Lentiviral vectors: Excellent tools for experimental gene transfer and promising candidates for gene therapy

Journal of Gene Medicine

Volumn 2, Issue 5, 2000, Pages 308-316

  Vigna, Elisa ^a   Naldini, Luigi ^a,b  

^a UNIVERSITY OF TURIN   (Italy)

^b UNIVERSITY OF TURIN   (Italy)

Author keywords

Biosafety; Gene therapy; Gene transfer; HIV derived vector; Lentiviral vector; Non primate lentiviral vectors

Indexed keywords

PRIMATES;

ANIMAL; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; LENTIVIRINAE; METHODOLOGY; REVIEW; SAFETY;

ANIMALS; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HIV-1; HUMANS; LENTIVIRUS; SAFETY;

EID: 0034278156     PISSN: 1099498X     EISSN: None     Source Type: Journal    
DOI: 10.1002/1521-2254(200009/10)2:5<308::aid-jgm131>3.0.co;2-3     Document Type: Review

References (84)

1. Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector [see comments]. Science 1996; 272: 263-267.
2. Akkina RK, Walton RM, Chen ML, Li QX, Planelles V, Chen IS. High-efficiency gene transfer into CD34+ cells with a human immunodeficiency virus type 1-based retroviral vector pseudo-typed with vesicular stomatitis virus envelope glycoprotein G. J Virol 1996; 70: 2581-2585.
3. Reiser J, Harmison G, Kluepfel-Stahl S, Brady RO, Karlsson S, Schubert M. Transduction of nondividing cells using pseudo-typed defective high-titer HIV type 1 particles. Proc Natl Acad Sci U S A 1996; 93: 15266-15271.
4. Mann R, Mulligan RC, Baltimore D. Construction of a retrovirus packaging mutant and its use to produce helper-free defective retrovirus. Cell 1983; 33: 153-159.
5. Miller AD, Miller DG, Garcia JV, Lynch CM. Use of retroviral vectors for gene transfer and expression. Methods Enzymol 1993; 217: 581-599.
6. Naldini L. Lentiviruses as gene transfer agents for delivery to non-dividing cells. Curr Opin Biotechnol 1998; 9: 457-463.
7. Palmer TD, Rosman GJ, Osborne WR, Miller AD. Genetically modified skin fibroblasts persist long after transplantation but gradually inactivate introduced genes. Proc Natl Acad Sci U S A 1991; 88: 1330-1334.
8. Challita PM, Kohn DB. Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci U S A 1994; 91: 2567-2571.
9. Fenjves ES, Yao SN, Kurachi K, Taichman LB. Loss of expression of a retrovirus-transduced gene in human keratinocytes. J Invest Dermatol 1996; 106: 576-578.
10. Ghazizadeh S, Carroll JM, Taichman LB. Repression of retrovirus-mediated transgene expression by interferons: implications for gene therapy. J Virol 1997; 71: 9163-9169.
11. Chen WY, Bailey EC, McCune SL, Dong JY, Townes TM. Reactivation of silenced, virally transduced genes by inhibitors of histone deacetylase. Proc Natl Acad Sci U S A 1997; 94: 5798-5803.
12. Cupelli L, Okenquist SA, Trubetskoy A, Lenz J. The secondary structure of the R region of a murine leukemia virus is important for stimulation of long terminal repeat-driven gene expression. J Virol 1998; 72: 7807-7814.
13. Oshima M, Odawara T, Hanaki K, Igarashi H, Yoshikura H. cis Elements required for high-level expression of unspliced Gag-containing message in Moloney murine leukemia virus. J Virol 1998; 72: 6414-6420.
14. King JA, Bridger JM, Gounari F, et al. The extended packaging sequence of MoMLV contains a constitutive mRNA nuclear export function. FEBS Lett 1998; 434: 367-371.
15. Deffaud C, Darlix JL. Characterization of an internal ribosomal entry segment in the 5′ leader of murine leukemia virus env RNA. J Virol 2000; 74: 846-850.
16. Drysdale CM, Pavlakis GN. Rapid activation and subsequent down-regulation of the human immunodeficiency virus type 1 promoter in the presence of Tat: possible mechanisms contributing to latency. J Virol 1991; 65: 3044-3051.
