Lentiviral vectors: Basic to translational

Biochemical Journal

Volumn 443, Issue 3, 2012, Pages 603-618

  Sakuma, Toshie ^a   Barry, Michael A ^a   Ikeda, Yasuhiro ^a  

^a Mayo Clinic   (United States)

Author keywords

Cell targeting; Central polypurine tract (cPPT); Gene therapy; HIV 1; Lentivirus; Self inactivating (SIN)

Indexed keywords

ANTIRETROVIRUS AGENT; BETA GLOBIN; CHLORAMPHENICOL ACETYLTRANSFERASE; GAG PROTEIN; LENTIVIRUS VECTOR; LIGAND; NEF PROTEIN; POL PROTEIN; THROMBOSPONDIN; VIF PROTEIN; VPU PROTEIN;

ACQUIRED IMMUNE DEFICIENCY SYNDROME; ANTISENSE THERAPY; BETA THALASSEMIA; BIOENGINEERING; BIOSAFETY; BIOTECHNOLOGY; CELL LINE; CHROMATIN; CODON; GENE DELETION; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE INSERTION; GENE SILENCING; GENE TARGETING; HUMAN IMMUNODEFICIENCY VIRUS 1; HUMAN IMMUNODEFICIENCY VIRUS 1 INFECTION; IMMUNOMODULATION; IN VIVO STUDY; MURINE LEUKEMIA VIRUS; MUTAGENESIS; NONHUMAN; PRIORITY JOURNAL; PSEUDOTYPING; REVIEW; RNA INTERFERENCE; SYNTHESIS; TRANSGENICS; VESICULAR STOMATITIS VIRUS; VIRION; VIROLOGY; VIRUS GENOME; VIRUS REPLICATION; VIRUS TITRATION; WOODCHUCK HEPATITIS VIRUS;

GENETIC VECTORS; GENOME, VIRAL; HIV-1; PLASMIDS; PROTEIN BIOSYNTHESIS; VIRUS REPLICATION;

EID: 84860133437     PISSN: 02646021     EISSN: 14708728     Source Type: Journal    
DOI: 10.1042/BJ20120146     Document Type: Review

References (236)

