Modified human immunodeficiency virus-based lentiviral vectors display decreased sensitivity to trans-dominant Rev

Human Gene Therapy

Volumn 11, Issue 6, 2000, Pages 895-908

  Mautino, Mario R ^a   Ramsey, W Jay ^a   Reiser, Jakob ^b   Morgan, Richard A ^a  

^a NATIONAL HUMAN GENOME RESEARCH INSTITUTE   (United States)

^b NATIONAL INSTITUTES OF HEALTH   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DNA; MESSENGER RNA; REV PROTEIN; VIRUS VECTOR;

ARTICLE; HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRINAE; MUTATION; NONHUMAN; POLYMERASE CHAIN REACTION;

BASE SEQUENCE; CELL LINE; GENE PRODUCTS, REV; GENES, DOMINANT; GENETIC VECTORS; HIV; HUMANS; MUTATION; PURINES; RETROVIRUSES, SIMIAN; RNA SPLICING; TRANSGENES;

HUMAN IMMUNODEFICIENCY VIRUS; LENTIVIRUS; RNA VIRUSES; SIMIAN AIDS RETROVIRUS SRV-1;

EID: 0034630353     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/10430340050015509     Document Type: Article

References (57)

1. ADACHI, A., GENDELMAN, H.E., KOENIG, S., FOLKS, T., WILLEY, R., RABSON, A., and MARTIN, M.A. (1986). Production of acquired immunodeficiency syndrome-associated retrovirus in human and nonhuman cells transfected with an infectious molecular clone. J. Virol. 59, 284-291.
2. BIGNER, D.D., BIGNER, S.H., PONTEN, J., WESTERMARK, B., MAHALEY, M.S., RUOSLAHTI, E., HERSCHMAN, H., ENG, L.F., and WIKSTRAND, C.J. (1981). Heterogeneity of genotypic and phenotypic characteristics of fifteen permanent cell lines derived from human gliomas. J. Neuropathol. Exp. Neurol. 40, 201-229.
3. BORG, K.T., FAVARO, J.P., and ARRIGO, S.J. (1997). Involvement of human immunodeficiency virus type-1 splice sites in the cytoplasmic accumulation of viral RNA. Virology 236, 95-103.
4. BUKOVSKY A.A., SONG, J.P., and NALDINI, L. (1999). Interaction of human immunodeficiency virus-derived vectors with wild-type virus in transduced cells. J. Virol. 73, 7087-7092.
5. BUNNELL, B.A., METZGER, M., BYRNE, E., MORGAN, R.A., and DONAHUE, R.E. (1997). Efficient in vivo marking of primary CD4+ T lymphocytes in nonhuman primates using a gibbon ape leukemia virus-derived retroviral vector. Blood 89, 1987-1995.
6. CARA, A., RYBAK, S.M., NEWTON, D.L., CROWLEY, R., ROTTSCHAFER, S.E., REITZ, M.S., Jr., and GUSELLA, G.L. (1998). Inhibition of HIV-1 replication by combined expression of gag dominant negative mutant and a human ribonuclease in a tightly controlled HIV-1 inducible vector. Gene Ther. 5, 65-75.
7. CHANG, D.D., and SHARP, P.A. (1989). Regulation by HIV Rev depends upon recognition of splice sites. Cell 59, 789-795.
8. CHARNEAU, P., ALIZON, M., and CLAVEL, F. (1992). A second origin of DNA plus-strand synthesis is required for optimal human immunodeficiency virus replication. J. Virol. 66, 2814-2820.
9. CHARNEAU, P., MIRAMBEAU, G., ROUX, P., PAULOUS, S., BUC, H., and CLAVEL, F. (1994). HIV-1 reverse transcription. A termination step at the center of the genome. J. Mol. Biol. 241, 651-662.
10. CLEVER, J.L., ECKSTEIN, D.A., and PARSLOW, T.G. (1999). Genetic dissociation of the encapsidation and reverse transcription functions in the 5′ R region of human immunodeficiency virus type 1. J. Virol. 73, 101-109.
11. CORBEAU P., and WONG-STAAL, F. (1998). Anti-HIV effects of HIV vectors. Virology 243, 268-274.
