Foamy combinatorial anti-HIV vectors with MGMTP140K potently inhibit HIV-1 and SHIV replication and mediate selection in vivo

Gene Therapy

Volumn 17, Issue 1, 2010, Pages 37-49

  Kiem, H P ^a   Wu, R A ^a   Sun, G ^b   Von Laer, D ^c   Rossi, J J ^b   Trobridge, G D ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

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

Author keywords

Foamy vector; Hematopoietic stem cell; Human immunodeficiency virus (HIV); Methylguanine methyltransferase; Simian human immunodeficiency virus(SHIV)

Indexed keywords

FOAMY VIRUS ANTI HUMAN IMMUNODEFICIENCY VIRUS VECTOR; METHYLGUANINE METHYLTRANSFERASE; METHYLTRANSFERASE; REV PROTEIN; SHORT HAIRPIN RNA; TRANSACTIVATOR PROTEIN; UNCLASSIFIED DRUG; VIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIVIRAL ACTIVITY; ARTICLE; CELL POPULATION; CELL TRANSPLANTATION; CHIMERA; CONTROLLED STUDY; DNA MODIFICATION; DRUG TARGETING; GENETIC TRANSDUCTION; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; MOUSE; NONHUMAN; PRIORITY JOURNAL; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS REPLICATION; VIRUS TITRATION; XENOTRANSPLANTATION;

ANIMALS; DNA MODIFICATION METHYLASES; DNA REPAIR ENZYMES; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HIV-1; HUMANS; MICE; SIMIAN FOAMY VIRUS; SIMIAN IMMUNODEFICIENCY VIRUS; TRANSDUCTION, GENETIC; TRANSGENES; TUMOR SUPPRESSOR PROTEINS; VIRUS REPLICATION;

HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN SPUMARETROVIRUS; SIMIAE; SIMIAN-HUMAN IMMUNODEFICIENCY VIRUS;

EID: 74349091890     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2009.118     Document Type: Article

References (64)

