Protecting from R5-tropic HIV: Individual and combined effectiveness of a hammerhead ribozyme and a single-chain Fv antibody that targets CCR5

Gene Therapy

Volumn 11, Issue 22, 2004, Pages 1627-1637

  Cordelier, P ^a   Kulkowsky, J W ^a   Ko, C ^a   Matskevitch, A A ^a   McKee, H J ^a   Rossi, J J ^b   Bouhamdan, M ^a   Pomerantz, R J ^a   Kari, G ^a   Strayer, D S ^a  

^a THOMAS JEFFERSON UNIVERSITY   (United States)

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

Author keywords

CCR5; Intrabody; Intrakine; Macrophages; Microglia; rSV40

Indexed keywords

CHEMOKINE RECEPTOR CCR5; DNA; MACROPHAGE INFLAMMATORY PROTEIN 1ALPHA; MACROPHAGE INFLAMMATORY PROTEIN 1BETA; RIBOZYME; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; VIRUS VECTOR;

ANIMAL CELL; ARTICLE; CELL LINE; CELL MEMBRANE; CELL PROTECTION; CONTROLLED STUDY; CYTOFLUOROMETRY; DNA MODIFICATION; DRUG EFFICACY; DRUG POTENCY; DRUG TARGETING; ENDOPLASMIC RETICULUM; FLUORESCENCE ACTIVATED CELL SORTING; GENE DELETION; GENE THERAPY; GENETIC POLYMORPHISM; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNOHISTOCHEMISTRY; MACROPHAGE; MICROGLIA; MONOCYTE; NONHUMAN; NORTHERN BLOTTING; PRIORITY JOURNAL; SIMIAN VIRUS 40; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS IMMUNITY; VIRUS RECOMBINANT; VIRUS RESISTANCE; VIRUS STRAIN;

CELL LINE; CELL MEMBRANE; CELLS, CULTURED; FLOW CYTOMETRY; GENE TARGETING; GENE THERAPY; GENETIC ENGINEERING; GENETIC VECTORS; HIV INFECTIONS; HIV-1; HUMANS; IMMUNOGLOBULIN FRAGMENTS; MACROPHAGE INFLAMMATORY PROTEIN-1; MACROPHAGES; NEUROGLIA; RECEPTORS, CCR5; RNA, CATALYTIC; SIMIAN VIRUS 40; TRANSGENES;

EID: 8844259701     PISSN: 09697128     EISSN: None     Source Type: Journal    
DOI: 10.1038/sj.gt.3302329     Document Type: Article

References (62)

