Oncoretroviral gene transfer to NOD/SCID repopulating cells using three different viral envelopes

Journal of Gene Medicine

Volumn 4, Issue 2, 2002, Pages 122-132

  Relander, Thomas ^a   Karlsson, Stefan ^a   Richter, Johan ^a  

^a LUND UNIVERSITY HOSPITAL   (Sweden)

Author keywords

Amphotropic; CD34+; GALV; Gene transfer; NOD SCID; VSV G

Indexed keywords

GREEN FLUORESCENT PROTEIN; RETROVIRUS VECTOR; VIRUS ENVELOPE PROTEIN; ADENOSINE DIPHOSPHATE RIBOSYL CYCLASE; CD34 ANTIGEN; CD38 ANTIGEN; CD38 PROTEIN, HUMAN; CD38 PROTEIN, MOUSE; DIFFERENTIATION ANTIGEN; LEUKOCYTE ANTIGEN; MEMBRANE PROTEIN; NICOTINAMIDE ADENINE DINUCLEOTIDE NUCLEOSIDASE; PHOTOPROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BONE MARROW; CD34 SELECTION; CELL BASED GENE THERAPY; CELL POPULATION; CELL STIMULATION; CELL TRANSPLANTATION; CONTROLLED STUDY; CYTOKINE PRODUCTION; FLOW CYTOMETRY; FLUORESCENCE ACTIVATED CELL SORTING; GENE TRANSFER; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; IN VITRO STUDY; MOUSE; MOUSE MUTANT; NONHUMAN; PRIORITY JOURNAL; UMBILICAL CORD BLOOD; VIRAL GENE DELIVERY SYSTEM; VIRUS ENVELOPE; ANIMAL; BIOSYNTHESIS; BONE MARROW CELL; CELL SEPARATION; CULTURE MEDIUM; FETUS BLOOD; GENE VECTOR; GENETICS; METABOLISM; RETROVIRUS; TIME;

GIBBON APE LEUKEMIA VIRUS;

ADP-RIBOSYL CYCLASE; ANIMALS; ANTIGENS, CD; ANTIGENS, CD34; ANTIGENS, CD38; ANTIGENS, DIFFERENTIATION; BONE MARROW CELLS; CELL SEPARATION; CULTURE MEDIA, SERUM-FREE; FETAL BLOOD; FLOW CYTOMETRY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HUMANS; LUMINESCENT PROTEINS; MEMBRANE GLYCOPROTEINS; MICE; MICE, SCID; NAD+ NUCLEOSIDASE; RETROVIRIDAE; TIME FACTORS; VIRAL ENVELOPE PROTEINS;

EID: 0036518338     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/jgm.246     Document Type: Article

References (43)