1. Yeni PG, Hammer SM, Carpenter CC, Cooper DA, Fischl MA, Gatell JM et al. Antiretroviral treatment for adult HIV infection in 2002: updated recommendations of the International AIDS Society-USA Panel [published erratum appears in JAMA 2003; 11: 32]. JAMA 2002; 288: 222-235.
2. Carr A. Toxicity of antiretroviral therapy and implications for drug development (review). Nat Rev Drug Discov 2003; 2: 624-634.
3. Klausner RD, Fauci AS, Corey L, Nabel GJ, Gayle H, Berkley S et al. Medicine. The need for a global HIV vaccine enterprise. Science 2003; 300: 2036-2039.
4. Sekaly RP. The failed HIV Merck vaccine study: a step back or a launching point for future vaccine development? J Exp Med 2008; 205: 7-12.
5. Cold shower for AIDS vaccines (Editorial). Nat Med 2007; 13: 1389-1390.
6. Rossi JJ, June CH, Kohn DB. Genetic therapies against HIV (Review). Nat Biotechnol 2007; 25: 1444-1454.
7. Van Lunzen J, Glaunsinger T, Stahmer I, von BV, Baum C, Schilz A et al. Transfer of autologous gene-modified T cells in HIV-infected patients with advanced immunodeficiency and drug-resistant virus. Mol Ther 2007; 15: 1024-1033.
8. Bauer G, Selander D, Engel B, Carbonaro D, Csik S, Rawlings S et al. Gene therapy for pediatric AIDS (Review). Ann NY Acad Sci 2000; 918: 318-329.
9. Kohn DB, Bauer G, Rice CR, Rothschild JC, Carbonaro DA, Valdez P et al. A clinical trial of retroviral-mediated transfer of a rev-responsive element decoy gene into CD34(+) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 1999; 94: 368-371.
10. Amado RG, Mitsuyasu RT, Rosenblatt JD, Ngok FK, Bakker A, Cole S et al. Anti-human immunodeficiency virus hematopoietic progenitor cell-delivered ribozyme in a phase I study: myeloid and lymphoid reconstitution in human immunodeficiency virus type-1-infected patients. Hum Gene Ther 2004; 15: 251-262.
11. Humeau LM, Binder GK, Lu X, Slepushkin V, Merling R, Echeagaray P et al. Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load. Mol Ther 2004; 9: 902-913.
12. + hematopoietic progenitor cells potently inhibits human immunodeficiency virus-1 replication. Mol Ther 2007; 15: 76-85.
13. Mautino MR, Morgan RA. Potent inhibition of human immunodeficiency virus type 1 replication by conditionally replicating human immunodeficiency virus-based lentiviral vectors expressing envelope antisense mRNA. Hum Gene Ther 2000; 11: 2025-2037.
14. Falcone V, Schweizer M, Neumann-Haefelin D. Replication of primate foamy viruses in natural and experimental hosts (Review). Curr Top Microbiol Immunol 2003; 277: 161-180.
15. Hooks JJ, Gibbs Jr CJ. The foamy viruses (Review). Bacteriol Rev 1975; 39: 169-185.
16. Flugel RM. Spumaviruses: a group of complex retroviruses (Review). J Acquir Immune Defic Syndr 1991; 4: 739-750.
17. Trobridge G, Vassilopoulos G, Josephson N, Russell DW. Gene transfer with foamy virus vectors. Methods Enzymol 2002; 346: 628-648.
18. Trobridge G, Josephson N, Vassilopoulos G, Mac J, Russell DW. Improved foamy virus vectors with minimal viral sequences. Mole Ther 2002; 6: 321-328.
19. Vassilopoulos G, Trobridge G, Josephson NC, Russell DW. Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors. Blood 2001; 98: 604-609.
20. Josephson NC, Trobridge G, Russell DW. Transduction of long-term and mobilized peripheral blood-derived NOD/SCID repopulating cells by foamy virus vectors. Hum Gene Ther 2004; 15: 87-92.
21. Josephson NC, Vassilopoulos G, Trobridge GD, Priestly GV, Wood BL, Papayannopoulou T et al. Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors. Proc Natl Acad Sci USA 2002; 99: 8295-8300.
22. Kiem H-P, Allen J, Trobridge G, Olson E, Keyser K, Peterson L et al. Foamy virus-mediated gene transfer to canine repopulating cells. Blood 2007; 109: 65-70.
23. Trobridge GD, Miller DG, Jacobs MA, Allen JM, Kiem H-P, Kaul R et al. Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci 2006; 103: 1498-1503.
24. Trobridge G, Beard BC, Kiem H-P. Hematopoietic stem cell transduction and amplification in large animal models. Hum Gene Ther 2005; 16: 1355-1366.
25. Crone TM, Goodtzova K, Edara S, Pegg AE. Mutations in human O6-alkylguanine-DNA alkyltransferase imparting resistance to O6-benzylguanine. Cancer Res 1994; 54: 6221-6227.
26. Xu-Welliver M, Kanugula S, Pegg AE. Isolation of human O6-alkylguanine-DNA alkyltransferase mutants highly resistant to inactivation by O6-benzylguanine. Cancer Res 1998; 58: 1936-1945.
27. Zielske SP, Reese JS, Lingas KT, Donze JR, Gerson SL. In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/ SCID repopulating cells without pretransplant irradiation conditioning. J Clin Invest 2003; 112: 1561-1570.
28. Neff T, Horn PA, Peterson LJ, Thomasson BM, Thompson J, Williams DA et al. Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest 2003; 112: 1581-1588.
29. Neff T, Beard BC, Peterson LJ, Anandakumar P, Thompson J, Kiem H-P. Polyclonal chemoprotection against temozolomide in a large-animal model of drug resistance gene therapy. Blood 2005; 105: 997-1002.
30. Joag SV, Li Z, Foresman L, Stephens EB, Zhao LJ, Adany I et al. Chimeric simian/human immunodeficiency virus that causes progressive loss of CD4+ T cells and AIDS in pig-tailed macaques. J Virol 1996; 70: 3189-3197.
31. Schambach A, Bohne J, Baum C, Hermann FG, Egerer L, von Laer D et al. Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Therapy 2006; 13: 641-645.
32. Trobridge GD, Beard BC, Gooch C, Wohlfahrt M, Olsen P, Fletcher J et al. Efficient transduction of pigtailed macaque hemtopoietic repopulating cells with HIV-based lentiviral vectors. Blood 2008; 111: 5537-5543.