1. Bukrinsky, M. I., Haggerty, S., Dempsey, M. P., Sharova, N., Adzhubel, A., Spitz, L., Lewis, P., Goldfarb, D., Emerman, M. and Stevenson, M. (1993) A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365, 666-669 (Pubitemid 23350541)
2. Freed, E. O. and Martin, M. A. (1994) HIV-1 infection of non-dividing cells. Nature 369, 107-108
3. Garvey, K. J., Oberste, M. S., Elser, J. E., Braun, M. J. and Gonda, M. A. (1990) Nucleotide sequence and genome organization of biologically active proviruses of the bovine immunodeficiency-like virus. Virology 175, 391-409 (Pubitemid 20133441)
4. Gojobori, T., Moriyama, E. N., Ina, Y., Ikeo, K., Miura, T., Tsujimoto, H., Hayami, M. and Yokoyama, S. (1990) Evolutionary origin of human and simian immunodeficiency viruses. Proc. Natl. Acad. Sci. U.S.A. 87, 4108-4111
5. Langley, R. J., Hirsch, V. M., O'Brien, S. J., Adger-Johnson, D., Goeken, R. M. and Olmsted, R. A. (1994) Nucleotide sequence analysis of puma lentivirus (PLV-14): genomic organization and relationship to other lentiviruses. Virology 202, 853-864 (Pubitemid 24254899)
6. Olmsted, R. A., Langley, R., Roelke, M. E., Goeken, R. M., Adger-Johnson, D., Goff, J. P., Albert, J. P., Packer, C., Laurenson, M. K. and Caro, T. M. (1992) Worldwide prevalence of lentivirus infection in wild feline species: epidemiologic and phylogenetic aspects. J. Virol. 66, 6008-6018
7. Olmsted, R. A., Hirsch, V. M., Purcell, R. H. and Johnson, P. R. (1989) Nucleotide sequence analysis of feline immunodeficiency virus: genome organization and relationship to other lentiviruses. Proc. Natl. Acad. Sci. U.S.A. 86, 8088-8092 (Pubitemid 19265325)
8. Smith, T. F., Srinivasan, A., Schochetman, G., Marcus, M. and Myers, G. (1988) The phylogenetic history of immunodeficiency viruses. Nature 333, 573-575
9. Sonigo, P., Alizon, M., Staskus, K., Klatzmann, D., Cole, S., Danos, O., Retzel, E., Tiollais, P., Haase, A. and Wain-Hobson, S. (1985) Nucleotide sequence of the visna lentivirus: relationship to the AIDS virus. Cell 42, 369-382 (Pubitemid 16237586)
10. von Schwedler, U. K., Stemmler, T. L., Klishko, V. Y., Li, S., Albertine, K. H., Davis, D. R. and Sundquist, W. I. (1998) Proteolytic refolding of the HIV-1 capsid protein amino-terminus facilitates viral core assembly. EMBO J. 17, 1555-1568 (Pubitemid 28119109)
11. Henderson, L. E., Bowers, M. A., Sowder, 2nd, R. C., Serabyn, S. A., Johnson, D. G., Bess, Jr, J. W., Arthur, L. O., Bryant, D. K. and Fenselau, C. (1992) Gag proteins of the highly replicative MN strain of human immunodeficiency virus type 1: posttranslational modifications, proteolytic processings, and complete amino acid sequences. J. Virol. 66, 1856-1865
12. Mervis, R. J., Ahmad, N., Lillehoj, E. P., Raum, M. G., Salazar, F. H., Chan, H. W. and Venkatesan, S. (1988) The gag gene products of human immunodeficiency virus type 1: alignment within the gag open reading frame, identification of posttranslational modifications, and evidence for alternative gag precursors. J. Virol. 62, 3993-4002
13. Fanales-Belasio, E., Raimondo, M., Suligoi, B. and Butto, S. (2010) HIV virology and pathogenetic mechanisms of infection: a brief overview. Ann. Ist. Super. Sanita 46, 5-14
14. Cimarelli, A. and Darlix, J. L. (2002) Assembling the human immunodeficiency virus type 1. Cell. Mol. Life Sci. 59, 1166-1184
15. Warrilow, D., Tachedjian, G. and Harrich, D. (2009) Maturation of the HIV reverse transcription complex: putting the jigsaw together. Rev. Med. Virol. 19, 324-337
16. Wei, P., Garber, M. E., Fang, S. M., Fischer, W. H. and Jones, K. A. (1998) 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 92, 451-462 (Pubitemid 28101109)
