RNA-based anti-HIV-1 gene therapeutic constructs in SCID-hu mouse model

Molecular Therapy

Volumn 6, Issue 6, 2002, Pages 770-782

  Bail, Jirong ^a,d   Banda, Nirmal ^b   Lee, Nan Sook ^c   Rossi, John ^c   Akkina, Ramesh ^a  

^a Department of Environmental and Radiological Health Sciences   (United States)

^b UNIVERSITY OF COLORADO   (United States)

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

^d DANA FARBER CANCER INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

APTAMER; CD14 ANTIGEN; CD34 ANTIGEN; CD4 ANTIGEN; CELL MARKER; CHEMOKINE RECEPTOR CCR5; CHEMOKINE RECEPTOR CXCR4; RETROVIRUS VECTOR; REV PROTEIN; RIBOZYME; TRANSACTIVATOR PROTEIN; VIRUS ENVELOPE PROTEIN; VIRUS RECEPTOR; VIRUS RNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; BONE MARROW CELL; CELL ASSAY; CELL DIFFERENTIATION; CELL FUNCTION; CELL HYBRIDIZATION; CELL JUNCTION; CELL LINEAGE; CELL PROTECTION; CELL SURFACE; COLONY FORMING UNIT; CONTROLLED STUDY; ENZYME RECONSTITUTION; ERYTHROID CELL; GENE CONSTRUCT; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HLA SYSTEM; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; HUMAN TISSUE; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; IN VIVO STUDY; LIVER GRAFT; MACROPHAGE; MOUSE; MOUSE STRAIN; NONHUMAN; PROGENY; PROTEIN EXPRESSION; PROTEIN TARGETING; PROVIRUS; PROVOCATION TEST; REGULATORY MECHANISM; REVIEW; SEQUENCE ANALYSIS; SOUTHERN BLOTTING; THYMOCYTE; THYMUS GRAFT; VIRAL GENE THERAPY; VIRUS CELL INTERACTION; VIRUS INHIBITION; VIRUS REPLICATION;

ANIMALIA; HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; RNA VIRUSES; UNIDENTIFIED RETROVIRUS;

EID: 0036940586     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2002.0800     Document Type: Review

References (56)