33. Keller A, Partin KM, Lochelt M, Bannert H, Flugel RM, Cullen BR. Characterization of the transcriptional transactivator of human foamy retrovirus. J Virol 1991; 65: 2589-2594.
34. Lochelt M, Zentgraf H, Flugel RM. Construction of an infectious DNA clone of the full-length human spumaretrovirus genome and mutagenesis of the bel 1 gene. Virology 1991; 184: 43-54.
35. Boden D, Pusch O, Lee F, Tucker L, Ramratnam B. Human immunodeficiency virus type 1 escape from RNA interference. J Virol 2003; 77: 11531-11535.
36. Li MJ, Bauer G, Michienzi A, Yee JK, Lee NS, Kim J et al. Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs. Mole Ther 2003; 8: 196-206.
37. Lee NS, Dohjima T, Bauer G, Li H, Li MJ, Ehsani A et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 2002; 20: 500-505.
38. Samson M, Libert F, Doranz BJ, Rucker J, Liesnard C, Farber CM et al. Resistance to HIV-1 infection in Caucasian individuals bearing mutant alleles of the CCR-5 chemokine receptor gene. Nature 1996; 382: 722-725.
39. Liu R, Paxton WA, Choe S, Ceradini D, Martin SR, Horuk R et al. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection. Cell 1996; 86: 367-377.
40. An DS, Qin FX, Auyeung VC, Mao SH, Kung SK, Baltimore D et al. Optimization and functional effects of stable short hairpin RNA expression in primary human lymphocytes via lentiviral vectors. Mole Ther 2006; 14: 494-504.
41. Egelhofer M, Brandenburg G, Martinius H, Schult-Dietrich P, Melikyan G, Kunert R et al. Inhibition of human immunodeficiency virus type 1 entry in cells expressing gp41-derived peptides. J Virol 2004; 78: 568-575.
42. Hermann FG, Martinius H, Egelhofer M, Giroglou T, Tonn T, Roth SD et al. Protein scaffold and expression level determine antiviral activity of membrane-anchored antiviral peptides. Hum Gene Ther 2009; 20: 325-336.
43. Ngoi SM, Chien AC, Lee CG. Exploiting internal ribosome entry sites in gene therapy vector design (Review). Curr Gene Ther 2004; 4: 15-31.
44. Kimpton C, Walton A, Gill P. A further tetranucleotide repeat polymorphism in the vWF gene. Hum Mol Genet 1992; 1: 287.
45. Chackerian B, Long EM, Luciw PA, Overbaugh J. Human immunodeficiency virus type 1 coreceptors participate in post-entry stages in the virus replication cycle and function in simian immunodeficiency virus infection. J Virol 1997; 71: 3932-3939.
46. Tuschl T. Expanding small RNA interference. Nat Biotechnol 2002; 20: 446-448.
47. ter Brake O, t Hooft K, Liu YP, Centlivre M, von Eije KJ, Berkhout B. Lentiviral vector design for multiple shRNA expression and durable HIV-1 inhibition. Mol Ther 2008; 16: 557-564.
48. Aagaard L, Zhang J, von Eije KJ, Li H, Saetrom P, Amarzguioui M et al. Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs. Gene Therapy 2008; 15: 1566.
49. Shultz LD, Lyons BL, Burzenski LM, Gott B, Chen X, Chaleff S et al. Human lymphoid and myeloid cell development in NOD/ LtSz-scid IL2R gamma null mice engrafted with mobilized human hemopoietic stem cells. J Immunol 2005; 174: 6477-6489.
50. Flugel RM, Rethwilm A, Maurer B, Darai G. Nucleotide sequence analysis of the env gene and its flanking regions of the human spumaretrovirus reveals two novel genes [published erratum appears in EMBO J 1990;9:3806]. EMBO J 1987; 6: 2077-2084.
51. Bukovsky AA, Song JP, Naldini L. Interaction of human immunodeficiency virus-derived vectors with wild-type virus in transduced cells. J Virol 1999; 73: 7087-7092.
52. Taylor JA, Vojtech L, Bahner I, Kohn DB, Laer DV, Russell DW et al. Foamy virus vectors expressing anti-HIV trans-genes efficiently block HIV-1 replication. Mol Ther 2008; 16: 46-51.
53. Park J, Nadeau P, Zucali JR, Johnson CM, Mergia A. Inhibition of simian immunodeficiency virus by foamy virus vectors expressing siRNAs. Virology 2005; 343: 275-282.
54. An DS, Donahue RE, Kamata M, Poon B, Metzger M, Mao SH et al. Stable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates. Proc Natl Acad Sci 2007; 104: 13110-13115.
55. Schambach A, Schiedlmeier B, Kuhlcke K, Verstegen M, Margison GP, Li Z et al. Towards hematopoietic stem cell-mediated protection against infection with human immunodeficiency virus. Gene Therapy 2006; 13: 1037-1047.
56. Mezquita P, Beard B, Kiem H-P. NOD/SCID repopulating cells contribute only to short-term repopulation in the baboon. Gene Therapy 2008; 15: 1460-1462.
57. Levy JA. The value of primate models for studying human immunodeficiency virus pathogenesis. 1996; 25: 163-174.
58. Nathanson N, Hirsch VM, Mathieson BJ. The role of nonhuman primates in the development of an AIDS vaccine (Review). AIDS 1999; 13 (Suppl A): S113-S120.
59. Warren J. Preclinical AIDS vaccine research: survey of SIV, SHIV, and HIV challenge studies in vaccinated nonhuman primates. J Med Primatol 2002; 31: 237-256.
60. Heinkelein M, Dressler M, Jarmy G, Rammling M, Imrich H, Thurow J et al. Improved primate foamy virus vectors and packaging constructs. J Virol 2002; 76: 3774-3783.
61. Salter RD, Howell DN, Cresswell P. Genes regulating HLA class I antigen expression in T-B lymphoblast hybrids. Immunogenetics 1985; 21: 235-246.
62. Trkola A, Matthews J, Gordon C, Ketas T, Moore JP. A cell line-based neutralization assay for primary human immunodeficiency virus type 1 isolates that use either the CCR5 or the CXCR4 coreceptor. J Virol 1999; 73: 8966-8974.
63. Boussif O, Lezoualc'h F, Zanta MA, Mergny MD, Scherman D, Demeneix B et al. A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine. Proc Natl Acad Sci 1995; 92: 7297-7301.
64. Collman R, Balliet JW, Gregory SA, Friedman H, Kolson DL, Nathanson N et al. An infectious molecular clone of an unusual macrophage-tropic and highly cytopathic strain of human immunodeficiency virus type 1. J Virol 1992; 66: 7517-7521.