17. Malim, M. H., Hauber, J., Le, S. Y., Maizel, J. V. and Cullen, B. R. (1989) The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA. Nature 338, 254-257 (Pubitemid 19086926)
18. Haseltine, W. A. (1988) Replication and pathogenesis of the AIDS virus. J. Acquired Immune Defic. Syndr. 1, 217-240
19. Reference deleted
20. Terwilliger, E. F., Godin, B., Sodroski, J. G. and Haseltine, W. A. (1989) Construction and use of a replication-competent human immunodeficiency virus (HIV-1) that expresses the chloramphenicol acetyltransferase enzyme. Proc. Natl. Acad. Sci. U.S.A. 86, 3857-3861 (Pubitemid 19141844)
21. Helseth, E., Kowalski, M., Gabuzda, D., Olshevsky, U., Haseltine, W. and Sodroski, J. (1990) Rapid complementation assays measuring replicative potential of human immunodeficiency virus type 1 envelope glycoprotein mutants. J. Virol. 64, 2416-2420 (Pubitemid 20148494)
22. Page, K. A., Landau, N. R. and Littman, D. R. (1990) Construction and use of a human immunodeficiency virus vector for analysis of virus infectivity. J. Virol. 64, 5270-5276 (Pubitemid 20364514)
23. Landau, N. R., Page, K. A. and Littman, D. R. (1991) Pseudotyping with human T-cell leukemia virus type I broadens the human immunodeficiency virus host range. J. Virol. 65, 162-169
24. 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 (Pubitemid 23180724)
25. Burns, J. C., Friedmann, T., Driever, W., Burrascano, M. and Yee, J. K. (1993) 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. U.S.A. 90, 8033-8037 (Pubitemid 23260849)
26. Coil, D. A. and Miller, A. D. (2004) Phosphatidylserine is not the cell surface receptor for vesicular stomatitis virus. J. Virol. 78, 10920-10926 (Pubitemid 39299632)
27. + cells with a human immunodeficiency virus type 1-based retroviral vector pseudotyped with vesicular stomatitis virus envelope glycoprotein G. J. Virol. 70, 2581-2585
28. Naldini, L., Blomer, U., Gallay, P., Ory, D., Mulligan, R., Gage, F. H., Verma, I. M. and Trono, D. (1996) In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272, 263-267 (Pubitemid 26119138)
29. Fouchier, R. A., Simon, J. H., Jaffe, A. B. and Malim, M. H. (1996) Human immunodeficiency virus type 1 Vif does not influence expression or virion incorporation of gag-, pol-, and env-encoded proteins. J. Virol. 70, 8263-8269 (Pubitemid 26378400)
30. Harris, R. S., Bishop, K. N., Sheehy, A. M., Craig, H. M., Petersen-Mahrt, S. K., Watt, I. N., Neuberger, M. S. and Malim, M. H. (2003) DNA deamination mediates innate immunity to retroviral infection. Cell 113, 803-809 (Pubitemid 36724943)
31. Neil, S. J., Zang, T. and Bieniasz, P. D. (2008) Tetherin inhibits retrovirus release and is antagonized by HIV-1 Vpu. Nature 451, 425-430 (Pubitemid 351158893)
32. Sakuma, T., Noda, T., Urata, S., Kawaoka, Y. and Yasuda, J. (2009) Inhibition of Lassa and Marburg virus production by tetherin. J. Virol. 83, 2382-2385
33. Collins, K. L., Chen, B. K., Kalams, S. A., Walker, B. D. and Baltimore, D. (1998) HIV-1 Nef protein protects infected primary cells against killing by cytotoxic T lymphocytes. Nature 391, 397-401 (Pubitemid 28093515)
34. Zufferey, R., Nagy, D., Mandel, R. J., Naldini, L. and Trono, D. (1997) Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat. Biotechnol. 15, 871-875
35. Yu, S. F., von Ruden, T., Kantoff, P. W., Garber, C., Seiberg, M., Ruther, U., Anderson, W. F., Wagner, E. F. and Gilboa, E. (1986) Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl. Acad. Sci. U.S.A. 83, 3194-3198 (Pubitemid 16043003)
36. Miyoshi, H., Blomer, U., Takahashi, M., Gage, F. H. and Verma, I. M. (1998) Development of a self-inactivating lentivirus vector. J. Virol. 72, 8150-8157