1. Berger EA, Murphy PM, Farber JM. Chemokine receptors as HIV-1 coreceptors: roles in viral entry, tropism, disease. Annu Rev Immunol 1999; 17: 657-700.
2. Collman RG, Yi Y. Cofactors for human immunodeficiency virus entry into primary macrophages. J Infect Dis 1999; 179: S422-S426.
3. Liu 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.
4. Samson M 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.
5. Dean M et al. Genetic restriction of HIV-1 infection, progression to AIDS by a deletion allele of the CKR5 structural gene. Hemophilia Growth, Development Study, Multicenter AIDS Cohort Study, Multicenter Hemophilia Cohort Study, San Francisco City Cohort, ALIVE Study. Science 1996; 273: 1856-1862.
6. Huang Y et al. The role of a mutant CCR5 allele in HIV-1 transmission, disease progression. Nat Med 1996; 2: 1240-1243.
7. Eugen-Olsen J et al. Heterozygosity for a deletion in the CKR-5 gene leads to prolonged AIDS-free survival, slower CD4 T-cell decline in a cohort of HIV-seropositive individuals. Aids 1997; 11: 305-310.
8. O'Brien TR et al. 2nd HIV-1 infection in a man homozygous for CCR5 delta 32. Lancet 1997; 349: 1219.
9. Theodorou I et al. HIV-1 infection in an individual homozygous for CCR5 delta 32. Seroco Study Group. Lancet 1997; 349: 1219-1220.
10. Quillent C et al. HIV-1-resistance phenotype conferred by combination of two separate inherited mutations of CCR5 gene. Lancet 1998; 351: 14-18.
11. Kupfer B et al. Protection against parenteral HIV-1 infection by homozygous deletion in the C-C chemokine receptor 5 gene. Aids 1999; 13: 1025-1028.
12. Ometto L et al. Restriction of HIV type 1 infection in macrophages heterozygous for a deletion in the CC-chemokine receptor 5 gene. AIDS Res Hum Retroviruses 1999; 15: 1441-1452.
13. Ward SG, Bacon K, Westwick J. Chemokines, T lymphocytes: more than an attraction. Immunity 1998; 9: 1-11.
14. Ward SG, Westwick J. Chemokines: understanding their role in T-lymphocyte biology. Biochem J 1998; 333: 457-470.
15. Cagnon L, Rossi JJ. Downregulation of the CCR5 beta-chemokine receptor, inhibition of HIV-1 infection by stable VA1-ribozyme chimeric transcripts. Antisense Nucleic Acid Drug Dev 2000; 10: 251-261.
16. Feng Y et al. Inhibition of CCR5-dependent HIV-1 infection by hairpin ribozyme gene therapy against CC-chemokine receptor 5. Virology 2000; 276: 271-278.
17. Rossi JJ, Sarver N. RNA enzymes (ribozymes) as antiviral therapeutic agents. Trends Biotechnol 1990; 8: 179-183.
18. Rossi JJ et al. Ribozymes as therapies for AIDS. Ann NY Acad Sci 1990; 616: 184-200.
19. Sarver N et al. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 1990; 247: 1222-1225.
20. Steinberger P et al. Functional deletion of the CCR5 receptor by intracellular immunization produces cells that are refractory to CCR5-dependent HIV-1 infection and cell fusion. Proc Natl Acad Sci USA 2000; 97: 805-810.
21. Königs C et al. Monoclonal antibody screening of a phage-displayed random peptide library reveals mimotopes of chemokine receptor CCR5: implications for the tertiary structure of the receptor and for an N-terminal binding site for HIV-1 gp120. Eur J Immunol 2000; 30: 1162-1171.
22. Chen JD et al. Inactivation of HIV-1 chemokine co-receptor CXCR-4 by a novel intrakine strategy. Nat Med 1997; 3: 1110-1116.
23. Strayer DS, Milano J. SV40 mediates stable gene transfer in vivo. Gene Therapy 1996; 3: 581-587.
24. Strayer DS. SV40 as an effective gene transfer vector in vivo. J Biol Chem 1996; 271: 24741-24746.
25. Strayer DS, Kondo R, Milano J, Duan LX. Use of SV40-based vectors to transduce foreign genes to normal human peripheral blood mononuclear cells. Gene Therapy 1997; 4: 219-225.
26. Strayer DS, Zern MA. Gene delivery to the liver using simian virus 40-derived vectors. Semin Liver Dis 1999; 19: 71-81.
27. Strayer DS. Effective gene transfer using viral vectors based on SV40. Methods Mol Biol 2000; 133: 61-74.
28. Strayer DS et al. Durability of transgene expression and vector integration: recombinant SV40-derived gene therapy vectors. Mol Ther 2002; 2: 227-237.
29. Kondo R, Feitelson MA, Strayer DS. Use of SV40 to immunize against hepatitis B surface antigen: implications for the use of SV40 for gene transduction and its use as an immunizing agent. Gene Therapy 1998; 5: 575-582.
30. Strayer DS et al. Generation of recombinant SV40 vectors for gene transfer. Methods Mol Biol 2001; 165: 103-117.
31. Lee B et al. Epitope mapping of CCR5 reveals multiple conformational sites and distinct but overlapping structures involved in chemokine and coreceptor function. J Biol Chem 1999; 274: 9617-9626.
32. Goldstein H et al. Gene therapy using a simian virus 40-derived vector inhibits the development of in vivo human immunodeficiency virus type 1 infection of severe combined immunodeficiency mice implanted with human fetal thymic and liver tissue. J Infect Dis 2002; 185: 1425-1430.
33. BouHamdan M et al. Inhibition of HIV-1 infection by down-regulation of the CXCR4 co-receptor using an intracellular single chain variable fragment against CXCR4. Gene Therapy 2001; 8: 408-418.
