Prospects for the therapeutic application of lentivirus-based gene therapy to HIV-1 infection

Current Gene Therapy

Volumn 8, Issue 1, 2008, Pages 1-8

  Yamamoto, Takuya ^a,b   Tsunetsugu Yokota, Yasuko ^a  

^a NATIONAL INSTITUTE OF INFECTIOUS DISEASES   (Japan)

^b UNIVERSITY OF TOKYO   (Japan)

Author keywords

Gene therapy; HIV infection; Lentivirus vector; RNAi; Vaccines

Indexed keywords

ADENOVIRUS VECTOR; ANTIRETROVIRUS AGENT; GAG PROTEIN; GLYCOPROTEIN GP 120; HUMAN IMMUNODEFICIENCY VIRUS VACCINE; IMMUNOSTIMULATING AGENT; LENTIVIRUS VECTOR; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; SHORT HAIRPIN RNA; SMALL INTERFERING RNA; VIRUS DNA;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ADENOVIRUS 5; ANTIBODY SPECIFICITY; ANTIGEN PRESENTATION; AUTOIMMUNITY; CD4+ T LYMPHOCYTE; CD8+ T LYMPHOCYTE; CELLULAR IMMUNITY; CLINICAL TRIAL; DENDRITIC CELL; DRUG EFFICACY; DRUG RESPONSE; DRUG SAFETY; GENE EXPRESSION SYSTEM; GENE MUTATION; GENE TARGETING; HELPER CELL; HIGHLY ACTIVE ANTIRETROVIRAL THERAPY; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; IMMUNOSTIMULATION; LYMPHOCYTE PROLIFERATION; MACROPHAGE; MEMORY T LYMPHOCYTE; NONHUMAN; NONVIRAL GENE THERAPY; REVIEW; RNA INTERFERENCE; SIMIAN IMMUNODEFICIENCY VIRUS; T LYMPHOCYTE ACTIVATION; VACCINE PRODUCTION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIREMIA; VIRUS CELL INTERACTION; VIRUS GENE; VIRUS GENOME; VIRUS INHIBITION; VIRUS LOAD; VIRUS REPLICATION; VIRUS RESISTANCE; VIRUS STRAIN;

GENE THERAPY; HIV INFECTIONS; HIV-1; HUMANS; LENTIVIRUS;

HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRUS;

EID: 41749106705     PISSN: 15665232     EISSN: None     Source Type: Journal    
DOI: 10.2174/156652308783688536     Document Type: Review

References (59)