17. Miyoshi H, Takahashi M, Gage FH, Verma IM. Stable and efficient gene transfer into the retina using an HIV-based lentiviral vector. Proc Natl Acad Sci U S A 1997; 94: 10319-10323.
18. Kung SK, An DS, Chen IS. A murine leukemia virus (MuLV) long terminal repeat derived from rhesus macaques in the context of a lentivirus vector and MuLV gag sequence results in high-level gene expression in human T lymphocytes. J Virol 2000; 74: 3668-3681.
19. Zufferey R, Donello JE, Trono D, Hope TJ. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 1999; 73: 2886-2892.
20. Deglon N, Tseng JL, Bensadoun JC, et al. Self-inactivating lentiviral vectors with enhanced transgene expression as potential gene transfer system in Parkinson's disease. Hum Gene Ther 2000; 11: 179-190.
21. Hu WS, Temin HM. Retroviral recombination and reverse transcription. Science 1990; 250: 1227-1233.
22. White SM, Renda M, Nam NY, et al. Lentivirus vectors using human and simian immunodeficiency virus elements. J Virol 1999; 73: 2832-2840.
23. Kaye JF, Lever AM. Nonreciprocal packaging of human immunodeficiency virus type 1 and type 2 RNA: a possible role for the p2 domain of Gag in RNA encapsidation. J Virol 1998; 72: 5877-5885.
24. zurMegede J, Chen MC, Doe B, et al. Increased expression and immunogenicity of sequence-modified human immunodeficiency virus type 1 gag gene. J Virol 2000; 74: 2628-2635.
25. Cullen BR. HIV-1 auxiliary proteins: making connections in a dying cell. Cell 1998; 93: 685-692.
26. Zufferey R, Nagy D, Mandel RJ, Naldini L, Trono D. Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 1997; 15: 871-875.
27. Gasmi M, Glynn J, Jin MJ, Jolly DJ, Yee JK, Chen ST. Requirements for efficient production and transduction of human immunodeficiency virus type 1-based vectors. J Virol 1999; 73: 1828-1834.
28. Mochizuki H, Schwartz JP, Tanaka K, Brady RO, Reiser J. High-titer human immunodeficiency virus type 1-based vector systems for gene delivery into nondividing cells. J Virol 1998; 72: 8873-8883.
29. Kim VN, Mitrophanous K, Kingsman SM, Kingsman AJ. Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1. J Virol 1998; 72: 811-816.
30. Wei P, Garber ME, Fang SM, Fischer WH, Jones KA. A novel CDK9-associated C-type cyclin interacts directly with HIV-1 Tat and mediates its high-affinity, loop-specific binding to TAR RNA. Cell 1998; 92: 451-462.
31. Bieniasz PD, Grdina TA, Bogerd HP, Cullen BR. Recruitment of cyclin T1/P-TEFb to an HIV type 1 long terminal repeat promoter proximal RNA target is both necessary and sufficient for full activation of transcription. Proc Natl Acad Sci U S A 1999; 96: 7791-7796.
32. Ho DD, Neumann AU, Perelson AS, Chen W, Leonard JM, Markowitz M. Rapid turnover of plasma virions and CD4 lymphocytes in HIV-1 infection [see comments]. Nature 1995; 373: 123-126.
33. Dull T, Zufferey R, Kelly M, et al. A third-generation lentivirus vector with a conditional packaging system. J Virol 1998; 72: 8463-8471.
34. Neville M, Stutz F, Lee L, Davis LI, Rosbash M. The importin-beta family member Crm1p bridges the interaction between Rev and the nuclear pore complex during nuclear export. Curr Biol 1997; 7: 767-775.
35. Fornerod M, Ohno M, Yoshida M, Mattaj IW. CRM1 is an export receptor for leucine-rich nuclear export signals [see comments]. Cell 1997; 90: 1051-1060.
36. Stade K, Ford CS, Guthrie C, Weis K. Exportin 1 (Crm1p) is an essential nuclear export factor. Cell 1997; 90: 1041-1050.
37. Pollard VW, Malim MH. The HIV-1 Rev protein. Annu Rev Microbiol 1998; 52: 491-532.
38. Schneider R, Campbell M, Nasioulas G, Felber BK, Pavlakis GN. Inactivation of the human immunodeficiency virus type 1 inhibitory elements allows Rev-independent expression of Gag and Gag/protease and particle formation. J Virol 1997; 71: 4892-4903.