37. Zufferey, R., Dull, T., Mandel, R. J., Bukovsky, A., Quiroz, D., Naldini, L. and Trono, D. (1998) Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880 (Pubitemid 28520853)
38. Iwakuma, T., Cui, Y. and Chang, L. J. (1999) Self-inactivating lentiviral vectors with U3 and U5 modifications. Virology 261, 120-132
39. Laspia, M. F., Rice, A. P. and Mathews, M. B. (1989) HIV-1 Tat protein increases transcriptional initiation and stabilizes elongation. Cell 59, 283-292 (Pubitemid 19264374)
40. Terwilliger, E., Burghoff, R., Sia, R., Sodroski, J., Haseltine, W. and Rosen, C. (1988) The art gene product of human immunodeficiency virus is required for replication. J. Virol. 62, 655-658 (Pubitemid 18064216)
41. Dull, T., Zufferey, R., Kelly, M., Mandel, R. J., Nguyen, M., Trono, D. and Naldini, L. (1998) A third-generation lentivirus vector with a conditional packaging system. J. Virol. 72, 8463-8471 (Pubitemid 28471781)
42. Kim, V. N., Mitrophanous, K., Kingsman, S. M. and Kingsman, A. J. (1998) Minimal requirement for a lentivirus vector based on human immunodeficiency virus type 1. J. Virol. 72, 811-816 (Pubitemid 28048905)
43. Escarpe, P., Zayek, N., Chin, P., Borellini, F., Zufferey, R., Veres, G. and Kiermer, V. (2003) Development of a sensitive assay for detection of replication-competent recombinant lentivirus in large-scale HIV-based vector preparations. Mol. Ther. 8, 332-341 (Pubitemid 37062843)
44. 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
45. Zennou, V., Petit, C., Guetard, D., Nerhbass, U., Montagnier, L. and Charneau, P. (2000) HIV-1 genome nuclear import is mediated by a central DNA flap. Cell 101, 173-185 (Pubitemid 32004745)
46. Dvorin, J. D., Bell, P., Maul, G. G., Yamashita, M., Emerman, M. and Malim, M. H. (2002) Reassessment of the roles of integrase and the central DNA flap in human immunodeficiency virus type 1 nuclear import. J. Virol. 76, 12087-12096 (Pubitemid 35304308)
47. Zennou, V., Serguera, C., Sarkis, C., Colin, P., Perret, E., Mallet, J. and Charneau, P. (2001) The HIV-1 DNA flap stimulates HIV vector-mediated cell transduction in the brain. Nat. Biotechnol. 19, 446-450 (Pubitemid 32428287)
48. Van Maele, B., De Rijck, J., De Clercq, E. and Debyser, Z. (2003) Impact of the central polypurine tract on the kinetics of human immunodeficiency virus type 1 vector transduction. J. Virol. 77, 4685-4694 (Pubitemid 36402818)
49. Demaison, C., Parsley, K., Brouns, G., Scherr, M., Battmer, K., Kinnon, C., Grez, M. and Thrasher, A. J. (2002) High-level transduction and gene expression in hematopoietic repopulating cells using a human imunodeficiency virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum. Gene Ther. 13, 803-813 (Pubitemid 34493974)
50. Park, F. and Kay, M. A. (2001) Modified HIV-1 based lentiviral vectors have an effect on viral transduction efficiency and gene expression in vitro and in vivo. Mol. Ther. 4, 164-173 (Pubitemid 32851279)
51. Donello, J. E., Loeb, J. E. and Hope, T. J. (1998) Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J. Virol. 72, 5085-5092 (Pubitemid 28215689)
52. Zufferey, R., Donello, J. E., Trono, D. and Hope, T. J. (1999) Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886-2892 (Pubitemid 29135675)
53. Brun, S., Faucon-Biguet, N. and Mallet, J. (2003) Optimization of transgene expression at the posttranscriptional level in neural cells: implications for gene therapy. Mol. Ther. 7, 782-789 (Pubitemid 36790255)
54. Ramezani, A., Hawley, T. S. and Hawley, R. G. (2000) Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther. 2, 458-469
55. Kingsman, S. M., Mitrophanous, K. and Olsen, J. C. (2005) Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther. 12, 3-4 (Pubitemid 40090038)
