Amphotropic and ecotropic retroviral vector viruses transduce midgestational murine fetal liver cells in a dual-chambered cocultivation system

Gene Therapy

Volumn 4, Issue 1, 1997, Pages 39-44

  Casal, M L ^a   Wolfe, J H ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

Amphotropic retrovirus; Dual chambered cocultivation; Ecotropic retrovirus; In utero transplantation; Mucopolysaccharidosis type VII; Murine fetal liver cells

Indexed keywords

BETA GLUCURONIDASE; COMPLEMENTARY DNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL TRANSPLANTATION; CONTROLLED STUDY; ECOTROPIC VIRUS; ENZYME ACTIVITY; ENZYME RELEASE; EXPRESSION VECTOR; FETUS; FETUS LIVER; GENE EXPRESSION; GENETIC TRANSDUCTION; HISTOCHEMISTRY; LIVER CELL; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PRIORITY JOURNAL; RETROVIRUS;

ANIMALIA; MURINAE; UNIDENTIFIED RETROVIRUS;

EID: 0031023435     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3300352     Document Type: Article

References (28)

1. Sly WS, Quinton BA, McAlister WJ, Rimoin DL. β-Glucuronidase deficiency. Report of clinical, radiologic and biochemical features of a new mucopolysaccharidosis. J Pediatr 1973; 82: 249-257.
2. Birkenmeier EH et al. Murine mucopolysaccharidosis type VII. Characterization of a mouse with β-glucuronidase deficiency. J Clin Invest 1989; 83: 1258-1266.
3. Haskins ME et al. β-Glucuronidase deficiency in a dog: a model of mucopolysaccharidosis VII. Pediatr Res 1984; 18:980-984.
4. Neufeld EF, Muenzer J. The mucopolysaccharidoses. In: Scriver CR, Beaudet al, Sly WS, Valle D (eds). Metabolic Basis of Inherited Disease, 7th edn. McGraw Hill: New York, 1995, pp 2265-2494.
5. Vogler C et al. A murine model of mucopolysaccharidosis type VII Characterization of a mouse with β-glucuronidase deficiency. Am J Pathol 1990; 136:207-217.
6. Birkenmeier EH et al. Increased life span and correction of metabolic defects in murine MPS VII following syngeneic bone marrow transplantation. Blood 1991; 78: 3081-3092.
7. Sands MS et al. Treatment of murine mucopolysaccharidosis type VII by syngeneic bone marrow transplantation in neonates. Lab Invest 1993; 68:676-686.
8. Sands M et al. Enzyme replacement therapy for murine mucopolysaccharidosis type VII. J Clin Invest 1994; 93: 2324-2331.
9. Wolfe JH et al. Reversal of pathology in murine mucopolysaccharidosis type VII by somatic cell gene transfer. Nature 1992; 360:749-753.
10. Moullier P, Bohl D, Heard JM, Danos O. Correction of lysosomal storage in the liver and spleen of MPS VII mice by implantation of genetically modified skin fibroblasts. Nat Genet 1993; 4:154-159.
11. Marechal V, Naffakh N, Danos O, Heard JM. Disappearance of lysosomal storage in spleen and liver of mucopolysaccharidosis VII mice after transplantation of genetically modified bone marrow cells. Blood 1993; 82: 1358-1365.
12. Flake AW, Harrison MR, Adzick NS, Zanjani ED. Transplantation of fetal hematopoietic stem cells in utero: the creation of hematopoietic chimeras. Science 1986; 233:776-778
13. Zanjani ED et al. Engraftment and long term expression of human fetal hematopoietic stem cells in sheep following transplantation in utero. J Clin Invest 1992; 89: 1178-1188.
14. Fleischman RA, Mintz B. Prevention of genetic anemias in mice by microinjection of normal hematopoietic stem cells into the fetal placenta. Proc Natl Acad Sci USA 1979; 76: 5736-5740.
15. Barker JE, Wolfe JH, Rowe LB, Birkenmeier EH. Advantages of gradient vs 5-fluorourcil enrichment of stem cells for retroviral-mediated gene transfer. Exp Hematol 1993; 21:47-54.
16. Wolfe JH et al. Restoration of normal lysosomal function in mucopolysaccharidosis type VII cells by retroviral vector-mediated gene transfer. Proc Natl Acad Sci USA 1990; 87: 2877-2881.
17. Parente MK, Wolfe JH. Production of increased titer retroviral vectors from stable producer cell lines by superinfection and concentration. Gene Therapy 1996; 3:756-760.
18. Taylor RM, Wolfe JH. Cross-correction of β-glucuronidase deficiency by retroviral vector-mediated gene transfer. Exp Cell Res 1994; 214:606-613.
19. Germeraad WTV et al. Efficient retrovirus-mediated gene transduction into murine hematopoietic stem cells and long-lasting expression using a transwell coculture system. Blood 1994; 84:780-788.
20. Markowitz D, Goff S, Bank A. A safe packaging line for gene transfer: separating viral genes on two different plasmids. J Virol 1988; 62: 1120-1124.
21. Markowitz D, Goff S, Bank A. Construction and use of a safe and efficient amphotrophic packaging cell line. Virol 1988; 167:400-406.
22. Neufeld EF, Fratantoni JC. Inborn errors of mucopolysaccharide metabolism. Science 1970; 169:141-146.
23. Richardson C, Ward M, Podda S, Bank A. Mouse fetal liver cells lack functional amphotropic retroviral receptors. Blood 1994; 84:433-439.
24. Casal ML, Haskins ME, Wolfe JH In vitro retroviral vector-mediated transfer of the rat beta-glucuronidase cDNA into canine fetal liver cells and weanling MPS VII bone marrow cells. In: Abraham NG et al. (eds). Molecular Biology of Hematopoiesis. Plenum Press: New York, 1996, pp 331-337.
25. Matzner U, Von Figura K, Pohlmann R, Expression of the two mannose 6-phosphate receptors is spatially and temporally different during mouse embryogenesis. Development 1992; 114:965-972.
26. Bodine DM, Karlsson S, Nienhuis AW. Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sci USA 1989; 86: 8897-8901.
27. Wolfe JH, Sands MS. Murine mucopolysaccharidosis type VII: a model system for gene therapy of the central nervous system. In: P Lowenstein, L Enquist (eds). Protocols for Gene Transfer into Neurones: Towards Gene Therapy of Neurologic Disorders. Wiley: Sussex, pp 263-274.
28. Wolfe JH et al. High level expression and export of β-glucuronidase from murine mucopolysaccharidosis VII cells corrected by a double-copy retrovirus vector. Gene Therapy 1995; 2:70-78.