12. CUI, Y., IWAKUMA, T., and CHANG, L.J. (1999). Contributions of viral splice sites and cis-regulatory elements to lentivirus vector function. J. Virol. 73, 6171-6176.
13. D'AGOSTINO, D.M., FELBER, B.K., HARRISON, J.E., and PAVLAKIS, G.N. (1992). The Rev protein of human immunodeficiency virus type 1 promotes polysomal association and translation of gag/pol and vpu/env mRNAs. Mol. Cell. Biol. 12, 1375-1386.
14. DAS, A.T., KLAVER, B., and BERKHOUT, B. (1999). A hairpin structure in the R region of the human immunodeficiency virus type 1 RNA genome is instrumental in polyadenylation site selection. J. Virol. 73, 81-91.
15. DONAHUE, R.E., BUNNELL, B.A., ZINK, M.C., METZGER, M.E., WESTRO, R.P., KIRBY, M.R., UNANGST, T., CLEMENTS, J.E., and MORGAN, R.A. (1998). Reduction in SIV replication in rhesus macaques infused with autologous lymphocytes engineered with antiviral genes. Nature Med. 4, 181-186.
16. DROPULIC, B., HERMANKOVA, M., and PITHA, P.M. (1996). A conditionally replicating HIV-1 vector interferes with wild-type HIV-1 replication and spread. Proc. Natl. Acad. Sci. U.S.A. 93, 11103-11108.
17. ERNST, R.K., BRAY, M., REKOSH, D., and HAMMARSKJOLD, M.L. (1997). Secondary structure and mutational analysis of the Mason-Pfizer monkey virus RNA constitutive transport element. RNA 3, 210-222.
18. FENG, S., and HOLLAND, E.C. (1988). HIV-1 tat trans-activation requires the loop sequence within tar. Nature (London) 334, 165-167.
19. 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., and SILICIANO, R.F. (1997). Indentification of a reservoir for HIV-1 in patients on highly active antiretroviral therapy. Science 278, 1295-1300. [See comments].
20. 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., and SILICIANO, R.F. (1999). Latent infection of CD4+ T cells provides a mechanism for lifelong persistence of HIV-1, even in patients on effective combination therapy. Nature Med. 5, 512-517.
21. GRUTER, P., TABERNERO, C., VON KOBBE, C., SCHMITT, C., SAAVEDRA, C., BACHI, A., WILM, M., FELBER, B.K., and IZAURRALDE, E. (1998). TAP, the human homolog of Mex67p, mediates CTE-dependent RNA export from the nucleus. Mol. Cell 1, 649-659.
22. HAMMARSKJOLD, M.L., LI, H., REKOSH, D., and PRASAD, S. (1994). Human immunodeficiency virus env expression becomes Rev-independent if the env region is not defined as an intron. J. Virol. 68, 951-958.
23. HO, S.N., HUNT, H.D., HORTON, R.M., PULLEN, J.K., and PEASE, L.R. (1989). Site-directed mutagenesis by overlap extension using the polymerase chain reaction. Gene 77, 51-59.
24. HOPE, T.J., KLEIN, N.P., ELDER, M.E., and PARSLOW, T.G. (1992). Trans-dominant inhibition of human immunodeficiency virus type 1 Rev occurs through formation of inactive protein complexes. J. Virol. 66, 1849-1855.
25. HORTON, R.M., HUNT, H.D., HO, S.N., PULLEN, J.K., and PEASE, L.R. (1989). Engineering hybrid genes without the use of restriction enzymes: Gene splicing by overlap extension. Gene 77, 61-68.
26. KLASENS, B.I., THIESEN, M., VIRTANEN, A., and BERKHOUT, B. (1999). The ability of the HIV-1 AAUAAA signal to bind polyadenylation factors is controlled by local RNA structure. Nucleic Acids Res. 27, 446-454.
27. LAUGHREA, M., and JETTE, L. (1996). Kissing-loop model of HIV-1 genome dimerization: HIV-1 RNAs can assume alternative dimeric forms, and all sequences upstream or downstream of hairpin 248-271 are dispensable for dimer formation. Biochemistry 35, 1589-1598.