56. Zanta-Boussif, M. A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A. J., Hope, T. J. and Galy, A. (2009) Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS. Gene Ther. 16, 605-619
57. Otto, E., Jones-Trower, A., Vanin, E. F., Stambaugh, K., Mueller, S. N., Anderson, W. F. and McGarrity, G. J. (1994) Characterization of a replication-competent retrovirus resulting from recombination of packaging and vector sequences. Hum. Gene Ther. 5, 567-575 (Pubitemid 24310547)
58. Wu, X., Wakefield, J. K., Liu, H., Xiao, H., Kralovics, R., Prchal, J. T. and Kappes, J. C. (2000) Development of a novel trans-lentiviral vector that affords predictable safety. Mol. Ther. 2, 47-55
59. Sastry, L., Xu, Y., Johnson, T., Desai, K., Rissing, D., Marsh, J. and Cornetta, K. (2003) Certification assays for HIV-1-based vectors: frequent passage of gag sequences without evidence of replication-competent viruses. Mol. Ther. 8, 830-839 (Pubitemid 37527459)
60. Ikeda, Y., Takeuchi, Y., Martin, F., Cosset, F. L., Mitrophanous, K. and Collins, M. (2003) Continuous high-titer HIV-1 vector production. Nat. Biotechnol. 21, 569-572 (Pubitemid 36532025)
61. Segall, H. I., Yoo, E. and Sutton, R. E. (2003) Characterization and detection of artificial replication-competent lentivirus of altered host range. Mol. Ther. 8, 118-129 (Pubitemid 36939303)
62. Schwartz, S., Felber, B. K. and Pavlakis, G. N. (1992) Distinct RNA sequences in the gag region of human immunodeficiency virus type 1 decrease RNA stability and inhibit expression in the absence of Rev protein. J. Virol. 66, 150-159
63. Kotsopoulou, E., Kim, V. N., Kingsman, A. J., Kingsman, S. M. and Mitrophanous, K. A. (2000) A Rev-independent human immunodeficiency virus type 1 (HIV-1)-based vector that exploits a codon-optimized HIV-1 gag-pol gene. J. Virol. 74, 4839-4852 (Pubitemid 30237912)
64. Wagner, R., Graf, M., Bieler, K., Wolf, H., Grunwald, T., Foley, P. and Uberla, K. (2000) Rev-independent expression of synthetic gag-pol genes of human immunodeficiency virus type 1 and simian immunodeficiency virus: implications for the safety of lentiviral vectors. Hum. Gene Ther. 11, 2403-2413
65. Gasmi, M., Glynn, J., Jin, M. J., Jolly, D. J., Yee, J. K. and Chen, S. T. (1999) Requirements for efficient production and transduction of human immunodeficiency virus type 1-based vectors. J. Virol. 73, 1828-1834 (Pubitemid 29098082)
66. Mautino, M. R., Keiser, N. and Morgan, R. A. (2000) Improved titers of HIV-based lentiviral vectors using the SRV-1 constitutive transport element. Gene Ther. 7, 1421-1424 (Pubitemid 30662249)
67. Anson, D. S. and Fuller, M. (2003) Rational development of a HIV-1 gene therapy vector. J. Gene Med. 5, 829-838 (Pubitemid 40310407)
68. Cockrell, A. S., Ma, H., Fu, K., McCown, T. J. and Kafri, T. (2006) A trans-lentiviral packaging cell line for high-titer conditional self-inactivating HIV-1 vectors. Mol. Ther. 14, 276-284 (Pubitemid 43964495)
69. White, S. M., Renda, M., Nam, N. Y., Klimatcheva, E., Zhu, Y., Fisk, J., Halterman, M., Rimel, B. J., Federoff, H., Pandya, S. et al. (1999) Lentivirus vectors using human and simian immunodeficiency virus elements. J. Virol. 73, 2832-2840 (Pubitemid 29135670)
70. Poeschla, E., Gilbert, J., Li, X., Huang, S., Ho, A. and Wong-Staal, F. (1998) Identification of a human immunodeficiency virus type 2 (HIV-2) encapsidation determinant and transduction of nondividing human cells by HIV-2-based lentivirus vectors. J. Virol. 72, 6527-6536 (Pubitemid 28323967)
71. Negre, D., Mangeot, P. E., Duisit, G., Blanchard, S., Vidalain, P. O., Leissner, P., Winter, A. J., Rabourdin-Combe, C., Mehtali, M., Moullier, P. et al. (2000) Characterization of novel safe lentiviral vectors derived from simian immunodeficiency virus (SIVmac251) that efficiently transduce mature human dendritic cells. Gene Ther. 7, 1613-1623