1. Pierson T, McArthur J, Siliciano RF. Reservoirs for HIV-1: mechanisms for viral persistence in the presence of antiviral immune responses and antiretroviral therapy. Annu Rev Immunol (18): 665-708 (2000).
2. Aiuti F, Mezzaroma, I. Failure to reconstitute CD4+ T-cells despite suppression of HIV replication under HAART. AIDS Rev 8 (2): 88-97 (2006).
3. Autran B, Carcelain G, Li TS, et al. Positive effects of combined antiretroviral therapy on CD4+ T cell homeostasis and function in advanced HIV disease. Science 277 (5322): 112-6 (1997).
4. Li TS, Tubiana R, Katlama, C, et al. Long-lasting recovery in CD4 T-cell function and viral-load reduction after highly active antiretroviral therapy in advanced HIV-1 disease. Lancet 351 (9117): 1682-6 (1998).
5. Rosenberg ES, Billingsley JM, Caliendo AM, et al. Vigorous HIV-1-specific CD4+ T cell responses associated with control of viremia. Science 278 (5342): 1447-50 (1997).
6. Autran B, Carcelain G, Combadiere B, Debre P. Therapeutic vaccines for chronic infections. Science 305 (5681): 205-8 (2004).
7. Letvin NL. Progress and obstacles in the development of an AIDS vaccine. Nat Rev Immunol 6 (12): 930-9 (2006).
8. Elbashir SM, Harborth J, Lendeckel W, et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells targeted mRNA degradation by double-stranded RNA in vitro. Nature 411 (6836): 494-8 (2001).
9. Bumcrot D, Manoharan M, Koteliansky V, Sah DW. RNAi therapeutics: a potential new class of pharmaceutical drugs. Nat Chem Biol 2 (12): 711-9 (2006).
10. Kim DH, Rossi JJ. Strategies for silencing human disease using RNA interference. Nat Rev Genet 8 (3): 173-84 (2007).
11. Morris KV, Rossi JJ. Lentivirus-mediated RNA interference therapy for human immunodeficiency virus type 1 infection. Hum Gene Ther 17 (5): 479-86 (2006).
12. Nekhai S, Jerebtsova M. Therapies for HIV with RNAi. Curr Opin Mol Ther 8 (1): 52-61 (2006).
13. Scherer L, Rossi JJ, Weinberg MS. Progress and prospects: RNA-based therapies for treatment of HIV infection. Gene Ther 14 (14): 1057-64 (2007).
14. Wiznerowicz M, Trono D. Harnessing HIV for therapy, basic research and biotechnology. Trends Biotechnol 23 (1): 42-7 (2005).
15. Cohen J. Public health. AIDS vaccine trial produces disappointment and confusion. Science 299 (5611): 1290-1 (2003).
16. Graham BS, Mascola JR. Lessons from failure-preparing for future HIV-1 vaccine efficacy trials. J Infect Dis 191 (5): 647-9 (2005).
17. Humbert M, Dietrich U. The role of neutralizing antibodies in HIV infection. AIDS Rev 8 (2): 51-9 (2006).
18. Haynes BF, Fleming J, St Clair EW, et al. Cardiolipin polyspecific autoreactivity in two broadly neutralizing HIV-1 antibodies. Science 308 (5730): 1906-8 (2005).
19. Borrow P, Lewicki H, Hahn BH, Shaw GM, Oldstone MB. Virus-specific CD8+ cytotoxic T-lymphocyte activity associated with control of viremia in primary human immunodeficiency virus type 1 infection. J Virol 68 (9): 6103-10 (1994).
20. Koup RA, Safrit JT, Cao Y, et al. Temporal association of cellular immune responses with the initial control of viremia in primary human immunodeficiency virus type 1 syndrome. J Virol 68 (7): 4650-5 (1994).
21. Schmitz JE, Kuroda MJ, Santra S, et al. Control of viremia in simian immunodeficiency virus infection by CD8+ lymphocytes. Science 283 (5403): 857-60 (1999).
22. Matano T, Kobayashi M, Igarashi H, et al. Cytotoxic T lymphocyte-based control of simian immunodeficiency virus replication in a preclinical AIDS vaccine trial. J Exp Med 199 (12): 1709-18 (2004).
23. Capodici J, Kariko K, Weissman D. Inhibition of HIV-1 infection by small interfering RNA-mediated RNA interference. J Immunol 169 (9): 5196-201 (2002).
24. Dave RS, Pomerantz RJ. Antiviral effects of human immunodeficiency virus type 1-specific small interfering RNAs against targets conserved in select neurotropic viral strains. J Virol 78 (24): 13687-96 (2004).
25. Jacque JM, Triques K, Stevenson M. Modulation of HIV-1 replication by RNA interference. Nature 418 (6896): 435-8 (2002).
26. Novina CD, Murray MF, Dykxhoom DM, et al. siRNA-directed inhibition of HIV-1 infection. Nat Med 8 (7): 681-6 (2002).
27. Song E, Lee SK, Dykxhoom DM, et al. Sustained small interfering RNA-mediated human immunodeficiency virus type 1 inhibition in primary macrophages. J Virol 77 (13): 7174-81 (2003).
28. Yamamoto T, Miyoshi H, Yamamoto N, et al. Lentivirus vectors expressing short hairpin RNAs against the U3-overlapping region of HIV nef inhibit HIV replication and infectivity in primary macrophages. Blood 108 (10): 3305-12 (2006).
29. Boden D, Pusch O, Lee F, Tucker L, Ramratnam B. Human immunodeficiency virus type 1 escape from RNA interference. J Virol 77 (21): 11531-5 (2003).
