Characterization of Anti-CCR5 Ribozyme-Transduced CD34+ Hematopoietic Progenitor Cells in Vitro and in a SCID-hu Mouse Model in Vivo

Molecular Therapy

Volumn 1, Issue 3, 2000, Pages 244-254

  Bai, Jirong ^a   Gorantla, Santhi ^a   Banda, Nirmal ^b   Cagnon, Laurence ^c   Rossi, John ^c   Akkina, Ramesh ^a  

^a Immunology and Pathology   (United States)

^b UNIVERSITY OF COLORADO   (United States)

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

Author keywords

AIDS; HIV 1 gene therapy; chemokine CCR5 coreceptor; ribozyme gene therapy; CD34+ progenitor cells; stem cell gene therapy; SCID hu mouse model

Indexed keywords

CD34 ANTIGEN; CHEMOKINE RECEPTOR CCR5; MESSENGER RNA; RIBOZYME;

ANIMAL; ARTICLE; CLONOGENIC ASSAY; CYTOLOGY; DOWN REGULATION; DRUG ANTAGONISM; GENE THERAPY; GENE TRANSFER; GENETICS; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; IMMUNOPHENOTYPING; MACROPHAGE; METABOLISM; METHODOLOGY; MOUSE; MOUSE MUTANT; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; T LYMPHOCYTE;

ANIMALS; ANTIGENS, CD34; COLONY-FORMING UNITS ASSAY; DOWN-REGULATION; GENE THERAPY; GENE TRANSFER TECHNIQUES; HEMATOPOIETIC STEM CELLS; HIV INFECTIONS; HIV-1; HUMANS; IMMUNOPHENOTYPING; MACROPHAGES; MICE; MICE, SCID; RECEPTORS, CCR5; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, CATALYTIC; RNA, MESSENGER; T-LYMPHOCYTES;

EID: 0034149743     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1006/mthe.2000.0038     Document Type: Article

References (67)