39. Valentin A, Aldrovandi G, Zolotukhin AS, et al. Reduced viral load and lack of CD4 depletion in SCID-hu mice infected with Rev-independent clones of human immunodeficiency virus type 1. J Virol 1997; 71: 9817-9822.
40. Indraccolo S, Feroli F, Minuzzo S, et al. DNA immunization of mice against SIVmac239 Gag and Env using Rev-independent expression plasmids. AIDS Res Hum Retroviruses 1998; 14: 83-90.
41. Kotsopoulou E, Kim VN, Kingsman AJ, Kingsman SM, Mitrophanous KA. A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene. J Virol 2000; 74: 4839-4852.
42. Zufferey R, Dull T, Mandel RJ, et al. Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 1998; 72: 9873-9880.
43. Miyoshi H, Blomer U, Takahashi M, Gage FH, Verma IM. Development of a self-inactivating lentivirus vector. J Virol 1998; 72: 8150-8157.
44. Iwakuma T, Cui Y, Chang LJ. Self-inactivating lentiviral vectors with U3 and US modifications. Virology 1999; 261: 120-132.
45. Bukovsky AA, Song JP, Naldini L. Interaction of human immunodeficiency virus-derived vectors with wild-type virus in transduced cells. J Virol 1999; 73: 7087-7092.
46. Bonyhadi ML, Kaneshima H. The SCID-hu mouse: an in vivo model for HIV-1 infection in humans. Mol Med Today 1997; 3: 246-253.
47. Naldini L, Blomer U, Gage FH, Trono D, Verma IM. 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 1996; 93: 11382-11388.
48. Johnston JC, Gasmi M, Lim LE, et al. Minimum requirements for efficient transduction of dividing and nondividing cells by feline immunodeficiency virus vectors. J Virol 1999; 73: 4991-5000.
49. Bartz SR, Vodicka MA. Production of high-titer human immunodeficiency virus type 1 pseudotyped with vesicular stomatitis virus glycoprotein. Methods 1997; 12: 337-342.
50. Aiken C. Pseudotyping human immunodeficiency virus type 1 (HIV-1) by the glycoprotein of vesicular stomatitis virus targets HIV-1 entry to an endocytic pathway and suppresses both the requirement for Nef and the sensitivity to cyclosporin A. J Virol 1997; 71: 5871-5877.
51. Follenzi A, Ailles LE, Bakovic S, Geuna M, Naldini L. Gene transduction by lentiviral vectors is limited by nuclear translocation and rescued by incorporation of HIV-1 pol sequences. Nat Genet 2000; 25: 217-222.
52. Zennou V, Petit C, Guetard D, Nerhbass U, Montagnier L, Chameau P. HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 2000; 101: 173-185.
53. Chameau P, Alizon M, Clavel F. A second origin of DNA plus-strand synthesis is required for optimal human immunodeficiency virus replication. J Virol 1992; 66: 2814-2820.
54. Chameau P, Mirambeau G, Roux P, Paulous S, Bue H, Clavel F. HIV-1 reverse transcription. A termination step at the center of the genome. J Mol Biol 1994; 241: 651-662.
55. Ilyinskii PO, Desrosiers RC. Identification of a sequence element immediately upstream of the polypurine tract that is essential for replication of simian immunodeficiency virus. EMBO J 1998; 17: 3766-3774.
56. Johnston J, Power C. Productive infection of human peripheral blood mononuclear cells by feline immunodeficiency virus: implications for vector development. J Virol 1999; 73: 2491-2498.
57. Poeschla EM, Wong-Staal F, Looney DJ. Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nat Med 1998; 4: 354-357.
58. Olsen JC. Gene transfer vectors derived from equine infectious anemia virus. Gene Ther 1998; 5: 1481-1487.
59. Mitrophanous K, Yoon S, Rohll J, et al. Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther 1999; 6: 1808-1818.
60. Mselli-Lakhal L, Favier C, Da Silva T, et al. Defective RNA packaging is responsible for low transduction efficiency of CAEV-based vectors. Arch Virol 1998; 143: 681-695.
61. Metharom P, Takyar S, Huiqin Xia H, et al. Novel bovine lentiviral vectors based on Jembrana disease virus. J Gene Med 2000; 2: 176-185.
