High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum-free conditions

Blood

Volumn 92, Issue 9, 1998, Pages 3163-3171

  Schilz, Andrea J ^b   Brouns, Gaby ^b   Knöß, Heike ^b   Ottmann, Oliver G ^b   Hoelzer, Dieter ^b   Fauser, Axel A ^b   Thrasher, Adrian J ^b   Grez, Manuel ^a  

^a INSTITUTE FOR TUMOR BIOLOGY AND EXPERIMENTAL THERAPY   (Germany)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

NERVE GROWTH FACTOR RECEPTOR;

ARTICLE; CLONING VECTOR; COMBINED IMMUNODEFICIENCY; GENE TRANSFER; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; MARKER GENE; PRIORITY JOURNAL; RETROVIRUS;

EID: 0032213335     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v92.9.3163     Document Type: Article

References (63)

1. Hawley RG, Fong AZC, Burns BF, Hawley TS: Transplantable myeloproliferative disease induced in mice by an interleukin 6 retrovirus. J Exp Med 176:1149, 1992
2. Ding C, Kume A, Björgvinsdóttir H, Hawley RG, Pech N, Dinauer MC: High-level reconstitution of respiratory burst activity in a human X-linked chronic granulomatous disease (X-CGD) cell line and correction of murine X-CGD bone marow cells by retroviral mediated gene transfer of human gp91-phox. Blood 88:1834, 1996
3. Pawliuk R, Eaves CJ, Humphries RK: Sustained high-level reconstitution of the hematopoietic system by preselected hematopoietic cells expressing a transduced cell-surface antigen. Hum Gene Ther 8:1595, 1997
4. Brenner MK, Hill DR, Holladay MS, Heslop HE, Moen RC, Buschle M, Krance RA, Santana VM, Anderson FW, Ihle JN: Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342:1134, 1993
5. + bone marrow cells. Blood 82:1975, 1993
6. + cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow in CML. Blood 83:3068, 1994
7. Kiem HP, Darovsky B, von Kalle C, Goehle S, Stewart D, Graham T, Hackman R, Appelbaurn FR, Deeg HJ, Miller AD, Storb R, Schuening FG: Retro virus-mediated gene transduction into canine peripheral blood repopulating cells. Blood 83:1467, 1994
8. + hematopoietic cell transduction with cell-free retroviral vector preparations. Proc Natl Acad Sci USA 92:4372, 1995
9. Dunbar CE, Cottler-Fox M, O'Shaughnessy JA, Doren S, Carter C, Berenson R, Brown S, Moen RC, Greenblat J, Stewart FM, Leitman SF, Wilson WH, Cowan K, Young NS, Nienhuis AW: Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation Blood 85:3048, 1995
10. Bordignon C, Notarangelo LD, Nobili N, Ferrari G, Casorati G, Panina P, Mazzolari E, Maggiori D, Rossi C, Servida P, Ugazio AG, Mavilio F: Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270:470, 1995
11. Kohn DB, Winberg KI, Nolta JA, Heiss LN, Lenarsky C, Crooks GM, Hanley ME, Annett G, Brooks JS, El-Khoureiy A, Lawrence K, Wells S, Moen RC, Bastian J, William-Herman DE, Elder M, Wara D, Bowen T, Hershfield MS, Mullen CA, Blease RM, Parkman R: Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med 1:1017, 1995
12. Malech HL, Maples PB, Whiting-Theobald N, Union GF, Sekhsaria S, Vowells SJ, Li F, Miller JA, DeCarlo E, Holland SM, Leitman SF, Carter CS, Butz RE, Reads EJ, Fleischer TA, Schneiderman RD, Van Epps DE, Spratt SK, Maack CA, Rokovich JA, Cohen LK, Gallin JI: Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc Natl Acad Sci USA 94:12133, 1997
13. Kiem HP, Heyward S, Winkler A, Potter J, Allen JM, Miller AD, Andrews RG: Gene transfer into marrow repopulating cells: Comparison between amphotropic and gibbon ape leukemia virus pseudotyped retroviral vectors in a competitive repopulation assay in baboons. Blood 90:4638, 1997
14. Miller DG, Adam MA, Miller AD: Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10:4239, 1990
15. Hao QL, Thiemann FT, Petersen D, Smogorzewska EM, Crooks GM: Extended long-term culture reveals a highly quiescent and primitive human hematopoietic progenitor population. Blood 88:3306, 1996
16. + bone marrow progenitor cells. Blood 82:3290, 1993
17. Orlic D, Girard LJ, Jordan CT, Anderson SM, Cline AP, Bodine DM: The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc Natl Acad Sci USA 93:11097, 1996
18. Kamel-Reid S, Dick JE: Engraftment of immune-deficient mice with human hematopoietic stem cells. Science 242:1706, 1988
19. McCune JM, Namikawa R, Kaneshima H, Shultz LD, Lieberman M, Weissman IL: The SCID-hu Mouse: Murine model for the analysis of human hematolymphoid differentiation and function. Science 241:1632, 1988