1. Balter M. Global program struggles to stem the flood of new cases. Science. 280:1998;1863-1864.
2. Balter M. HIV survives drug onslaught by hiding out in T cells. Science. 278:1999;1227.
3. Ho D. Toward HIV eradication or remission: The tasks ahead. Science. 280:1998;1866-1867.
4. Balter M. On World AIDS Day, a shadow looms over Southern Africa. Science. 280:1998;1790-1791.
5. Finzi D., Blankson J., Siliciano J. D., Margolick J. B., Chadwick K., Pierson T., Smith K., Lisziewicz J., Lori F., Flexner C., Quinn T. C., Chaisson R. E., Rosenberg E., Walker B., Gange S., Gallant J., Siliciano R. F. Latent infection of CD4 T cells provides a mechanism for lifelong persistence of HIV-1 infection, even in patients on effective combination therapy. Nat. Med. 5:1999;512-517.
6. Finzi D., Hermankova M., Pierson T., Carruth L. M., Buck C., Chaisson R. E., Quinn T. C., Chadwick K., Margolick J., Brookmeyer R., Gallant J., Markowitz M., Ho D. D., Richman D. D., Siliciano R. F. Identification of a reservoir for HIV-1 in patients on highly active antiretroviral therapy. Science. 278:1997;1295-1300.
7. Wong J. K., Hezareh M., Günthard H. F., Havlir D. V., Ignacio C. C., Spina C. A., Richman D. D. Recovery of replication-competent HIV despite prolonged suppression of plasma viremia. Science. 278:1997;1291-1295.
8. Brown A. J., Richman D. D. HIV-1: Gambling on the evolution of drug resistance. Nat. Med. 3:1997;268-271.
9. Bushman F., Landau N. R., Emini E. A. New developments in the biology and treatment of HIV. Proc. Natl. Acad. Sci. USA. 95:1998;11041-11042.
10. Perrin L., Telenti A. HIV treatment failure: Testing for HIV resistance in clinical practice. Science. 280:1998;1871-1872.
11. Cagnon L., Cucchiarini M., Lefebvre J.-C., Dglio A. Protection of a T-cell line from human immunodeficiency virus replication by the stable expression of a short antisense RNA sequence carried by a shuttle RNA molecule. J. AIDS Hum. Retrovirol. 9:1995;349-358.
12. + monocytic cells treated with multitargeting HIV-1 antisense sequences incorporated into U1 snRNA. J. Virol. 71:1997;4079-4085.
13. + cells with an anti-HIV-1 ribozyme gene. Adv. Drug Delivery Rev. 17:1995;363-368.
14. Zhou C., Bahner I., Larson G. P., Zaia J. A., Rossi J. J., Kohn D. B. Inhibition of HIV-1 in human T-lymphocytes by retrovirally transduced anti-tat and rev hammerhead ribozymes. Gene. 149:1994;33-39.
15. + hematopoietic progenitor cells by a retroviral vector expressing an RRE decoy inhibits human immunodeficiency virus type 1 replication in myelomonocytic cells produced in long term culture. J. Virol. 70:1996;4352-4360.
16. Lisziewicz J., Sun D., Smythe J., Lusso P., Lori F., Louie A., Markham P., Rossi J., Reitz M., Gallo R. C. Inhibition of human immunodeficiency virus type 1 replication by regulated expression of a polymeric Tat activation response RNA decoy as a strategy for gene therapy in AIDS. Proc. Natl. Acad. Sci. USA. 90:1993;8000-8004.
17. + hematopoietic progenitor cells with an antitat gene protects T-cell and macrophage progeny from AIDS virus infection. J. Virol. 71:1997;2740-2746.
18. Bonyhadi M., Moss K., Voytovich A., Auten J., Kalfoglou C., Plavec I., Forestell S., Su L., Bohnlein E., Kaneshima H. RevM10-expressing T cells derived in vivo from transduced human hematopoietic stem-progenitor cells inhibit human immunodeficiency virus replication. J. Virol. 71:1997;4707-4716.
19. Caputo A., Rossi C., Bozzini R., Betti M., Grossi M. P., Barbanti-Brodano G., Balboni P. G. Studies on the effect of the combined expression of anti-tat and anti-rev genes on HIV-1 replication. Gene Ther. 4:1997;288-295.
20. Ulich C., Harrich D., Estes P., Gaynor R. B. Inhibition of human immunodeficiency virus type 1 replication is enhanced by a combination of transdominant Tat and Rev proteins. J. Virol. 70:1996;4871-4876.
21. Banda N., Akkina R. K., Terrell K., Shpall E. J., Tomczak J., Campain J., Clamam H., Cagle L., Harrison G. S. Diphtheria toxin A gene-mediated HIV-1 protection of cord blood-derived T cells in the SCID-hu mouse model. J. Hematother. 7:1998;319-331.
22. Inouye R. T., Du B., Boldt-Houle D., Ferrante A., Park I.-W., Hammer S. M., Duan L., Groopman J. E., Pomerantz R. J., Terwilliger E. F. Potent inhibition of human immunodeficiency virus type 1 in primary T cells and alveolar macrophages by a combination anti-rev strategy delivered in an adeno-associated virus vector. J. Virol. 71:1997;4071-4078.
