Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy

Molecular Therapy

Volumn 12, Issue 5, 2005, Pages 900-909

  Li, Ming Jie ^a   Kim, James ^a   Li, Shirley ^a   Zaia, John ^a   Yee, Jiing Kuan ^a   Anderson, Joseph ^b   Akkina, Ramesh ^b   Rossi, John J ^a  

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

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

Author keywords

CCR5; HIV AIDS gene therapy; Lentiviral vector; Ribozyme; RNA decoy; shRNA; TAR; Transgenic macrophages

Indexed keywords

CHEMOKINE RECEPTOR CCR5; HUMAN IMMUNODEFICIENCY VIRUS ANTIBODY; LENTIVIRUS VECTOR; MESSENGER RNA; RIBOZYME; SHORT HAIRPIN RNA; SMALL NUCLEOLAR RNA;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ANTIVIRAL ACTIVITY; ARTICLE; CD34 SELECTION; CELL TYPE; CONTROLLED STUDY; DISEASE COURSE; GENE CASSETTE; GENE EXPRESSION; GENE TARGETING; GENETIC TRANSCRIPTION; GENETIC TRANSFECTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; LONG TERM CARE; PROMOTER REGION; RNA VECTOR; VALIDATION PROCESS; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS LOAD; VIRUS REPLICATION;

CELLS, CULTURED; DNA POLYMERASE III; GENE EXPRESSION; GENE TRANSFER TECHNIQUES; GENES, TAT; GENETIC VECTORS; HIV INFECTIONS; HIV-1; HUMANS; LENTIVIRUS; LYMPHOCYTES; PROMOTER REGIONS (GENETICS); RECEPTORS, CCR5; RNA, CATALYTIC; TRANSDUCTION, GENETIC; TRANSGENES;

HUMAN IMMUNODEFICIENCY VIRUS; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS;

EID: 27744605569     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2005.07.524     Document Type: Article

References (46)