20. Namikawa R, Weilbaecher KN, Kaneshima H, Yee EJ, McCune JM: Long-term human hematopoiesis in the SCID-hu mouse. J Exp Med 172:1055, 1990
21. Lapidot T, Pflumio F, Doedens M, Murdoch B, Williams DE, Dick JE: Cytokine stimulation of multilineage hematopoiesis from immature human cells engraftet in SCID mice. Science 255:1137, 1992
22. Nolta JA, Hanley MB, Kohn DB: Sustained human hematopoiesis in immundeficient mice by cotransplantation of marrow stroma expressing human IL-3: Analysis of gene transduction of long-lived progenitors. Blood 83:3041, 1994
23. Larochelle A, Vormoor J, Hanenberg H, Wang JCY, Bhatia M, Lapidot T, Moritz T, Murdoch B, Xiao XL, Kato I, Williams DA, Dick JE: Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: Implications for gene therapy. Nat Med 2:13329, 1996
24. Nolta JA, Dao MA, Wells S, Smogorzewska EM, Kohn DB: Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice. Proc Natl Acad Sci USA 93:2414, 1996
25. Vormoor J, Lapidot T, Pflumio F, Risdon G, Patterson B, Broxmeyer HE, Dick JE: Immature human cord blood progenitors engraft and proliferate to high levels in severe combined immunodeficient mice. Blood 83:2489, 1994
26. Bhatia M, Wang JCY, Kapp U, Bonnet D, Dick JE: Purification of primitive human hematopoietic cells capable of repopulating immunedeficient mice. Proc Natl Acad Sci USA 94:5320, 1997
27. Cashman JD, Lapidot T, Wang JCY, Doedens M, Shultz LD, Lansdorp P, Dick JE, Eaves CJ: Kinetic evidence of the regeneration of multilineage hematopoiesis from primitive cells in normal human bone marrow transplantated into immunodeficient mice. Blood 89:4307, 1997
28. +- enriched cells from umbilical cord blood in NOD/LtSz-scid/scid mice. Blood 90:85, 1997
29. Can OI, Murdoch B, Larochelle A, Dick JE: Differential maintenance of primitive SCID-repopulating cells, clonogenic progenitors, and long term culture initiating cells after incubation on human bone marrow stromal cells. Blood 90:641, 1997
30. + progenitors to sustain long-term hematopoiesis in immune-deficient mice after ex vivo retroviral- mediated transduction. Blood 89:446, 1997
31. Kotani H, Newton PB, Zhang S, Chiang YL, Otto E, Weaver L, Blaese RM, Anderson WF, McGarrity GJ: Improved methods of retroviral vector transduction and production for gene therapy. Hum Gene Ther 5:19, 1994
32. Breems DA, Blockland EAW, Neben S, Ploemacher RE: Frequency analysis of human primitive hematopoietic stem cell subsets using a cobblestone area forming cell assay. Leukemia 8:1095, 1994
33. von Kalle C, Kiem HP, Goehle S, Darovsky B, Heimfeld S, Torok-Storb B, Storb R, Schuening FG: Increased gene transfer into human hematopoietic progenitor cells by extended in vitro exposure to a pseudotyped retroviral vector. Blood 84:2890, 1994
34. +/CD381o human bone marrow cells reselected after long-term exposure to GALV-pseudotyped retroviral vector. Hum Gene Ther 8:2079, 1997
35. Bauer TR, Miller AD, Hickstein DD: Improved transfer of the leukocyte integration CD18 subunit into hematopoietic cell lines by using retroviral vectors having a gibbon ape leukemia virus envelope. Blood 86:2379, 1995
36. Mavilio F, Ferrari G, Rossini S, Nobili N, Bonini C, Casorati G, Traversari C, Bordignon C: Peripheral blood lymphocytes as target cells of retroviral vector-mediated gene transfer. Blood 83:1988, 1994
37. Valtieri M, Schiro R, Chelucci C, Masella B, Testa U, Casella I, Montesoro E, Mariani G, Hassan HJ, Peschle C: Efficient transfer of selectable and membrane reporter genes in hematopoietic progenitor and stem cells purified from human peripheral blood. Cancer Res 54:4398, 1994
38. + cells transduced with retroviral vectors containing an intracytoplasmatically truncated version of the human low affinity nerve growth fact receptor (ΔLNGFR) gene. Hum Gene Ther 8:1815, 1997
39. +-restricted phenotypes of human hematopoietic progenitor cells by retrovirus-mediated gene transfer. Hum Gene Ther 8:1611, 1997
40. Moritz T, Patel VP, Williams DA: Bone marrow extracellular matrix molecules improve gene transfer into human hematopoietic cells via retroviral vectors. J Clin Invest 93:1451, 1994
41. Hanenberg H, Xiao XL, Dillo D, Hashino K, Kato I, Williams DA: Co-localization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction mammalian cells. Nat Med 8:876, 1996
42. + hematopoietic cells. Hum Gene Ther 8:2193, 1997