1. Karlsson S. Treatment of genetic defects in hematopoietic cell function by gene transfer. Blood 1991; 78: 2481-2492.
2. 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 1990; 10: 4239-4242.
3. Richter J. Gene transfer to hematopoietic cells - the clinical experience. Eur J Haematol 1997; 59: 67-75.
4. Conneally E, Eaves CJ, Humphries RK. Efficient retroviral-mediated gene transfer to human cord blood stem cells with in vivo repopulating potential. Blood 1998; 91: 3487-3493.
5. Demaison C, Brouns G, Blundell MP, et al. A defined window for efficient gene marking of severe combined immunodeficient-repopulating cells using a gibbon ape leukemia virus-pseudotyped retroviral vector. Hum Gene Ther 2000; 11: 91-100.
6. Hennemann B, Conneally E, Pawliuk R, et al. Optimization of retroviral-mediated gene transfer to human NOD/SCID mouse repopulating cord blood cells through a systematic analysis of protocol variables. Exp Hematol 1999; 27: 817-825.
7. Schilz AJ, Brouns G, Knoss H, et al. High efficiency gene transfer to human hematopoietic SCID-repopulating cells under serum-free conditions. Blood 1998; 92: 3163-3171.
8. van Hennik PB, Verstegen MM, Bierhuizen MF, et al. Highly efficient transduction of the green fluorescent protein gene in human umbilical cord blood stem cells capable of cobblestone formation in long-term cultures and multilineage engraftment of immunodeficient mice. Blood 1998; 92: 4013-4022.
9. Marandin A, Dubart A, Pflumio F, et al. Retrovirus-mediated gene transfer into human CD34+38low primitive cells capable of reconstituting long-term cultures in vitro and nonobese diabetic-severe combined immunodeficiency mice in vivo. Hum Gene Ther 1998; 9: 1497-1511.
10. Ramsfjell V, Bryder D, Bjorgvinsdottir H, et al. Distinct requirements for optimal growth and in vitro expansion of human CD34(+)CD38(-) bone marrow long-term culture-initiating cells (LTC-IC), extended LTC-IC, and murine in vivo long-term reconstituting stem cells. Blood 1999; 94: 4093-4102.
11. Piacibello W, Sanavio F, Severino A, et al. Engraftment in nonobese diabetic severe combined immunodeficient mice of human CD34(+) cord blood cells after ex vivo expansion: evidence for the amplification and self-renewal of repopulating stem cells. Blood 1999; 93: 3736-3749.
12. Luens KM, Travis MA, Chen BP, et al. Thrombopoietin, kit ligand, and flk2/flt3 ligand together induce increased numbers of primitive hematopoietic progenitors from human CD34+Thy-1+Lin- cells with preserved ability to engraft SCID-hu bone. Blood 1998; 91: 1206-1215.
13. Murray L, Luens K, Tushinski R, et al. Optimization of retroviral gene transduction of mobilized primitive hematopoietic progenitors by using thrombopoietin, Flt3, and Kit ligands and RetroNectin culture. Hum Gene Ther 1999; 10: 1743-1752.
14. Kavanaugh MP, Miller DG, Zhang W, et al. Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters. Proc Natl Acad Sci USA 1994; 91: 7071-7075.
15. Chien ML, Foster JL, Douglas JL, et al. The amphotropic murine leukemia virus receptor gene encodes a 71-kilodalton protein that is induced by phosphate depletion. J Virol 1997; 71: 4564-4570.
16. Pedersen L, van Zeijl M, Johann SV, et al. Fungal phosphate transporter serves as a receptor backbone for gibbon ape leukemia virus. J Virol 1997; 71: 7619-7622.
17. Emi N, Friedmann T, Yee JK. Pseudotype formation of murine leukemia virus with the G protein of vesicular stomatitis virus. J Virol 1991; 65: 1202-1207.
18. Burns JC, Friedmann T, Driever W, et al. Vesicular stomatitis virus G glycoprotein pseudotyped retroviral vectors: concentration to very high titer and efficient gene transfer into mammalian and nonmammalian cells. Proc Natl Acad Sci U S A 1993; 90: 8033-8037.
19. Schlegel R, Tralka TS, Willingham MC, et al. Inhibition of VSV binding and infectivity by phosphatidylserine: is phosphatidyl-serine a VSV-binding site? Cell 1983; 32: 639-646.
20. von Kalle C, Kiem HP, Goehle S, et al. Increased gene transfer into human hematopoietic progenitor cells by extended in vitro exposure to a pseudotyped retroviral vector. Blood 1994; 84: 2890-2897.
21. von Laer D, Corovic A, Vogt B, et al. Amphotropic and VSV-G-pseudotyped retroviral vectors transduce human hematopoietic progenitor cells with similar efficiency. Bone Marrow Transplant 2000; 25 (Suppl 2): S75-S79.