1. J. Martinez-Picado et al. Antiretroviral resistance during successful therapy of HIV type 1 infection Proc. Natl. Acad. Sci. USA 97 2000 10948-10953
2. M.A. Winters et al. Frequency of antiretroviral drug resistance mutations in HIV-1 strains from patients failing triple drug regimens. The Terry Beirn Community Programs for Clinical Research on AIDS Antiviral Ther. 5 2000 57-63
3. L.T. Bacheler et al. Human immunodeficiency virus type 1 mutations selected in patients failing efavirenz combination therapy Antimicrob. Agents Chemother. 44 2000 2475-2484
4. L. Naldini U. Blomer F.H. Gage D. Trono and I.M. Verma 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 1996 11382-11388
5. L. Naldini et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector Science 272 1996 263-267
6. U. Blomer et al. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector J. Virol. 71 1997 6641-6649
7. T. Kafri U. Blomer D.A. Peterson F.H. Gage and I.M. Verma Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors Nat. Genet. 17 1997 314-317
8. C.D. Novina et al. siRNA-directed inhibition of HIV-1 infection Nat. Med. 8 2002 681-686
9. + cells Blood 2004
10. F. Galimi and I.M. Verma Opportunities for the use of lentiviral vectors in human gene therapy Curr. Top. Microbiol. Immunol. 261 2002 245-254
11. P.A. Sharp RNA interference-2001 Genes Dev. 15 2001 485-490
12. S.M. Elbashir et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells Nature 411 2001 494-498
13. T.R. Brummelkamp R. Bernards and R. Agami A system for stable expression of short interfering RNAs in mammalian cells Science 296 2002 550-553
14. J. Capodici K. Kariko and D. Weissman Inhibition of HIV-1 infection by small interfering RNA-mediated RNA interference J. Immunol. 169 2002 5196-5201
15. J.M. Jacque K. Triques and M. Stevenson Modulation of HIV-1 replication by RNA interference Nature 418 2002 435-438
16. N.S. Lee et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells Nat. Biotechnol. 20 2002 500-505
17. X.F. Qin D.S. An I.S. Chen and D. Baltimore Inhibiting HIV-1 infection in human T cells by lentiviral-mediated delivery of small interfering RNA against CCR5 Proc. Natl. Acad. Sci. USA 100 2003 183-188
18. M.J. Li et al. Inhibition of HIV-1 infection by lentiviral vectors expressing Pol III-promoted anti-HIV RNAs Mol. Ther. 8 2003 196-206
19. + progenitor cell-derived macrophages Mol. Ther. 8 2003 62-71
20. L. Gitlin J.K. Stone and R. Andino Poliovirus escape from RNA interference: Short interfering RNA-target recognition and implications for therapeutic approaches J. Virol. 79 2005 1027-1035
21. D. Boden O. Pusch F. Lee L. Tucker and B. Ramratnam Human immunodeficiency virus type 1 escape from RNA interference J. Virol. 77 2003 11531-11535
22. M. Emerman and M.H. Malim HIV-1 regulatory/accessory genes: Keys to unraveling viral and host cell biology Science 280 1998 1880-1884
23. M.H. Malim S. Bohnlein J. Hauber and B.R. Cullen Functional dissection of the HIV-1 Rev trans-activator-Derivation of a trans-dominant repressor of Rev function Cell 58 1989 205-214
24. H. Deng et al. Identification of a major co-receptor for primary isolates of HIV-1 Nature 381 1996 661-666
25. G. Alkhatib et al. CC CKR5: A RANTES, MIP-1alpha, MIP-1beta receptor as a fusion cofactor for macrophage-tropic HIV-1 Science 272 1996 1955-1958
26. M. Dean et al. Genetic restriction of HIV-1 infection and progression to AIDS by a deletion allele of the CKR5 structural gene. Hemophilia Growth and Development Study, Multicenter AIDS Cohort Study, Multicenter Hemophilia Cohort Study, San Francisco City Cohort, ALIVE Study Science 273 1996 1856-1862
27. R. Liu et al. Homozygous defect in HIV-1 coreceptor accounts for resistance of some multiply-exposed individuals to HIV-1 infection Cell 86 1996 367-377
28. L. Cagnon and J.J. Rossi Downregulation of the CCR5 beta-chemokine receptor and inhibition of HIV-1 infection by stable VA1-ribozyme chimeric transcripts Antisense Nucleic Acid Drug Dev. 10 2000 251-261
29. C. Dingwall v HIV-1 tat protein stimulates transcription by binding to a U-rich bulge in the stem of the TAR RNA structure EMBO J. 9 1990 4145-4153
30. J. Zhang et al. HIV-1 TAR RNA enhances the interaction between Tat and cyclin T1 J. Biol. Chem. 275 2000 34314-34319
31. A. Michienzi S. Li J.A. Zaia and J.J. Rossi A nucleolar TAR decoy inhibitor of HIV-1 replication Proc. Natl. Acad. Sci. USA 99 2002 14047-14052
32. P.Y. Yam et al. Design of HIV vectors for efficient gene delivery into human hematopoietic cells Mol. Ther. 5 2002 479-484
33. C. Svensson and J. Akusjarvi A novel effect of adenovirus VA RNA1 on cytoplasmic mRNA abundance Virology 174 1990 613-617
34. X. Lu et al. Antisense-mediated inhibition of human immunodeficiency virus (HIV) replication by use of an HIV type 1-based vector results in severely attenuated mutants incapable of developing resistance J. Virol. 78 2004 7079-7088
35. N. Uchida et al. HIV, but not murine leukemia virus, vectors mediate high efficiency gene transfer into freshly isolated G0/G1 human hematopoietic stem cells Proc. Natl. Acad. Sci. USA 95 1998 11939-11944
36. K. Yamada v Phenotype correction of Fanconi anemia group A hematopoietic stem cells using lentiviral vector Mol. Ther. 8 2003 600-610
37. M.A. Martinez B. Clotet and J.A. Este RNA interference of HIV replication Trends Immunol. 23 2002 559-561
38. D. Lawrence RNAi could hold promise in the treatment of HIV Lancet 359 2002 2007
39. W.Y. Hu F.D. Bushman and A.C. Siva RNA interference against retroviruses Virus Res. 102 2004 59-64
40. H. Nishitsuji et al. Expression of small hairpin RNA by lentivirus-based vector confers efficient and stable gene-suppression of HIV-1 on human cells including primary non-dividing cells Microbes Infect. 6 2004 76-85
41. N.S. Lee and J.J. Rossi Control of HIV-1 replication by RNA interference Virus Res. 102 2004 53-58
42. E.M. Westerhout M. Ooms M. Vink A.T. Das and B. Berkhout HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome Nucleic Acids Res. 33 2005 796-804
43. A.T. Das et al. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition J. Virol. 78 2004 2601-2605
44. A.J. Bridge S. Pebernard A. Ducraux A.L. Nicoulaz and R. Iggo Induction of an interferon response by RNAi vectors in mammalian cells Nat. Genet. 34 2003 263-264
45. C.E. Samuel Knockdown by RNAi-Proceed with caution Nat. Biotechnol. 22 2004 280-282
46. G. Lizee et al. Real-time quantitative reverse transcriptase-polymerase chain reaction as a method for determining lentiviral vector titers and measuring transgene expression Hum. Gene Ther. 14 2003 497-507