1. Luciw, P. (1996). Human immunodeficiency viruses and their replication. In Fields Virology (B. N. Fields, D. M. Knipe, and P. M. Howley, Eds.), pp. 1881-1952. Lippincott-Raven, Philadelphia.
2. Emerman, M., and Malim, M. H. (1998). HIV-1 regulatory/accessory genes: Keys to unraveling viral and host cell biology. Science 280: 1880-1884.
3. Miller, R. H., and Sarver, N. (1997). HIV accessory proteins as therapeutic targets. Nat. Med. 3: 389-394.
4. Maddon, P. J., et al. (1986). The T4 gene encodes the AIDS virus receptor and is expressed in the immune system and the brain. Cell 47: 333-348.
5. Michael, N. L., and Moore, J. P. (1999). HIV-1 entry inhibitors: Evading the issue. Nat. Med. 5: 740-742.
6. Coffin, J. (1996). Retroviridae: The viruses and their replication. In Fields Virology (B. N. Fields, D. M. Knipe, and P. M. Howley, Eds.), 3rd ed., pp. 1767-1848. Lippincott-Raven, Philadelphia.
7. Zhou, C., et al. (1994). Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes. Gene 149: 33-39.
8. Zhou, C., Bahner, I., Rossi, J. J., and Kohn D. B. (1996). Expression of hammerhead ribozymes by retroviral vectors to inhibit HIV-1 replication: Comparison of RNA levels and viral inhibition. Antisense Nucleic Acid Drug Dev. 6: 17-24.
9. Konokpa, K., Duzgunes, N., Rossi, J., and Lee, N. S. (1998). Receptor ligand-facilitated cationic liposome delivery of anti-HIV-1 Rev-binding aptamer and ribozyme DNAs. J. Drug Target 5: 247-259.
10. Good, P. D., et al. (1997). Expression of small, therapeutic RNAs in human cell nuclei. Gene Ther. 4: 45-54.
11. Dunbar, C. E. (1996). Gene transfer to hematopoietic stem cells: Implications of gene therapy of human disease. Annu. Rev. Med. 47: 11-20.
12. Kohn, D. B. (1995). The current status of gene therapy using hematopoietic stem cells. Curr. Opin. Pediatr. 7: 56-63.
13. Ho, A. D., et al. (1995). Stem cells as vehicles for gene therapy: Novel strategy for HIV infection. Stem Cells 13: 100-105.
14. Miller, A. D., and Rosman, G. J. (1989). Improved retroviral vectors for gene transfer and expression. BioTechniques 7: 980-990.
15. Symensma, T. L., Giver, L., Zapp, M., Takle, G. B., and Ellington, A. D. (1996). RNA aptamers selected to bind human immunodeficiency virus type 1 Rev in vitro are Rev responsive in vivo. J. Virol. 70: 179-187.
16. 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.
17. + hematopoietic progenitor cells in vitro and in a SCID-hu mouse model in vivo. Mol. Ther. 1: 244-254.
18. + cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G. J. Virol. 70: 2581-2585.
19. Aldrovandi, G. M., et al. (1993). The SCID-hu mouse as a model for HIV-1 infection. Nature 363: 732-736.
20. McCune, J. M., et al. (1988). The SCID-hu mouse: Murine model for the analysis of human hematolymphoid differentiation and function. Science 241: 1632-1639.
21. Akkina, R. K., Rosenblatt, J. D., Campbell, A. G., Chen, I. S. Y., and Zack, J. A. (1994). Modeling human lymphoid precursor cell gene therapy in the SCID-hu mice. Blood 84: 1393-1398.
22. Banda, N., et al. (1998). Diphtheria toxin A gene-mediated HIV-1 protection of cord blood-derived T cells in the SCID-hu mouse model. J. Hematother. 7: 319-331.
23. Fauci, A. S., and Desrosiers, R. C. (1997). Pathogenesis of HIV and SIV. In Retroviruses (J. M. Coffin, S. H. Hughes, and H. E. Varmus, Eds.), pp. 587-636. Cold Spring Harbor Laboratory Press, New York.
24. Marzio, P. D., Tse, J., and Landau, N. R. (1998). Chemokine receptor regulation and HIV type 1 tropism in monocyte-macrophages. AIDS Res. Hum. Retroviruses 14: 129-138.
25. Lebien, T. W. (1995). Lymphocyte ontogeny and homing receptors. In Williams Hematology (E. Beutler, M. A. Lichtman, and T. J. Kipps, Eds.), 5th ed., pp. 921-929., McGraw-Hill, New York.
26. Zou, Y., Kottmann, A. H., Kuroda, M., Taniuchi, I., and Littman, D. R. (1998). Function of the chemokine receptor CXCR4 in haematopoiesis and in cerebellar development. Nature 393: 595-598.
27. Nagasawa, T., et al. (1996). Defects of B-cell lymphopoiesis and bone-marrow myelopoiesis in mice lacking the CXC chemokine PBSF/SDF-1. Nature 382: 635-638.
28. Kawabata, K., et al. (1999). A cell-autonomous requirement for CXCR4 in long-term lymphoid and myeloid reconstitution. Proc. Natl. Acad. Sci. USA 96: 5663-5667.
29. Berkowitz, R. D., Beckerman, K. P., Schall, T. J., and McCune, J. M. (1998). CXCR4 and CCR5 expression delineates targets for HIV-1 disruption of T cell differentiation. J. Immunol. 161: 3702-3710.