28. LEAVITT, M.C., YU, M., YAMADA, O., KRAUS, G., LOONEY, D., POESCHLA, E., and WONG-STAAL, F. (1994). Transfer of an anti-HIV-1 ribozyme gene into primary human lymphocytes. Hum. Gene Ther. 5, 1115-1120.
29. LI, X., GERVAIX, A., KANG, D., LAW, P., SPECTOR, S.A., HO, A.D., and WONG-STAAL, F. (1998). Gene therapy targeting cord blood-derived CD34+ cells from HIV-exposed infants: Preclinical studies. Gene Ther. 5, 233-239.
30. LU, X.B., HEIMER, J., REKOSH, D., and HAMMARSKJOLD, M.L. (1990). U1 small nuclear RNA plays a direct role in the formation of a rev-regulated human immunodeficiency virus env mRNA that remains unspliced. Proc. Natl. Acad. Sci. U.S.A. 87, 7598-7602.
31. MALIM, M.H., BOHNLEIN, S., HAUBER, J., and CULLEN, B.R. (1989). Functional dissection of the HIV-1 Rev trans-activator -derivation of a trans-dominant repressor of Rev function. Cell 58, 205-214.
32. MARQUET, R., PAILLART, J.C., SKRIPKIN, E., EHRESMAN, C., and EHRESMANN, B. (1994). Dimerization of human immunodeficiency virus type 1 RNA involves sequences located upstream of the splice donor site. Nucleic Acids Res. 22, 145-151.
33. MASUDA, T., KURODA, M.J., and HARADA, S. (1998). Specific and independent recogintion of U3 and U5 att sites by human immunodeficiency virus type 1 integrase in vivo. J. Virol. 72, 8396-8402.
34. McBRIDE, M.S., SCHWARTZ, M.D., and PANGANIBAN, A.T. (1997). Efficient encapsidation of human immunodeficiency virus type 1 vectors and further characterization of cis elements required for encapsidation. J. Virol. 71, 4544-4554.
35. MIKAELIAN, I., KRIEG, M., GAIT, M.J., and KARN, J. (1996). Interactions of INS (CRS) elements and the splicing machinery regulate the production of Rev-responsive mRNAs. J. Mol. Biol. 257, 246-264.
36. MILLER, A.D., and ROSMAN, G.J. (1989). Improved retroviral vectors for gene transfer and expression. BioTechniques 7, 980-982, 984-986, 989-990.
37. MOCHIZUKI, H., SCHWARTZ, J.P., TANAKA, K., BRADY, R.O., and REISER, J. (1998). High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J. Virol. 72, 8873-8883.
38. MORGAN, R.A., and WALKER, R. (1996). Gene therapy for AIDS using retroviral mediated gene transfer to deliver HIV-1 antisense TAR and transdominant Rev protein genes to syngeneic lymphocytes in HIV-1 infected identical twins. Hum. Gene Ther. 7, 1281-1306.
39. NALDINI, L., BLOMER, U., GAGE, F.H., TRONO, D., and VERMA, I.M. (1996a). Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector. Proc. Natl. Acad. Sci. U.S.A. 93, 11382-11388.
40. NALDINI, L., BLOMER, U., GALLAY, P., ORY, D., MULLIGAN, R., GAGE, F.H., VERMA, I.M., and TRONO, D. (1996B). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267. [See comments].
41. NASIOULAS, G., ZOLOTUKHIN, A.S., TABERNERO, C., SOLOMIN, L., CUNNINGHAM, C.P., PAVLAKIS, G.N., and FELBER, B.K. (1994). Elements distinct from human immunodeficiency virus type 1 splice sites are responsible for the Rev dependence of env mRNA. J. Virol. 68, 2986-2993.
42. PEAR, W.S., NOLAN, G.P., SCOTT, M.L., and BALTIMORE D. (1993). Production of high-titer helper-free retroviruses by transient transfection. Proc. Natl. Acad. Sci. U.S.A. 90, 8392-8396.
43. POLLARD, V.W., and MALIM, M.H. (1998). The HIV-1 Rev protein. Annu. Rev. Microbiol. 52, 491-532.