30. Das AT, Brummelkamp TR, Westerhout EM, et al. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J Virol 78 (5): 2601-5 (2004).
31. Sabariegos R, Gimenez-Barcons M, Tapia N, Clotet B, Martinez MA. Sequence homology required by human immunodeficiency virus type 1 to escape from short interfering RNAs. J Virol 80 (2): 571-7 (2006).
32. Engelhardt JF, Yang Y, Stratford-Perricaudet LD, et al. Direct gene transfer of human CFTR into human bronchial epithelia of xenografts with El-deleted adenoviruses. Nat Genet 4 (1): 27-34 (1993).
33. Campos SK, Barry MA. Current advances and future challenges in Adenoviral vector biology and targeting. Curr Gene Ther 7 (3): 189-204 (2007).
34. Gao G, Vandenberghe LH, Wilson JM. New recombinant serotypes of AAV vectors. Curr Gene Ther 5 (3): 285-97 (2005).
35. Mizukami H, Ozawa K. [Utility of AAV vectors derived from novel serotypes]. Yakugaku Zasshi 126 (11): 1021-8 (2006).
36. Vasileva A, Jessberger R. Precise hit: adeno-associated virus in gene targeting. Nat Rev Microbiol 3 (11): 837-47 (2005).
37. Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12 (3): 342-7 (2006).
38. Sinn PL, Sauter SL, McCray PB, Jr. Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production. Gene Ther 12 (14): 1089-98(2005).
39. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302 (5644): 415-9 (2003).
40. Aiuti A, Slavin S, Aker M, et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 296 (5577): 2410-3 (2002).
41. De Palma M, Montini E, Santoni de Sio FR, et al. Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 105 (6): 2307-15 (2005).
42. Schroder AR, Shinn P, Chen H, et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110 (4): 521-9 (2002).
43. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science 300 (5626): 1749-51 (2003).
44. Bai Y, Soda Y, Izawa K, et al. Effective transduction and stable transgene expression in human blood cells by a third-generation lentiviral vector. Gene Ther 10 (17): 1446-57 (2003).
45. Miyoshi H, Blomer U, Takahashi M, Gage FH, Verma IM. Development of a self-inactivating lentivirus vector. J Virol 72 (10): 8150-7 (1998).
46. Tan W, Dong Z, Wilkinson TA, Barbas CF, 3rd, Chow SA. Human immunodeficiency virus type 1 incorporated with fusion proteins consisting of integrase and the designed polydactyl zinc finger protein E2C can bias integration of viral DNA into a predetermined chromosomal region in human cells. J Virol 80 (4): 1939-48 (2006).
47. Tan W, Zhu K, Segal DJ, Barbas CF, 3rd, Chow SA. Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites. J Virol 78 (3): 1301-13 (2004).
48. Philippe S, Sarkis C, Barkats M, et al. Lentiviral vectors with a defective integrase allow efficient and sustained transgene expression in vitro and in vivo. Proc Natl Acad Sci U S A 103 (47): 17684-9 (2006).
49. Yanez-Munoz RJ, Balaggan KS, MacNeil A, et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 12 (3): 348-53(2006).
50. Hanawa H, Kelly PF, Nathwani AC, et al. Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol Ther 5 (3): 242-51 (2002).
51. Schauber CA, Tuerk MJ, Pacheco CD, Escarpe PA, Veres G. Lentiviral vectors pseudotyped with baculovirus gp64 efficiently transduce mouse cells in vivo and show tropism restriction against hematopoietic cell types in vitro. Gene Ther 11 (3): 266-75 (2004).
52. Kobinger GP, Weiner DJ, Yu QC, Wilson JM. Filovirus-pseudotyped lentiviral vector can efficiently and stably transduce airway epithelia in vivo. Nat Biotechnol 19 (3): 225-30 (2001).
53. Liu SL, Halbert CL, Miller AD. Jaagsiekte sheep retrovirus envelope efficiently pseudotypes human immunodeficiency virus type 1-based lentiviral vectors. J Virol 78 (5): 2642-7 (2004).
54. Yang L, Bailey L, Baltimore D, Wang P. Targeting lentiviral vectors to specific cell types in vivo. Proc Natl Acad Sci U S A 103 (31): 11479-84 (2006).
55. McNeil AC, Shupert WL, Iyasere CA, et al. High-level HIV-1 viremia suppresses viral antigen-specific CD4(+) T cell proliferation. Proc Natl Acad Sci U S A 98 (24): 13878-83 (2001).
56. Younes SA, Yassine-Diab B, Dumont AR, et al. HIV-1 viremia prevents the establishment of interleukin 2-producing HIV-specific memory CD4+ T cells endowed with proliferative capacity. J Exp Med 198 (12): 1909-22 (2003).
57. Jansen CA, De Cuyper IM, Steingrover R, et al. Analysis of the effect of highly active antiretroviral therapy during acute HIV-1 infection on HIV-specific CD4 T cell functions. Aids 19 (11): 1145-54 (2005).
58. Douek DC, Brenchley JM, Betts MR, et al. HIV preferentially infects HIV-specific CD4+ T cells. Nature 417 (6884): 95-8 (2002).
59. Rana TM. Illuminating the silence: understanding the structure and function of small RNAs. Nat Rev Mol Cell Biol 8 (1): 23-36 (2007).