23. Kitamura Y., Ishikawa T., Okui N., Kobayashi N., Kanda T., Shimada T., Miyake K., Yoshiike K. 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. Retroviral. 20:1999;105-114.
24. Berger E. A., Doms R. W., Fenyo E.-M., Korber B. T. M., Littman D. R., Moore J. P., Sattentau Q. J., Schuitemaker H., Sodroski J., Weiss R. A. A new classification for HIV-1. Nature. 391:1998;240.
25. Mummidi S., Ahuja S. S., Gonzalez E., Anderson S. A., Santiago E. N., Stephan K. T., Craig F. E., O'Connell P., Tryon V., Clark R. A., Dolan M. J., Ahuja S. Genealogy of the CCR5 locus and chemokine system gene variants associated with altered rates of HIV-1 disease progression. Nat. Med. 4:1998;786-793.
26. Paxton W. A., Kang S., Koup R. A. The HIV type 1 coreceptor CCR5 and its role in viral transmission and disease progression. AIDS Res. Hum. Retroviruses. 14:1998;89-92.
27. Wyatt R., Sodroski J. The HIV-1 envelope glycoproteins: Fusogenes, antigens, and immunogens. Science. 280:1998;1884-1888.
28. Michael N., Louie L. G., Rohrbaugh A. L., Schultz K., Dayhoff D. E., Wang C. E., Sheppard H. W. The role of CCR5 and CCR2 polymorphisms in HIV-1 transmission and disease progression. Nat. Med. 3:1997;1160-1162.
29. Zhou C., Bahner I., Rossi J. J., Kohn D. B. Expression of hammerhead ribozymes by retroviral vectors to inhibit HIV-1 replication: Comparison of RNA levels and viral inhibition. Antisense Nucleic Acid Drug Dev. 6:1996;17-24.
30. Broxmeyer H. E., Douglas G. W., Hangoc G., Cooper S., Bard J., English D., Arny M., Thomas L., Boyse E. A. Human umbilical cord blood as a potential source of transplantable hematopoietic stem/progenitor cells. Proc. Natl. Acad. Sci. USA. 86:1989;3828-3832.
31. McCune J. M., Namikawa R., Kaneshima H., Shultz L. D., Lieberman M., Weissman I. L. The SCID-hu mouse: Murine model for the analysis of human hematolymphoid differentiation and function. Science. 241:1988;1632-1639.
32. Baum C. M., Weissman I. L., Tsukamoto A. S., Buckle A.-M., Peault B. Isolation of a candidate human hematopoietic stem-cell population. Proc. Natl. Acad. Sci. USA. 89:1992;2804-2808.
33. Brandt J. E., Srour E. F., van Besien K., Hoffman R. Characterization of human hematopoietic stem cells. Prog. Clin. Biol. Res. 352:1990;29-36.
34. Civin C. I., Strauss L. C., Fackler M. J., Trischman T. M., Wiley J. M., Loken M. R. Positive stem cell selection - Basic science. Bone Marrow Purging and Processing. 1990;A. R. Liss. p. 387-402.
35. + cells in vitro. Nat. Med. 2:1996;46-51.
36. Sutherland H. J., Eaves C. J. Long term culture of human myeloid cells. Culture of Hematopoietic Cells. 1994;Wiley-Liss, New York. p. 139-162.
37. Sutherland H. J., Lansdorp P. M., Henkelman D. H., Eaves A. C. Functional characterization of individual human hematopoietic stem cells cultured at limited dilution on supportive marrow stromal layers. Proc. Natl. Acad. Sci. USA. 87:1990;3584-3588.
38. Akkina R. K., Rosenblatt J. D., Campbell A. G., Chen I. S. Y., Zack J. A. Modeling human lymphoid precursor cell gene therapy in the SCID-hu mouse. Blood. 84:1994;1393-1398.
39. + cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G. J. Virol. 70:1996;2581-2585.
40. Brugger W., Heimfeld S., Berenson R. J., Mertelsmann R., Kanz L. Reconstitution of hematopoiesis after high-dose chemotherapy by autologous progenitor cells generated ex vivo. N. Engl. J. Med. 333:1995;283-316.
41. + precursor cells. J. Exp. Med. 174:1991;1283-1286.
42. Spangrude G. J., Heimfeld S., Weissman I. L. Purification and characterization of mouse hematopoietic stem cells. Science. 241:1988;58-62.
43. Raport C. J., Gosling J., Schweickart V. L., Gray P. W., Charo I. F. Molecular cloning and functional characterization of a novel human CC chemokine receptor (CCR5) for RANTES, MIP-1beta, and MIP-1alpha. J. Biol. Chem. 271:1996;17161-17166.
44. Burns J. C., Friedmann T., Driever W., Burrascano M., Yee J.-K. 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. 90:1993;8033-8037.
45. Gartner S., Markovits P., Markovitz D. M., Kaplan M. H., Gallo R. C., Popovic M. The role of mononuclear phagocytes in HTLV-III/LAV infection. Science. 233:1986;215-219.
46. Aldrovandi G. M., Feuer G., Gao L., Jamieson B., Kristeva M., Chen I. S., Zack J. A. The SCID-hu mouse as a model for HIV-1 infection. Nature. 363:1993;732-736.
