavioural abnormalities in a murine model of a human lysosomal storage disease. Neuroreport. 4:1993;507.
16. Birkenmeier E.H., Davisson M.T., Beamer W.G., Ganschow R.E., Vogler C.A., Gwynn B., Lyford K.A., Maltias L.M., Wawrzyniak C.J. Murine mucopolysaccharidosis type VII. Characterization of a mouse with β-glucuronidase. J Clin Invest. 83:1989;1258.
17. Neufeld E.F., Muenzer J. The mucopolysaccharidoses. Scriver C.R., Beaudet A.L., Sly W.S., Valle D. Metabolic basis of inherited disease. 7th ed:1995;2465 McGraw-Hill, New York.
18. Sands M.S., Vogler C., Torrey A., Levy B., Gwynn B., Grubb J., Sly W.S., Birkenmeier E.H. Murine mucopolysaccharidosis type VII. long term therapeutic effects of enzyme replacement and enzyme replacement followed by bone marrow transplantation J Clin Invest. 99:1997;1596.
19. Vogler C., Sands M., Higgins A., Levy B., Grubb J., Birkenmeier E.H., Sly W.S. Enzyme replacement with recombinant β-glucuronidase in the newborn mucopolysaccharidosis type VII mouse. Pediat Res. 34:1993;837.
20. Volger C., Sands M.S., Levy B., Galvin N., Birkenmeier E.H., Sly W.S. Enzyme replacement with recombinant β-glucuronidase. impact of therapy during the first six weeks of life on subsequent lysosomal storage, growth and survival Pediatr Res. 39:1996;1050.
21. Marechal V., Naffakh N., Danos O., Heard J.M. Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells. Blood. 82:1993;1358.
22. Sands M.S., Vogler C., Kyle J.W., Grubb J.H., Levy B., Galvin N., Sly W.S., Birkenmeier E.H. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest. 93:1994;2324.
23. Volger C., Sands M.S., Galvin N., Levy B., Thorpe C., Barker J.E., Sly W.S. Murine mucopolysaccharidosis Type VII - the impact of therapies on the clinical coarse and pathology in a murine model of lysosomal storage disease. J Inher Met Disease. 21:1998;575.
24. Wolfe J.H., Sands M.S., Barker J.E., Gwynn B., Rowe L.B., Vogler C.A., Birkenmeier E.H. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature. 360:1992;749.
25. Spangrude G.L., Brooks D.M., Tumas D.B. Long-term repopulation of irradiated mice with limiting numbers of purified hematopoietic stem cells. in vivo expansion of stem cell phenotype but not function Blood. 85:1995;1006.
26. Goodell M.A., Brose K., Paradis G., Conner A.S., Mulligan R.C. Isolation and functional properties of murine hematopoietic stem cells that are replicating in vivo. J Exp Med. 183:1996;1797.
27. Goodell M.A., Rosenzweig M., Kim H., Marks D.F., DeMaria M., Paradis G., Grupp S.A., Sieff C.A., Mulligan R.C., Johnson R.P. Dye efflux studies suggest that hematopoietic stem cells expressing low or undetectable levels of CD34 antigen exist in multiple species. Nat Med. 3:1997;1337.
28. Wolfe J.H., Sands M.S. Murine mucopolysaccharidosis type VII. a model system for somatic gene therapy of the central nervous system Lowenstein P.R., Enquist L.W. Protocols for gene therapy in neuroscience. towards gene therapy of neurological disorders:1996;263 John Wiley & Sons Ltd, Chichester.
29. Petrov R.V., Egorov I.K., Seslavina L.S., Panteleev E.I., Egorova O.S. [Inactivation of allogeneic stem cells by lymphocytes identical as regards the H2 system of tissue compatibility]. Dokl Akad Nauk SSSR. 219:1974;1489.
30. Eicher E.M., Washburn L.L. Assignment of genes to regions of mouse chromosomes. Proc Natl Acad Sci U S A. 75:1978;946.
31. Eppig J.J., Kozak L.P., Eicher E.M., Stevens L.C. Ovarian teratomas in mice are derived from oocytes that have completed the first meiotic division. Nature. 269:1977;517.
32. Whitney J.B. Simplified typing of mouse hemoglobin (Hbb) phenotypes using cystamine. Biochem Genet. 16:1978;667.
33. Barker J.E., Compton S.T. Hematopoietic repopulation of adult mice with beta-thalassemia. Blood. 83:1994;828.
34. Birkenmeier E.H., Barker J.E., Vogler C.A., Kyle J.W., Sly W.S., Gwynn B., Levy B., Pegors C. Increased life span and correction of metobolic defects in murine mucopolysaccharidosis type VII after syngeneic bone marrow transplantation. Blood. 78:1991;3081.
35. Russell E.S. Hereditary anemias of the mouse. a review for geneticists Adv Genet. 20:1979;357.
36. Harrison D.E., Astle C.M. Lymphoid and erythroid repopulation in B6 W-anemic mice. a new unirradiated recipient Exp Hematol. 19:1991;374.
37. v mice with limiting dilutions of +/+ donor marrow cells. Proc Natl Acad Sci U S A. 85:1988;7332.
38. Harrison D.E., Stone M., Astle C.M. Effects of transplantation on the primitive immunohematopoietic stem cell. J Exp Med. 172:1990;431.
39. v mice by injection of 5-fluorouracil-treated bone marrow cells of Pgk-1b/Pgk-1a mice. Blood. 70:1987;1758.
40. Jones D.R., Bui T.H., Anderson E.M., Ek S., Liu D., Ringden O., Westgren M. In utero haematopoietic stem cell transplantation. current perspectives and future potential Bone Marrow Transplant. 18:1996;831.
41. Fleischman R.A., Mintz B. Development of adult bone marrow stem cells in H-2-compatible and -incompatible mouse fetuses. J Exp Med. 159:1984;731.
42. Roodman G.D., Kuehl T.J., Vandeberg J.L., Muirhead D.Y. In utero bone marrow transplantation of fetal baboons with mismatched adult baboon marrow. Blood Cells. 17:1991;367.
43. Toles J.F., Chui D.H., Belbeck L.W., Starr E., Barker J.E. Hemopoietic stem cells in murine embryonic yolk sac and peripheral blood. Proc Natl Acad Sci U S A. 86:1989;7456.
44. Blazar B.R., Taylor P.A., Vallera D.A. In utero transfer of adult bone marrow cells into recipients with severe combined immunodeficiency disorder yields lymphoid progeny with T- and B-cell functional capabilities. Blood. 86:1995;4353.