Long-term transfer and expression of the human β-globin gene in a mouse transplant model

Blood

Volumn 90, Issue 9, 1997, Pages 3414-3422

  Raftopoulos, Harry ^b   Ward, Maureen ^b   Leboulch, Philippe ^b   Bank, Arthur ^a  

^a Och Spine at New York Presbyterian Hospitals   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; FETUS; GENE EXPRESSION; GENE TRANSFER; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL; SOUTHERN BLOTTING; STEM CELL TRANSPLANTATION;

EID: 0030836297     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v90.9.3414     Document Type: Article

References (32)

1. Podda S, Ward M, Himelstein A, Richardson C, de la Flor-Weiss E, Smith L, Gottesman M, Pastan I, Bank A: Transfer and expression of the human multiple drug resistance gene into live mice. Proc Natl Acad Sci USA 89:9676, 1992
2. Sorrentino BP, Brandt SJ, Bodino G, Gottesman M, Pastan I, Cline A, Nienhuis AW: Selection of drug-resistant bone marrow cells in vivo after retroviral transfer of human MDR1. Science 257:99, 1992
3. Bank A: Human somatic cell gene therapy. Bioessays 18:999, 1996
4. Brenner MK: Gene transfer to hematopoietic cells. N Eng J Med 335:337, 1996
5. Karlsson S, Bodine DM, Perry L, Papayannopoulou T, Nienhuis A: Expression of the human β globin gene following retroviral-mediated transfer into multipotential hematopoietic progenitors of mice. Proc Natl Acad Sci USA 85:6062, 1988
6. Dzierzak EA, Papayannopoulou T, Mulligan RC: Lineage-specific expression of a human beta-globin gene in murine bone marrow transplant recipients reconstituted with retro virus-transduced stem cells. Nature 331:35, 1988
7. Bender MA, Gelinas RE, Miller AD: A majority of mice show long-term expression of a human beta-globin gene after retrovirus transfer into hematopoietic stem cells. Mol Cell Biol 9:1426, 1989
8. Tuan D, Solomon W, Li O, London IM: The "beta-like-globin" gene domain in human erythroid cells. Proc Natl Acad Sci USA 82:6384, 1985
9. Grosveld F, van Assendelft GB, Greaves DR, Kollias G: Position-independent, high-level expression of the human beta-globin gene in transgenic mice. Cell 51:975, 1987
10. Forrester WC, Novak U, Gelinas R, Groudine M: Molecular analysis of the human beta-globin locus activation region. Proc Natl Acad Sci USA 86:5439, 1989
11. Strouboulis J, Dillon N, Grosveld F: Developmental regulation of a complete 70-kb beta globin locus in transgenic mice. Genes Dev 6:1857, 1992
12. Tuan DYH, Solomon WB, London IM, Lee DP: An erythroid-specific, developmental-stage-independent enhancer far upstream of the human "beta-like globin" genes. Proc Natl Acad Sci USA 86:2554, 1989
13. Novak U, Harris E, Forrester W, Groudine M, Gelinas R: High-level β globin expression after retroviral transfer of locus activation region-containing human β globin gene derivatives into murine erythroleukemia cells. Proc Natl Acad Sci USA 67:3386, 1990
14. Plavec I, Papayannopoulou T, Maury C, Meyer F: A human beta-globin gene fused to the human beta-globin locus control region is expressed at high levels in erythroid cells of mice engrafted with retrovirus-transduced hematopoietic stem cells. Blood 81:1384, 1993
15. Leboulch P, Huang GM, Humphries RK, Oh YH, Eaves CJ, Tuan DY, London IM: Mutagenesis of retroviral vectors transducing human beta-globin gene and beta-globin locus control region derivatives results in stable transmission of an active transcriptional structure. EMBO J 13:3065, 1994
16. Sadelain M, Wang CH, Antoniou M, Grosveld F, Mulligan RC: Generation of a high-titer retroviral vector capable of expressing high levels of the human beta-globin gene. Proc Natl Acad Sci USA 92:6728, 1995
17. Takekoshi KJ, Oh YH, Westerman KW, London IM, Leboulch P: Retroviral transfer of a human beta-globin/delta-globin hybrid gene linked to beta locus control region hypersensitive site 2 aimed at the gene therapy of sickle cell disease. Proc Natl Acad Sci USA 92:3014, 1995
18. Miller AD, Rosman GJ: Improved retroviral vectors for gene transfer and expression. Biotechniques 7:980, 1989
19. Miller AD, Bender MA, Harris EAS, Kaleko M, Gelinas RE: Design of retrovirus vectors for transfer and expression of the human β globin gene. J Virol 62:4337, 1988
20. Markowitz D, Goff S, Bank A: A safe packaging line for gene transfer: Separating viral genes on two different plasmids. J Virol 62:1120, 1988
21. Wigler M, Pellicer A, Silverstein S, Axel R, Urlaub G, Chasin L: DNA-mediated transfer of the adenine phosphoribosyltransferase locus into mammalian cells. Proc Natl Acad Sci USA 76:1373, 1979
22. Markowitz D, Goff S, Bank A: Construction and use of a safe and efficient amphotropic packaging cell line. Virology 167:400, 1988
23. Richardson C, Ward M, Podda S, Bank A: Mouse fetal liver cells lack functional amphotropic retroviral receptors. Blood 84:433, 1994
24. Richardson C, Bank A: Preselection of transduced murine hematopoietic stem cell populations leads to increased long-term stability and expression of the human multiple drug resistance gene. Blood 86:2579, 1995
25. Hughes PF, Eaves CJ, Hogge DE, Humphries RK: High-efficiency gene transfer to human hematopoietic cells maintained in long-term marrow culture [see comments]. Blood 74:1915, 1989
26. Van XQ, Briddell R, Hartley C, Stoney G, Samal B, McNiece I: Mobilization of long-term hematopoietic reconstituting cells in mice by the combination of stem cell factor plus granulocyte colony-stimulating factor. Blood 84:795, 1994
27. O'Neill D, Yang J, Bornschlegel K, Bank A: Delayed human gamma- to beta-globin gene switching in a transgenic mouse line by deletion of a nuclear protein binding site. Blood 86:7a, 1995 (abstr, suppl 1)
28. Bank A, Dow LW, Farace MG, O'Donnell JV, Ford S, Natta C: Changes in globin synthesis with erythroid cell maturation in sickle thalassemia. Blood 41:353, 1973
29. Platt OS: The sickle syndromes, in Handin R, Lux S, Stossel T (eds): Blood: Principles and Practice of Hematology. Philadelphia, PA, Lippincott, 1995, p 1645
30. Peterson KR, Li QL, Clegg CH, Furukawa T, Navas PA, Norton EJ, Kimbrough TG, Stamatoyannopoulos G: Use of yeast artificial chromosomes (YACs) in studies of mammalian development: Production of beta locus YAC mice carrying human globin developmental mutants. Proc Natl Acad Sci USA 92:5655, 1995
31. Ellis J, Tan-Un KC, Harper A, Michalovich D, Yannoutsos N, Philipsen S, Grosveld F: A dominant chromatin-opening activity in 5′ hypersensitive site 3 of the human beta-globin locus control region. EMBO J 15:562, 1996
32. Pawliuk R, Kay R, Lansdorp P, Humphries RK: Selection of retrovirally transduced hematopoietic cells using CD24 as a marker of gene transfer. Blood 84:2868, 1994