47. Fauci A. S., Desrosiers R. C. Pathogenesis of HIV and SIV. Coffin J. M., Hughes S. H., Varmus H. E. Retroviruses. 1997;587-636 Cold Spring Harbor Laboratory Press, New York.
48. Deng H., Liu R., Ellmeier W., Choe S., Unutmaz D., Burkhart M., DiMarzio P., Marmon S., Sutton R. E., Hill C. M., Davis C. B., Peiper S. C., Schall T. J., Littman D. R., Landau N. R. Identification of a major-co-receptor for primary isolates of HIV-1. Nature. 381:1996;661-666.
49. Landau N. R., Littman D. R. Packaging system for rapid production of murine leukemia virus vectors with variable tropism. J. Virol. 66:1992;5110-5113.
50. Coffin J. M. HIV population dynamics in vivo: Implications for genetic variation, pathogenesis, and therapy. Science. 267:1995;483-489.
51. Gao F., Morrison S. G., Thorton C. L., Craig S., Karlsson G., Sodroski J., Morgado M., Galvao-Castro B., von Briesen H., Beddows S., Weber J., Sharp P. M., Shaw G. M., Hahn B. H. Molecular cloning and analysis of functional envelope genes from human immunodeficiency virus type 1 sequence subtypes A through G. J. Virol. 70:1996;1651-1667.
52. Korber B., Theiler J., Wolinsky S. Limitations of a molecular clock applied to considerations of the origin of HIV-1. Science. 1998;1868-1871.
53. Koyanagi Y., Miles S., Mitsuysu R. T., Merrill J. E., Vinters H. V., Chen I. S. Y. Dual infection of the central nervous system by AIDS viruses with distinct cellular tropisms. Science. 236:1987;819-822.
54. Modrow S., Hahn B. H., Shaw G. M., Gallo R. C., Wong-Staal F., Wolf H. Computer-assisted analysis of envelope protein sequences of seven human immunodeficiency virus isolates: Prediction of antigenic epitopes in conserved and variable regions. J. Virol. 61:1987;570-578.
55. Schuitemaker H., Koot M., Kootstra N. A., Dercksen M. W., de Goede R. E. Y., van Steenwijk R. P., Lange J. M. A., Schattenkerk E. J. K. M., Miedema F., Tersmette M. Biological phenotype of human immunodeficiency virus type 1 clones at different stages of infection: Progression of disease is associated with a shift from monocytotropic to T-cell tropic virus populations. J. Virol. 66:1992;1354-1360.
56. Wei X., Ghosh S. K., Taylor M. E., Johnson V. A., Emini E. A., Deutsh P., Lifson J. D., Bonhoeffer S., Nowak M. A., Hahn B. H., Saag M. S., Shaw G. M. Viral dynamics in human immunodeficiency virus type 1 infection. Nature. 373:1995;117-122.
57. Bolognesi D. P., Mattews T. J. Viral envelope fails to deliver. Science. 391:1998;638-639.
58. Letvin N. L. Progress in the development of an HIV vaccine. Science. 1998;1875-1879.
59. Yu M., Poeschla E., Yamada O., Degrandis P. A., Leavitt M. C., Heusch M., Yees J.-K., Wong-Staal F., Hampel A. In vitro and in vivo characterization of a second functional hairpin ribozyme against HIV-1. Virology. 206:1995;381-386.
60. Cairns J. S., D'Souza P. Chemokines and HIV-1 second receptors: The therapeutic connection. Nat. Med. 4:1998;563-568.
61. Moore J. Coreceptors: Implications for HIV pathogenesis and therapy. Science. 276:1997;51-52.
62. Cohen O. J., Paolucci S., Bende S. M., Daucher M., Moriuchi H., Moriuchi M., Cicala C., Davey R. T., Baird B., Fauci A. S. CXCR4 and CCR5 genetic polymorphisms in long-term nonprogressive human immunodeficiency virus infection: Lack of association with mutations other than CCR5-Δ32. J. Virol. 1998;6215-6217.
63. Biti R., French R., Young J., Bennets B., Stewart G., Tong Liang. HIV-1 infection in an individual homozygous for the CCR5 deletion allele. Nat. Med. 3:1997;252-253.
64. Lapham C. K., Zaitseva M. B., Lee S., Romanstseva T., Golding H. Fusion of monocytes and macrophages with HIV-1 correlates with biochemical properties of CXCR4 and CCR5. Nat. Med. 5:1999;590.
65. Wu J., Paxton W. A., Kassam N., Ruffing N., Rottman J. B., Sullivan N., Choe H., Sodroski J., Newman W., Koup R., Mackay C. R. CCR5 levels and expression pattern correlate with infectability by macrophage-tropic HIV-1, in vitro. J. Exp. Med. 185:1997;1681-1691.
66. Goila R., Banerjea A. C. Sequence specific cleavage of the HIV-1 coreceptor CCR5 gene by a hammer-head ribozyme and a DNA-enzyme: Inhibition of the coreceptor function by DNA enzyme. FEBS Lett. 436:1998;233-238.
67. González M. A., Serrano F., Llorente M., Abad J. L., García-Ortiz M. J., Bernad A. A hammerhead ribozyme targeted to the human chemokine receptor CCR5. Biochem. Biophys. Res. Commun. 251:1998;592-596.