Lentivirus-mediated gene transfer into hematopoietic repopulating cells in baboons

Gene Therapy

Volumn 9, Issue 21, 2002, Pages 1464-1471

  Horn, P A ^a   Morris, J C ^a   Bukovsky, A A ^a   Andrews, R G ^a,b,c   Naldini, L ^d   Kurre, P ^a,b   Kiem, H P ^a,b,c  

^a Fred Hutchinson Cancer Research Center   (United States)

^b UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^c UNIVERSITY OF WASHINGTON   (United States)

^d UNIVERSITY OF TURIN   (Italy)

Author keywords

Gene transfer; Lentiviral vectors; Oncoretroviral vectors; Stem cells

Indexed keywords

CD34 ANTIGEN; GROWTH FACTOR; LENTIVIRUS VECTOR; RETROVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BABOON; CELL LINEAGE; CELL POPULATION; CONTROLLED STUDY; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL TRANSPLANTATION; INTERMETHOD COMPARISON; NONHUMAN; PRIORITY JOURNAL; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; ANTIGENS, CD34; CELLS, CULTURED; GENE EXPRESSION; GENE THERAPY; GREEN FLUORESCENT PROTEINS; HEMATOPOIETIC CELL GROWTH FACTORS; HEMATOPOIETIC STEM CELLS; LENTIVIRUS; LUMINESCENT PROTEINS; MODELS, ANIMAL; PAPIO; POLYMERASE CHAIN REACTION; RECOMBINANT PROTEINS; RETROVIRIDAE; STEM CELL TRANSPLANTATION; TRANSDUCTION, GENETIC; TRANSPLANTATION, AUTOLOGOUS;

ANIMALIA; LENTIVIRUS; PAPIO; PAPIO HAMADRYAS; UNIDENTIFIED RETROVIRUS;

EID: 0036846846     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3301820     Document Type: Article

References (39)

1. Jones RJ et al. Separation of pluripotent haematopoietic stem cells from spleen colony-forming cells. Nature 1990; 347: 188-189.
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. Roe T, Reynolds TC, Yu G, Brown PO. Integration of murine leukemia virus DNA depends on mitosis. EMBO J 1993; 12: 2099-2108.
4. Nolta JA, Kohn DB. Comparison of the effects of growth factors on retroviral vector-mediated gene transfer and the proliferative status of human hematopoietic progenitor cells. Hum Gene Ther 1990; 1: 257-268.
5. Luskey BD, Rosenblatt M, Zsebo K, Williams DA. Stem cell factor, interleukin-3, and interleukin-6 promote retroviral-mediated gene transfer into murine hematopoietic stem cells. Blood 1992; 80: 396-402.
6. Bodine DM, Karlsson S, Nienhuis AW. Combination of interleukins 3 and 6 preserves stem cell function in culture and enhances retrovirus-mediated gene transfer into hematopoietic stem cells. Proc Natl Acad Sci USA 1989; 86: 8897-8901.
7. Jordan CT, Yamasaki G, Minamoto D. High-resolution cell cycle analysis of defined phenotypic subsets within primitive human hematopoietic cell populations. Exp Hematol 1996; 24: 1347-1355.
8. Tisdale JF et al. Ex vivo expansion of genetically marked Rhesus peripheral blood progenitor cells results in diminished long-term repopulating ability. Blood 1998; 92: 1131-1141.
9. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
10. Lewis PF, Emerman M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 1994; 68: 510-516.
11. Mikkola H et al. Lentivirus gene transfer in murine 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.
12. 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 1997; 90: 4638-4645.
13. 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 1998; 92: 1878-1886.
14. Kurre P et al. 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 2001; 3: 920-927.
15. Huhn RD et al. Retroviral marking and transplantation of rhesus hematopoietic cells by nonmyeloablative conditioning. Hum Gene Ther 1999; 10: 1783-1790.
16. Dunbar CE. The use of nonhuman primate models to improve gene transfer into haematopoietic stem cells (review). J Intern Med 2001; 249: 329-338.
17. Follenzi A et al. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 2000; 25: 217-222.
18. + marrow cells transduced by RD114-pseudotype oncoretrovirus vectors. Blood 2001; 98: 2065-2070.
19. Donahue RE et al. High levels of lymphoid expression of enhanced green fluorescent protein in nonhuman primates transplanted with cytokine-mobilized peripheral blood CD34(+) cells. Blood 2000; 95: 445-452.
20. + cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
21. An DS et al. Marking and gene expression by a lentivirus vector in transplanted human and nonhuman primate CD34(+) cells. J Virol 2000; 74: 1286-1295.
22. An DS et al. Lentivirus vector-mediated hematopoietic stem cell gene transfer of common gamma-chain cytokine receptor in rhesus macaques. J Virol 2001; 75: 3547-3555.
23. Zack JA et al. HIV-1 entry into quiescent primary lymphocytes: molecular analysis reveals a labile, latent viral structure. Cell 1990; 61: 213-222.
24. Chou CS, Ramilo O, Vitetta ES. Highly purified CD25-resting T cells cannot be infected de novo with HIV-1. Proc Natl Acad Sci USA 1997; 94: 1361-1365.
25. Korin YD, Zack JA. Nonproductive human immunodeficiency virus type 1 infection in nucleoside-treated G0 lymphocytes. J Virol 1999; 73: 6526-6532.
26. Unutmaz D, KewalRamani VN, Marmon S, Littman DR. Cytokine signals are sufficient for HIV-1 infection of resting human T lymphocytes. J Exp Med 1999; 189: 1735-1746.
27. Kiem H-P et al. Improved gene transfer into canine hematopoietic repopulating cells using CD34-enriched marrow cells in combination with a gibbon ape leukemia virus-pseudotype retroviral vector. Gene Therapy 1999; 6: 966-972.
28. von Laer D et al. Amphotropic and VSV-G-pseudotyped retroviral vectors transduce human hematopoietic progenitor cells with similar efficiency. Bone Marrow Transplant 2000; 26: (Suppl. 2) S75-S79.
29. Gallardo HF, Tan C, Ory D, Sadelain M. 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.
30. Shi PA et al. Comparison in the rhesus monkey of in vivo gene marking with amphotrophic and VSV-G pseudotyped retroviral vectors. Blood 2001; 98: (Part 1) 209a.
31. Schmidt M et al. Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum Gene Ther 2001; 12: 743-749.
32. Kohn DB et al. Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat Med 1995; 1: 1017-1023.
33. Nolta JA et al. Transduction of pluripotent human hematopoietic stem cells demonstrated by clonal analysis after engraftment in immune-deficient mice. Proc Natl Acad Sci USA 1996; 93: 2414-2419.
34. Guenechea G, Gan OI, Dorrell C, Dick JE. Distinct classes of human stem cells that differ in proliferative and self-renewal potential. Nat Immunol 2001; 2: 75-82.
35. Miller AD, Rosman GJ. Improved retroviral vectors for gene transfer and expression. Biotechniques 1989; 7: 980-982.
36. Zufferey R, Donello JE, Trono D, Hope TJ. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 1999; 73: 2886-2892.
37. Zufferey R et al. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
38. Hanenberg H et al. Colocalization of retrovirus and target cells on specific fibronectin fragments increases genetic transduction of mammalian cells. Nat Med 1996; 2: 876-882.
39. Heid CA, Stevens J, Livak KJ, Williams PM. Real time quantitative PCR. Genome Res 1996; 6: 986-994.