62. Curran MA, Kaiser SM, Achacoso PL, Nolan GP. Efficient transduction of nondividing cells by optimized feline immunodeficiency virus vectors. Mol Ther 2000; 1: 31-38.
63. Wang G, Slepushkin V, Zabner J, et al. Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect [see comments]. J Clin Invest 1999; 104: R55-R62.
64. Blomer U, Naldini L, Kafri T, Trono D, Verma IM, Gage FH. Highly efficient and sustained gene transfer in adult neurons with a lentivirus vector. J Virol 1997; 71: 6641-6649.
65. Kordower JH, Bloch J, Ma SY, et al. Lentiviral gene transfer to the nonhuman primate brain. Exp Neurol 1999; 160: 1-16.
66. Blomer U, Kafri T, Randolph-Moore L, Verma IM, Gage FH. Bcl-xL protects adult septal cholinergic neurons from axotomized cell death. Proc Natl Acad Sci U S A 1998; 95: 2603-2608.
67. Galileo DS, Hunter K, Smith SB. Stable and efficient gene transfer into the mutant retinal pigment epithelial cells of the Mitf(vit) mouse using a lentiviral vector. Curr Eye Res 1999; 18: 135-142.
68. Takahashi M, Miyoshi H, Verma IM, Gage FH. Rescue from photoreceptor degeneration in the rd mouse by human immunodeficiency virus vector-mediated gene transfer. J Virol 1999; 73: 7812-7816.
69. Gallichan WS, Kafri T, Krahl T, Verma IM, Sarvetnick N. Lentivirus-mediated transduction of islet grafts with interleukin 4 results in sustained gene expression and protection from insulitis. Hum Gene Ther 1998; 9: 2717-2726.
70. Ju Q, Edelstein D, Brendel MD, et al. Transduction of non-dividing adult human pancreatic beta cells by an integrating lentiviral vector. Diabetologia 1998; 41: 736-739.
71. Giannoukakis N, Mi Z, Gambotto A, et al. Infection of intact human islets by a lentiviral vector. Gene Ther 1999; 6: 1545-1551.
72. Kafri T, Blomer U, Peterson DA, Gage FH, Verma IM. Sustained expression of genes delivered directly into liver and muscle by lentiviral vectors. Nat Genet 1997; 17: 314-317.
73. Park F, Ohashi K, Chiu W, Naldini L, Kay MA. Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet 2000; 24: 49-52.
74. Goldman MJ, Lee PS, Yang JS, Wilson JM. Lentiviral vectors for gene therapy of cystic fibrosis. Hum Gene Ther 1997; 8: 2261-2268.
75. Johnson LG, Olsen JC, Naldini L, Boucher RC. Pseudotyped human lentiviral vector-mediated gene transfer to airway epithelia in vivo. Gene Ther 7: 568-574.
76. Zhang Z, Schuler T, Zupancic M, et al. Sexual transmission and propagation of SIV and HIV in resting and activated CD4+ T cells. Science 1999; 286: 1353-1357.
77. Unutmaz D, KewalRamani VN, Marmon S, Littman DR. Cytokine signals are sufficient for HIV-1 infection of resting human T lymphocytes. J Exp Med 1999; 189: 1735-1746.
78. Kinoshita S, Chen BK, Kaneshima H, Nolan GP. Host control of HIV-1 parasitism in T cells by the nuclear factor of activated T cells. Cell 1998; 95: 595-604.
79. Uchida N, Sutton RE, Friera AM, 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 U S A 1998; 95: 11939-11944.
80. Case SS, Price MA, Jordan CT, et al. Stable transduction of quiescent CD34(+)CD38(-) human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci U S A 1999; 96: 2988-2993.
81. Miyoshi H, Smith KA, Mosier DE, Verma IM, Torbett BE. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 1999; 283: 682-686.
82. Evans JT, Kelly PF, O'Neill E, Garcia JV. Human cord blood CD34+CD38- cell transduction via lentivirus-based gene transfer vectors. Hum Gene Ther 1999; 10: 1479-1489.
83. Chang LJ, Urlacher V, Iwakuma T, Cui Y, Zucali J. Efficacy and safety analyses of a recombinant human immunodeficiency virus type 1 derived vector system. Gene Ther 1999; 6: 715-728.
84. An DS, Wersto RP, Agricola BA, et al. Marking and gene expression by a lentivirus vector in transplanted human and nonhuman primate CD34(+) cells. J Virol 2000; 74: 1286-1295.