Direct comparison of RD114-pseudotyped versus amphotropic-pseudotyped retroviral vectors for transduction of rhesus macaque long-term repopulating cells

Molecular Therapy

Volumn 8, Issue 4, 2003, Pages 611-617

  Hu, Jiong ^a   Kelly, Patrick ^b   Bonifacino, Aylin ^a   Agricola, Brian ^a   Donahue, Robert ^a   Vanin, Elio ^b   Dunbar, Cynthia E ^a  

^a NATIONAL HEART LUNG AND BLOOD INSTITUTE   (United States)

^b ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CD34 ANTIGEN; FLT3 LIGAND; GRANULOCYTE COLONY STIMULATING FACTOR; MEGAKARYOCYTE GROWTH AND DEVELOPMENT FACTOR; RETROVIRUS VECTOR; VIRUS ENVELOPE PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CELL CULTURE; CELL POPULATION; CONTROLLED STUDY; GENE THERAPY; GENETIC TRANSDUCTION; GRANULOCYTE; HEMATOPOIESIS; INTERMETHOD COMPARISON; MONONUCLEAR CELL; NONHUMAN; POLYMERASE CHAIN REACTION; RHESUS MONKEY; SOUTHERN BLOTTING; STEM CELL TRANSPLANTATION; WHOLE BODY RADIATION;

ANIMALIA; FELIDAE; MACACA MULATTA; UNIDENTIFIED RETROVIRUS;

EID: 0142150965     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1525-0016(03)00239-9     Document Type: Article

References (33)

1. Donahue, R. E., and Dunbar, C. E. (2001). An update on the use of non-human primate models for preclinical testing of gene therapy approaches targeting hematopoietic cells. Hum. Gene Ther. 12: 607-617.
2. Rosenzweig, M., et al. (1999). Efficient and durable gene marking of hematopoietic progenitor cells in nonhuman primates after nonablative conditioning. Blood 94: 2271-2286.
3. Moritz, T., et al. (1996). Fibronectin improves transduction of reconstituting hematopoietic stem cells by retroviral vectors: evidence of direct viral binding to chymotryptic carboxy-terminal fragments. Blood 88: 855-862.
4. + progenitors using clinically feasible methods. Mol. Ther. 1: 285-293.
5. Kiem, H. P., et al. (1998). 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: 1878-1886.
6. Kurre, P., et al. (2001). Gene transfer into baboon repopulating cells: a comparison of Flt-3 ligand and megakaryocyte growth and development factor versus IL-3 during ex vivo transduction. Mol. Ther. 3: 920-927.
7. Cavazzana-Calvo, M., et al. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
8. Aiuti, A., et al. (2002). Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloalalative conditioning. Science 296: 2410-2413.
9. Kavanaugh, M. P., et al. (1994). Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters. Proc. Natl. Acad. Sci. USA 91: 7071-7075.
10. Orlic, D., et al. (1996). 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: 11097-11102.
11. Orlic, D., et al. (1998). Identification of human and mouse hematopoietic stem cell populations expressing high levels of mRNA encoding retrovirus receptors. Blood 91: 3247-3254.
12. Relander, T., Karlsson, S., and Richter, J. (2002). Oncoretroviral gene transfer to NOD/SCID repopulating cells using three different viral envelopes. J. Gene Med. 4: 122-132.
13. Tailor, C. S., Nouri, A., Zhao, Y., Takeuchi, Y., and Kabat, D. J. (1999). A sodium-dependent neutral-amino-acid transporter mediates infections of feline and baboon endogenous retroviruses and simian type D retroviruses. Virology 73: 4470-4474.
14. Kelly, P. F., Vandergriff, J., Nathwani, A., Nienhuis, A. W., and Vanin, E. F. (2000). 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 96: 1206-1214.
15. Kelly, P. F., Carrington, J., Nathwani, A., and Vanin, E. F. (2001). RD114-pseudotyped oncoretroviral vectors. Biological and physical properties. Ann. N. Y. Acad. Sci. 938: 262-276.
16. Kelly, P. F., et al. (2003). Prolonged multilineage clonal hematopoiesis in a rhesus recipient of CD34-positive cells marked with a RD114 pseudotyped oncoretroviral vector. Blood Cells Mol. Dis. 30: 132-143.
17. Goerner, M., et al. (2001). Sustained multilineage gene persistence and expression in dogs transplanted with CD34(+) marrow cells transduced by RD114-pseudotype oncoretrovirus vectors. Blood 98: 2065-2070.
18. Gatlin J, Melkus M. W, Padgett A, Kelly P. F, Garcia J. V (2001): Engraftment of NOD/SCID mice with human CD34(+) cells transduced by concentrated oncoretroviral vector particles pseudotyped with the feline endogenous retrovirus (RD114) envelope protein. J. Virol 75: 9995-9999.
19. Takatoku, M., et al. (2001). Avoidance of stimulation improves engraftment of cultured and retrovirally transduced hematopoietic cells in primates. J. Clin. Invest. 108: 447-455.
20. Persons, D. A., et al. (1998). Use of the green fluorescent protein as a marker to identify and track genetically modified hematopoietic cells. Nat. Med. 4: 1201-1205.
21. Dunbar, C. E., et al. (1995). Retrovirally-marked CD34-enriched peripheral blood and bone marrow and cells contribute to long-term engraftment after autologous transplantation. Blood 85: 3048-3057.
22. + cells present in autologous transplants of chronic myelogenous leukemia (CML) contribute to relapse after autologous bone marrow transplantation in CML. Blood 83: 3068-3076.
23. + blood cells is superior to G-CSF alone or G-CSF-flt3L mobilized cells. Blood 101: 2199-2205.
24. Bordignon, C., et al. (1995). Gene therapy in peripheral blood lymphocytes and bone marrow for ADA-immunodeficient patients. Science 270: 470-475.
25. + cells in ADA-deficient SCID neonates. Nat. Med. 4: 775-780.
26. Liu, J. M., et al. (1999). Engraftment of hematopoietic progenitor cells transduced with the Fanconi anemia group C gene (FANCC). Hum. Gene Ther. 10: 2337-2346.
27. Burns, J. C., Friedmann, T., Driever, W., Burrascano, M., and Yee, J. K. (1993). 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. USA 90: 8033-8037.
28. Hanawa, H., et al. (2002). Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol. Ther. 5: 242-251.
29. Kiem, H. P., et al. (1997). 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: 4638-4645.
30. + cells in Thy-1 expression and cell cycle status in nonhuman primates mobilized or non mobilized with granulocyte colony-stimulating factor and/or stem cell factor. Blood 87: 1644-1653.
31. + cells for six hours without growth factors or on autologous stroma does not improve marking efficiency assessed in vivo. Blood 89: 4040-4046.
32. Nienhuis, A. W., et al. (1987). Recombinant human granulocyte-macrophage colony-stimulating factor (GM-CSF) shortens the period of neutropenia after autologous bone marrow transplantation in a primate model. J. Clin. Invest. 80: 573-577.
33. Cassel, A., Cottler-Fox, M., Doren, S., and Dunbar, C. E. (1993). Retroviral-mediated gene transfer into CD34-enriched human peripheral blood stem cells. Exp. Hematol. 21: 585-591.