22. Yam PY, Yee JK, Ito JI, et al. Comparison of amphotropic and pseudotyped VSV-G retroviral transduction in human CD34+ peripheral blood progenitor cells from adult donors with HIV-1 infection or cancer. Exp Hematol 1998; 26: 962-968.
23. Cheng L, Du C, Murray D, et al. A GFP reporter system to assess gene transfer and expression in human hematopoietic progenitor cells. Gene Ther 1997; 4: 1013-1022.
24. Relander T, Brun A, Hawley RG, et al. Retroviral transduction of human CD34+ cells on fibronectin fragment CH-296 is inhibited by high concentrations of virus containing medium. J. Gene Med 2001; 3: 207-218.
25. Gallardo HF, Tan C, Ory D, et al. Recombinant retroviruses pseudotyped with the vesicular stomatitis virus G glycoprotein mediate both stable gene transfer and pseudotransduction in human peripheral blood lymphocytes. Blood 1997; 90: 952-957.
26. Scarpa M, Cournoyer D, Muzny DM, et al. Characterization of recombinant helper retroviruses from Moloney-based vectors in ecotropic and amphotropic packaging cell lines. Virology 1991; 180: 849-852.
27. Taswell C. Limiting dilution assays for the determination of immunocompetent cell frequencies. I. Data analysis. J Immunol 1981; 126: 1614-1619.
28. Bhatia M, Wang JC, Kapp U, et al. Purification of primitive human hematopoietic cells capable of repopulating immune-deficient mice. Proc Natl Acad Sci USA 1997; 94: 5320-5325.
29. Conneally E, Cashman J, Petzer A, et al. 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 1997; 94: 9836-9841.
30. Ballen KK, Valinski H, Greiner D, et al. Variables to predict engraftment of umbilical cord blood into immunodeficient mice: usefulness of the non-obese diabetic-severe combined immunodeficient assay. Br J Haematol 2001; 114: 211-218.
31. MacNeill EC, Hanenberg H, Pollok KE, et al. Simultaneous infection with retroviruses pseudotyped with different envelope proteins bypasses viral receptor interference associated with colocalization of gp70 and target cells on fibronectin CH-296. J Virol 1999; 73: 3960-3967.
32. Bryder D, Jacobsen SE. Interleukin-3 supports expansion of long-term multilineage repopulating activity after multiple stem cell divisions in vitro. Blood 2000; 96: 1748-1755.
33. Bhatia M, Bonnet D, Wu D, et al. Bone morphogenetic proteins regulate the developmental program of human hematopoietic stem cells. J Exp Med 1999; 189: 1139-1148.
34. Mikkola H, Woods NB, Sjogren M, et al. Lentivirus gene transfer in routine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells. J Virol 2000; 74 11911-11918.
35. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000; 288: 669-672.
36. Kiem HP, Heyward S, Winkler A, et al. 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 1997; 90: 4638-4645.
37. Dorrell C, Gan OI, Pereira DS, et al. Expansion of human cord blood CD34(+)CD38(-) cells in ex vivo culture during retroviral transduction without a corresponding increase in SCID repopulating cell (SRC) frequency: dissociation of SRC phenotype and function. Blood 2000; 95: 102-110.
38. Novelli EM, Cheng L, Yang Y, et al. Ex vivo culture of cord blood CD34+ cells expands progenitor cell numbers, preserves engraftment capacity in nonobese diabetic/severe combined immunodeficient mice, and enhances retroviral transduction efficiency. Hum Gene Ther 1999; 10: 2927-2940.
39. Kelly PF, Vandergriff J, Nathwani A, et al. Highly efficient gene transfer into cord blood nonobese diabetic/severe combined immunodeficiency repopulating cells by oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. Blood 2000; 96: 1206-1214.
40. Larochelle A, Vormoor J, Hanenberg H, et al. Identification of primitive human hematopoietic cells capable of repopulating NOD/SCID mouse bone marrow: implications for gene therapy. Nature Med 1996; 2: 1329-1337.
41. Rebel VI, Tanaka M, Lee JS, et al. One-day ex vivo culture allows effective gene transfer into human nonobese diabetic/severe combined immune-deficient repopulating cells using high-titer vesicular stomatitis virus G protein pseudotyped retrovirus. Blood 1999; 93: 2217-2224.
42. Bhatia M, Bonnet D, Kapp U, et al. Quantitative analysis reveals expansion of human hematopoietic repopulating cells after short-term ex vivo culture. J Exp Med 1997; 186: 619-624.
43. Ueda T, Tsuji K, Yoshino H, et al. Expansion of human NOD/SCID-repopulating cells by stem cell factor, Flk2/Flt3 ligand, thrombopoietin, IL-6, and soluble IL-6 receptor. J Clin Invest 2000; 105: 1013-1021.