Efficient Transduction and Engraftment of G-CSF-Mobilized Peripheral Blood CD34+ Cells in Nonhuman Primates Using GALV-Pseudotyped Gammaretroviral Vectors

Molecular Therapy

Volumn 14, Issue 2, 2006, Pages 212-217

  Beard, Brian C ^a   Mezquita, Pau ^a   Morris, Julia C ^a   Kiem, Hans Peter ^a,b  

^a Fred Hutchinson Cancer Research Center   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

Author keywords

cytokines; gene therapy; mobilization; nonhuman primates

Indexed keywords

CD34 ANTIGEN; RECOMBINANT GRANULOCYTE COLONY STIMULATING FACTOR; RETROVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; BABOON; CELL POPULATION; CONTROLLED STUDY; DNA MODIFICATION; GENE TRANSFER; GENETIC TRANSDUCTION; GIBBON LEUKEMIA VIRUS; GRANULOCYTE; NONHUMAN; PERIPHERAL BLOOD STEM CELL; PRIMATE; STEM CELL GENE THERAPY; STEM CELL MOBILIZATION; TARGET CELL;

ANIMALS; ANTIGENS, CD34; FLOW CYTOMETRY; GENE TRANSFER TECHNIQUES; GRANULOCYTE COLONY STIMULATING FACTOR, RECOMBINANT; HEMATOPOIESIS; HEMATOPOIETIC STEM CELL MOBILIZATION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMANS; LEUKEMIA VIRUS, GIBBON APE; PAPIO; SODIUM-PHOSPHATE COTRANSPORTER PROTEINS, TYPE III; TRANSDUCTION, GENETIC;

ANIMALIA; GIBBON APE LEUKEMIA VIRUS; MACACA MULATTA; PAPIO; PRIMATES;

EID: 33745328918     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2006.01.016     Document Type: Article

References (28)

1. Kohn D.B. The current status of gene therapy using hematopoietic stem cells. Curr. Opin. Pediatr. 7 (1995) 56-63
2. Amos T.A., and Gordon M.Y. Sources of human hematopoietic stem cells for transplantation-a review. Cell Transplant. 4 (1995) 547-569
3. Horn P.A., Morris J.C., Neff T., and Kiem H.-P. Stem cell gene transfer-Efficacy and safety in large animals studies. Mol. Ther. 10 (2004) 417-431
4. Broxmeyer H.E. Cord blood as an alternative source for stem and progenitor cell transplantation. Curr. Opin. Pediatr. 7 (1995) 47-55
5. + progenitors using clinically feasible methods. Mol. Ther. 1 (2000) 285-293
6. Horn P.A., et al. Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood 103 (2004) 3710-3716
7. Neff T., et al. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J. Clin. Invest. 112 (2003) 1581-1588
8. Dunbar C.E., et al. Improved retroviral gene transfer into murine and rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor. Proc. Natl. Acad. Sci. USA 93 (1996) 11871-11876
9. Costa J.J., et al. Recombinant human stem cell factor (kit ligand) promotes human mast cell and melanocyte hyperplasia and functional activation in vivo. J. Exp. Med. 183 (1996) 2681-2686
10. Dunbar C.E., et al. Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85 (1995) 3048-3057
11. Malech H.L., et al. Prolonged production of NADPH oxidase-corrected granulocytes after gene therapy of chronic granulomatous disease. Proc. Natl. Acad. Sci. USA 94 (1997) 12133-12138
12. Liu J.M., et al. Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC). Hum. Gene Ther. 10 (1999) 2337-2346
13. Abonour R., et al. Efficient retrovirus-mediated transfer of the multidrug resistance 1 gene into autologous human long-term repopulating hematopoietic stem cells. Nat. Med. 6 (2000) 652-658
14. + cells is superior to G-CSF or G-CSF + Flt3-L-mobilized cells in nonhuman primates. Blood 101 (2003) 2199-2205
15. Andrews R.G., et al. The ligand for c-kit, stem cell factor, stimulates the circulation of cells that engraft lethally irradiated baboons. Blood 80 (1992) 2715-2720
16. Andrews R.G., et al. In vivo synergy between recombinant human stem cell factor and recombinant human granulocyte colony-stimulating factor in baboons: enhanced circulation of progenitor cells. Blood 84 (1994) 800-810
17. Andrews R.G., Briddell R.A., Knitter G.H., Rowley S.D., Appelbaum F.R., and McNiece I.K. Rapid engraftment by peripheral blood progenitor cells mobilized by recombinant human stem cell factor and recombinant human granulocyte colony-stimulating factor in nonhuman primates. Blood 85 (1995) 15-20
18. Kiem H.-P., 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 90 (1997) 4638-4645
19. Bauer Jr. T.R., Miller A.D., and Hickstein D.D. Improved transfer of the leukocyte integrin CD18 subunit into hematopoietic cell lines using retroviral vectors having a gibbon ape leukemia virus envelope. Blood 86 (1995) 2379-2387
20. Kavanaugh M.P., 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 91 (1994) 7071-7075
21. Orlic D., Girard L.J., Jordan C.T., Anderson S.M., Cline A.P., and Bodine D.M. 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 (1996) 11097-11102
22. Sabatino D.E., et al. Amphotropic or gibbon ape leukemia virus (GALV) retrovirus binding and transduction correlates with the level of receptor mRNA in human hematopoietic cell lines. Blood Cells Mol. Dis. 23 (1997) 422-433
23. Kiem H.-P., et al. Improved gene transfer into baboon marrow repopulating cells using recombinant human fibronectin fragment CH-296 in combination with interleukin-6, stem cell factor, FLT-3 ligand, and megakaryocyte growth and development factor. Blood 92 (1998) 1878-1886
24. Wu X., Li Y., Crise B., and Burgess S.M. Transcription start regions in the human genome are favored targets for MLV integration. Science 300 (2003) 1749-1751
25. Schmidt M., et al. Polyclonal long-term repopulating stem cell clones in a primate model. Blood 100 (2002) 2737-2743
26. Schmidt M., et al. Clonality analysis after retroviral-mediated gene transfer to CD34(+) cells from the cord blood of ADA-deficient SCID neonates. Nat. Med. 9 (2003) 463-468
27. Neff T., Beard B.C., Peterson L.J., Anandakumar P., Thompson J., and Kiem H.-P. Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy. Blood 105 (2005) 997-1002
28. de Revel T., Appelbaum F.R., Storb R., Schuening F., Nash R., Deeg J., et al. Effects of granulocyte colony-stimulating factor and stem cell factor, alone and in combination, on the mobilization of peripheral blood cells that engraft lethally irradiated dogs. Blood 83 (1994 (Jun. 15)) 3795-3799