43. Movassagh M, Desmyter, C, Baillou C, Chapel-Fernandes S, Guigon M, Klatzmann D, Lemoine FM: High level gene transfer to cord blood progenitors using gibbon ape leukemia virus pseudotype retroviral vectors and an improved clinically applicable protocol. Hum Gene Ther 9:225, 1998
44. - human hematopoietic progenitors assessed in immune-deficient mice. Blood 91:1243, 1998
45. Pflumio F, Izac B, Katz A, Shultz LD, Vainchenker W, Coulombel L: Phenotype and function of human hematopoietic cells engrafting immune-deficient CB17-severe combined immunodeficiency mice and nonobese diabetic-severe combined immunodeficiency mice after transplantation of human cord blood mononuclear cells. Blood 88:3731, 1996
46. Conneally E, Cashman J, Petzer A, Eaves C: Expansion in vitro of transplantable human cord blood stem cells demonstrated using a quantitative assay of their lympho-myeloid repopulating activity in nonobese diabetic-scid/scid mice. Proc Natl Acad Sci USA 94:9836, 1997
47. Sutherland HJ, Lansdorp PM, Henkelman DH, Eaves AC, Eaves CJ: Functional characterization of individual human hematopoietic stem cells cultured at limiting dilution on supportive marrow stroma cells. Proc Natl Acad Sci USA 87:3584, 1990
48. + mobilized peripheral blood stem cells. Blood 91:111, 1998
49. - cells and maintains progenitor cells in vitro. Blood 87:3563, 1996
50. + bone marrow progenitor cells in vitro. Blood 87:1317, 1996
51. Zandstra PW, Conneatly E, Petzer AL, Piret JM, Eaves CJ: Cytokine manipulation of primitive hematopoietic cell-self-renewal. Proc Natl Acad Sci USA 94:4698, 1997
52. - cell production in vitro. Br J Haematol 98:528, 1997
53. Haylock DN, Horsfall MJ, Dowse TL, Ramshaw HS, Niutta S, Protopsaltis S, Peng L, Burrell C, Rappold I, Buhring HJ, Simmons PJ: Increased recruitment of hematopoietic progenitor cells underlies the ex vivo expansion potential of FLT3 ligand. Blood 90:2260, 1997
54. Papayannopoulou T, Nakamoto B, Andrews RG, Lyman SD, Lee MY: In vivo effects of Flt3/Flk2 ligand on mobilization of hematopoietic progenitors in primates and potent synergistic enhancement with granulocyte colony-stimulating factor. Blood 90:620, 1997
55. Bhatia M, Bonnet D, Kapp U, Wang JCY, Murdoch B, Dick JE: Quantitative analysis reveals expansion of human hematopoietic repopulating cells after short-term ex vivo culture. J Exp Med 186:619, 1997
56. Sekhar M, Kotani H, Doren S, Agarwal R, McGarrity G, Dunbar CE: Retroviral transduction of CD34-enriched hematopoietic progenitor cells under serum-free conditions. Hum Gene Ther 7:33, 1996
57. Cashman JD, Eaves AC, Raines EW, Ross R, Eaves CJ: Mechanisms that regulate the cell cyclus status of very primitive hematopoietic cells in long-term human marrow cultures. I. Stimulatory role of a variety of mesenchymal cell activators and inhibitory role of TGF-beta. Blood 75:96, 1990
58. Eaves CJ, Cashman JD, Kay RJ, Dougherty GJ, Otsuka T, Gaboury LA, Hogge DE, Lansdorp PM, Eaves AC, Humphries RK: Mechanisms that regulate the cell cycle status of very primitive hematopoietic cells in long-term human marrow cultures. II. Analysis of positive and negative regulators produced by stromal cell swithin the adherent layer. Blood 78:110, 1991
59. Hatzfeld J, Li M, Brown EL, Sookdeo H, Levesque J, O'Toole T, Gurney C, Clark SC, Hatzfeld A: Release of early human hematopoietic progenitors from quiescence by antisense transforming growth factor-β1 oligonucleotides. J Exp Med 174:925, 1991
60. - human umbilical cord blood cells reveals their potentiality to engraft adults. Proc Natl Acad Sci USA 90:8707, 1993
61. Ploemacher RE, van Soest PL, Boudewijn A: Autocrine transforming growth factor β1 blocks colony formation and progenitor cell generation by hematopoietic stem cells stimulated with steel factor. Stem Cells 11:336, 1993
62. Soma T, Yu JM, Dunbar CE: Maintenance of murine long-term repopulating stem cells in ex vivo culture is affected by modulation of transforming growth factor-β but not macrophage inflammatory protein-1α activities. Blood 87:4561, 1996
63. Porter CD, Parkar MH, Collins MKL, Levinsky RJ, Kinon C: Efficient retroviral transduction of human bone marrow progenitor and long-term culture-initiating cells: Partial reconstitution of cells from patients with X-linked chronic granulomatous disease by gp91-phox expression. Blood 87:3722, 1996