44. PULLEN, K.A., and CHAMPOUX, J.J. (1990). Plus-strand origin for human immunodeficiency virus type 1: Implications for integration. J. Virol. 64, 6274-6277.
45. RAGHEB, J.A., BRESSLER, P., DAUCHER, M., CHIANG, L., CHUAH, M.K., VANDENDRIESSCHE, T., and MORGAN, R.A. (1995). Analysis of trans-dominant mutants of the HIV type 1 Rev protein for their ability to inhibit Rev function, HIV type 1 replication, and their use as anti-HIV gene therapeutics. AIDS Res. Hum. Retroviruses 11, 1343-1353.
46. RAGHEB, J.A., COUTURE, L., MULLEN, C., RIDGWAY, A., and MORGAN, R.A. (1999). Inhibition of human immunodeficiency virus type 1 by Tat/Rev-regulated expression of cytosine deaminase, interferon alpha2, or diphtheria toxin compared with inhibition by transdominant Rev. Hum. Gene Ther. 10, 103-112.
47. RANGA, U., WOFFENDIN, C., VERMA, S., XU, L., JUNE, C.H., BISHOP, D.K., and NABEL, G.J. (1998). Enhanced T cell engraftment after retroviral delivery of an antiviral gene in HIV-infected individuals. Proc. Natl. Acad. Sci. U.S.A. 95, 1201-1206.
48. REISER, J., HARMISON, G., KLUEPFEL-STAHL, S., BRADY, R.O., KARLSSON, S., and SCHUBERT, M. (1996). Transduction of non-dividing cells using pseudotyped defective high-titer HIV type 1 particles. Proc. Natl. Acad. Sci. U.S.A. 93, 15266-15271.
49. RHIM, H., PARK, J., and MORROW, C.D. (1991). Deletions in the tRNA(Lys) primer-binding site of human immunodeficiency virus type 1 identify essential regions for reverse transcription. J. Virol. 65, 4555-4564.
50. RICHARDSON, J.H., CHILD, L.A., and LEVER, A.M. (1993). Packaging of human immunodeficiency virus type 1 RNA requires cis-acting sequences outside the 5′ leader region. J. Virol. 67, 3997-4005.
51. SAAVEDRA, C., FELBER, B., and IZAURRALDE, E. (1997). The simian retrovirus-1 constitutive transport element, unlike the HIV-1 RRE, uses factors required for cellular mRNA export. Curr. Biol. 7, 619-628.
52. TAN, W., SCHALLING, M., ZHAO, C., LUUKKONEN, M., NILSSON, M., FENYÖ, E.M., PAVLAKIS, G.N., and SCHWARTZ, S. (1996). Inhibitory activity of the equine infectious anemia virus major 5′ splice site in the absence of Rev. J. Virol. 70, 3645-3658.
53. TANG, H., GAIETTA, G.M., FISCHER, W.H., ELLISMAN, M.H., and WONG-STAAL, F. (1997). A cellular cofactor for the constitutive transport element of type D retrovirus. Science 276, 1412-1415.
54. TRONO, D., FEINBERG, M.B., and BALTIMORE, D. (1989). HIV-1 Gag mutants can dominantly interfere with the replication of the wild-type virus. Cell 59, 113-120.
55. VANDENDRIESSCHE, T., CHUAH, M.K., CHIANG, L., CHANG, H.K., ENSOLI, B., and MORGAN, R.A. (1995). Inhibition of clinical human immunodeficiency virus (HIV) type 1 isolates in primary CD4+ T lymphocytes by retroviral vectors expressing anti-HIV genes. J. Virol. 69, 4045-4052.
56. WONG-STAAL, F., POESCHLA, E.M., and LOONEY, D.J. (1998). A controlled, phase 1 clinical trial to evaluate the safety and effects in HIV-1 infected humans of autologous lymphocytes transduced with a ribozyme that cleaves HIV-1 RNA. Hum. Gene Ther. 9, 2407-2425.
57. ZOLOTUKHIN, A.S., VALENTIN, A., PAVLAKIS, G.N., and FELBER, B.K. (1994). Continuous propagation of RRE(-) and Rev(-)RRE(-) human immunodeficiency virus type 1 molecular clones containing a cis-acting element of simian retrovirus type 1 in human peripheral blood lymphocytes. J. Virol. 68, 7944-7952.