30. Kitchen, S. G., and Zack, J. A. (1997). CXCR4 expression during lymphopoiesis: Implications for human immunodeficiency virus type 1 infection of the thymus. J. Virol. 71: 6928-6934.
31. Jamieson, B. D., Pang, S., Aldrovandi, G. M., Zha, J., and Zack, J. A. (1995). In vivo pathogenic properties of two clonal human immunodeficiency virus type 1 isolates. J. Virol. 69: 6259-6264.
32. Goldstein, H., et al. (1996). SCID-hu mice: A model for studying disseminated HIV infection. Semin. Immunol. 8: 223-231.
33. Kollmann, T. R., et al. (1994). Disseminated human immunodeficiency virus 1 (HIV-1) infection in SCID-hu mice after peripheral inoculation with HIV-1. J. Exp. Med. 179: 513-522.
34. Aldrovandi, G. M., and Zack, J. A. (1996). Replication and pathogenicity of human immunodeficiency virus type 1 accessory gene mutants in SCID-hu mice. J. Virol. 70: 1505-1511.
35. Jamieson, B. D., and Zack, J. A. (1999). Murine models for HIV disease. AIDS 13: S5-S11.
36. Brooks, D. G., Kitchen, S. G., Kitchen, C. M., Scripture-Adams, D. D., and Zack, J. A. (2001). Generation of HIV-1 latency during thymopoiesis. Nat. Med. 7: 459-464.
37. Koup, R. A. (2001). A new latent HIV reservoir. Nat. Med. 7: 404-405.
38. An, D. S., et al. (1997). High-efficiency transduction of human lymphoid progenitor cells and expression in differentiated T cells. J. Virol. 71: 1397-1404.
39. Bonyhadi, M. L., et al. (1997). RevM10-expressing T cells derived in vivo from transduced human hematopoietic stem-progenitor cells inhibit human immunodeficiency virus replication. J. Virol. 71: 4707-4716.
40. Bai, J., Rossi, J., and Akkina, R. (2001). Multi-valent anti-CCR5 ribozymes for stem cell based HIV-1 gene therapy. AIDS Res. Hum. Retroviruses 17: 385-399.
41. Gao, F., et al. (1996). Molecular cloning and analysis of functional envelope genes from human immunodeficiency virus type 1 sequence subtypes A through G. J. Virol. 70: 1651-1667.
42. Lee, N. S., et al. (2002). Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat. Biotechnol. 20: 500-5005.
43. Rossi, J. J. (2000). Ribozymes in the nucleolus. Science 285: 1685.
44. Mhashilkar, A. M., Biswas, D. K., La Vecchio, J., Pardee, A. B., and Marasco, W. A. (1997). Inhibition of human immunodeficiency virus type 1 replication in vitro by a novel combination of anti-Tat single chain-intrabodies and NF-κB antagonists. J. Virol. 71: 6486-6494.
45. Marin, M., et al. (1996). Targeted infection of human cells via major histocompatibility complex class I molecules by Moloney murine leukemia virus-derived viruses displaying single-chain antibody fragment-envelope fusion proteins. J. Virol. 70: 2957-2962.
46. Kitamura, Y., et al. (1999). Inhibition of replication of HIV-1 at both early and late stages of the viral life cycle by single-chain antibody against viral integrase. J. AIDS Hum. Retrovirol. 20: 105-114.
47. Steinberger, P., Andris-Widhopf, J., Buhler, B., Torbett, B. E., and Barbas, C. F., III (2000). 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 97: 805-810.
48. Amado, R. G., Mitsuyasu, R. T., and Zack, J. A. (1999). Gene therapy for the treatment of AIDS: Animal models and human clinical experience. Front. Biosci. 4: D468-475.
49. Weissman, I. L. (2000). Translating stem and progenitor cell biology to the clinic: Barriers and opportunities. Science 287: 1442-1446.
50. Kohn, D. B., et al. (1995). Engraftment of gene-modified umbilical cord blood cells in neonates with adenosine deaminase deficiency. Nat. Med. 1: 1017-1023.
51. + cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 94: 368-371.
52. Naldini, L., Blomer, U., Gage, F. H., Trono, D., and Verma, I. M. (1996). Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. USA 93: 11382-11388.
53. Amado, R. G., and Chen, I. S. Y. (1999). Lentiviral vectors-The promise of gene therapy within reach. Science 285: 674-676
54. Kay, M. A., Glorioso, J. C., and Naldini, L. (2001). Viral vectors for gene therapy: The art of turning infectious agents into vehicles of therapeutics. Nat. Med. 7: 33-40.
55. Ziegler, B. L., et al. (1999). KDR receptor: A key marker defining hematopoietic stem cells. Science 285: 1553-1558.
56. Bhatia, M., Wang, J. C., Kapp, U., Bonnet, D., and Dick, J. E. (1997). Purification of primitive human hematopoietic cells capable of repopulating immune-deficient mice. Proc. Natl. Acad. Sci. USA 94: 5320-5325.