34. Sauter BV et al. A replication-deficient rSV40 mediates liver-directed gene transfer and a long-term amelioration of jaundice in gunn rats. Gastroenterology 2000; 119: 1348-1357.
35. Signoret N et al. Differential regulation of CXCR4 and CCR5 endocytosis. J Cell Sci 1998; 111: 2819-2830.
36. Means RE et al. Ability of the V3 loop of simian immunodeficiency virus to serve as a target for antibody-mediated neutralization: correlation of neutralization sensitivity, growth in macrophages, and decreased dependence on CD4. J Virol 2001; 75 3903-3915.
37. Vincent F et al. Human blood-derived macrophages: differentiation in vitro of a large quantity of cells in serum-free medium. Exp Hematol 1992; 20: 17-23.
38. Eischen A et al. Long term cultures of human monocytes in vitro. Impact of GM-CSF on survival and differentiation. J Immunol Methods 1991; 143: 209-221.
39. Lee SC, Liu W, Brosnan CF, Dickson DW. Characterization of primary human fetal dissociated central nervous system cultures with an emphasis on microglia. Lab Invest 1992; 67: 465-476.
40. Lee S et al. Coreceptor competition for association with CD4 may change the susceptibility of human cells to infection with T-tropic and macrophage tropic isolates of human immunodeficiency virus type 1. J Virol 2000; 74: 5016-5023.
41. Wu L et al. CCR5 levels and expression pattern correlate with infectability by macrophage-tropic HIV-1, in vitro. J Exp Med 1997; 185: 1681-1691.
42. Engel BC et al. Intrakines - evidence for a trans cellular mechanism of action. Mol Ther 2000; 1: 165-170.
43. Strayer DS et al. Combination genetic therapy to inhibit HIV-1. Mol Ther 2002; 5: 33-41.
44. Ferkol T, Perales JC, Mularo F, Hanson RW. Receptor-mediated gene transfer into macrophages. Proc Natl Acad Sci USA 1996; 93 101-105.
45. Erbacher P et al. Gene transfer by DNA/glycosylated polylysine complexes into human blood monocyte-derived macrophages. Hum Gene Ther 1996; 7: 721-729.
46. Byrnes AP, Wood MJ, Charlton HM. Role of T cells in inflammation caused by adenovirus vectors in the brain. Gene Therapy 1996; 3: 644-651.
47. Cremer I, Vieillard V, De Maeyer E. Retrovirally mediated IFN-beta transduction of macrophages induces resistance to HIV, correlated with up-regulation of RANTES production and down-regulation of C-C chemokine receptor-5 expression. J Immunol 2000; 164: 1582-1587.
48. Naldini L et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272: 263-267.
49. Gervaix A et al. Gene therapy targeting peripheral blood CD34+ hematopoietic stem cells of HIV-infected individuals. Hum Gene Ther 1997; 8: 2229-2238.
50. Davis BR et al. Targeted transduction of CD34+ cells by transdominant negative Rev-expressing retrovirus yields partial anti-HIV protection of progeny macrophages. Hum Gene Ther 1998; 9: 1197-1207.
51. Junker U et al. Inhibition of human immunodeficiency virus type 1 replication in myelomonocytic cells derived from retroviral vector-transduced peripheral blood progenitor cells. Hum Gene Ther 1998; 9: 333-340.
52. Yu M et al. Intracellular immunization of human fetal cord blood stem/progenitor cells with a ribozyme against human immunodeficiency virus type 1. Proc Natl Acad Sci USA 1995; 92 699-703.
53. Qin XF, An DS, Chen IS, Baltimore D. Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5. Proc Natl Acad Sci USA 2003; 100 183-188.
54. Cordelier P, Morse B, Strayer DS. Targeting CCR5 with siRNAs: using recombinant SV40-derived vectors to protect macrophages and microglia from R5-tropic HIV. Oligonucleotides 2003; 13: 281-294.
55. Chatterjee S et al. Transduction of primitive human marrow and cord blood-derived hematopoietic progenitor cells with adeno-associated virus vectors. Blood 1999; 93: 1882-1894.
56. Norkin LC, Anderson HA, Wolfrom SA, Oppenheim A. Caveolar endocytosis of simian virus 40 is followed by brefeldin A-sensitive transport to the endoplasmic reticulum, where the virus disassembles. J Virol 2002; 76: 5156-5166.
57. Pelkmans L, Kartenbeck J, Helenius A. Caveolar endocytosis of simian virus 40 reveals a new two-step vesicular-transport pathway to the ER. Nat Cell Biol 2001; 3: 473-483.
58. Parton RG, Lindsay M. Exploitation of major histocompatibility complex class I molecules and caveolae by simian virus 40. Immunol Rev 1999; 168: 23-31.
59. Norkin LC. Simian virus 40 infection via MHC class I molecules and caveolae. Immunol Rev 1999; 168: 13-22.
60. Anderson HA, Chen Y, Norkin LC. Bound simian virus 40 translocates to caveolin-enriched membrane domains, and its entry is inhibited by drugs that selectively disrupt caveolae. Mol Biol Cell 1996; 7: 1825-1834.
61. Paxton WA, Kang S, Koup RA. The HIV type 1 coreceptor CCR5 and its role in viral transmission and disease progression. AIDS Res Hum Retroviruses 1998; 14: S89-S92.
62. Aarons EJ et al. Adaptation to blockade of human immunodeficiency virus type 1 entry imposed by the anti-CCR5 monoclonal antibody 2D7. Virology 2001; 287: 382-390.