72. Hatziioannou, T., Cowan, S. and Bieniasz, P. D. (2004) Capsid-dependent and -independent postentry restriction of primate lentivirus tropism in rodent cells. J. Virol. 78, 1006-1011 (Pubitemid 38067622)
73. Hanawa, H., Hematti, P., Keyvanfar, K., Metzger, M. E., Krouse, A., Donahue, R. E., Kepes, S., Gray, J., Dunbar, C. E., Persons, D. A. and Nienhuis, A. W. (2004) Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system. Blood 103, 4062-4069 (Pubitemid 38685345)
74. Naumann, N., De Ravin, S. S., Choi, U., Moayeri, M., Whiting-Theobald, N., Linton, G. F., Ikeda, Y. and Malech, H. L. (2007) Simian immunodeficiency virus lentivector corrects human X-linked chronic granulomatous disease in the NOD/SCID mouse xenograft. Gene Ther. 14, 1513-1524 (Pubitemid 47570710)
75. Shimojima, M., Miyazawa, T., Ikeda, Y., McMonagle, E. L., Haining, H., Akashi, H., Takeuchi, Y., Hosie, M. J. and Willett, B. J. (2004) Use of CD134 as a primary receptor by the feline immunodeficiency virus. Science 303, 1192-1195 (Pubitemid 38233584)
76. Ikeda, Y., Tomonaga, K., Kawaguchi, Y., Kohmoto, M., Inoshima, Y., Tohya, Y., Miyazawa, T., Kai, C. and Mikami, T. (1996) Feline immunodeficiency virus can infect a human cell line (MOLT-4) but establishes a state of latency in the cells. J. Gen. Virol. 77, 1623-1630 (Pubitemid 26278739)
77. Poeschla, E. M., Wong-Staal, F. and Looney, D. J. (1998) Efficient transduction of nondividing human cells by feline immunodeficiency virus lentiviral vectors. Nat. Med. 4, 354-357 (Pubitemid 28144098)
78. Mitrophanous, K., Yoon, S., Rohll, J., Patil, D., Wilkes, F., Kim, V., Kingsman, S., Kingsman, A. and Mazarakis, N. (1999) Stable gene transfer to the nervous system using a non-primate lentiviral vector. Gene Ther. 6, 1808-1818 (Pubitemid 29531276)
79. Olsen, J. C. (1998) Gene transfer vectors derived from equine infectious anemia virus. Gene Ther. 5, 1481-1487
80. Matukonis, M., Li, M., Molina, R. P., Paszkiet, B., Kaleko, M. and Luo, T. (2002) Development of second- and third-generation bovine immunodeficiency virus-based gene transfer systems. Hum. Gene Ther. 13, 1293-1303 (Pubitemid 35034509)
81. Curran, M. A., Kaiser, S. M., Achacoso, P. L. and Nolan, G. P. (2000) Efficient transduction of nondividing cells by optimized feline immunodeficiency virus vectors. Mol. Ther. 1, 31-38
82. Ikeda, Y., Collins, M. K., Radcliffe, P. A., Mitrophanous, K. A. and Takeuchi, Y. (2002) Gene transduction efficiency in cells of different species by HIV and EIAV vectors. Gene Ther. 9, 932-938 (Pubitemid 34812283)
83. Saenz, D. T., Teo, W., Olsen, J. C. and Poeschla, E. M. (2005) Restriction of feline immunodeficiency virus by Ref1, Lv1, and primate TRIM5α proteins. J. Virol. 79, 15175-15188 (Pubitemid 43032300)
84. Dropulic, B. (2001) Lentivirus in the clinic. Mol. Ther. 4, 511-512
85. Themis, M., Waddington, S. N., Schmidt, M., von Kalle, C., Wang, Y., Al-Allaf, F., Gregory, L. G., Nivsarkar, M., Themis, M., Holder, M. V. et al. (2005) Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol. Ther. 12, 763-771 (Pubitemid 41350158)
86. Labrador, M. and Corces, V. G. (2002) Setting the boundaries of chromatin domains and nuclear organization. Cell 111, 151-154 (Pubitemid 35292434)
87. Arumugam, P. I., Scholes, J., Perelman, N., Xia, P., Yee, J. K. and Malik, P. (2007) Improved human β-globin expression from self-inactivating lentiviral vectors carrying the chicken hypersensitive site-4 (cHS4) insulator element. Mol. Ther. 15, 1863-1871 (Pubitemid 47434195)
88. Aker, M., Tubb, J., Groth, A. C., Bukovsky, A. A., Bell, A. C., Felsenfeld, G., Kiem, H. P., Stamatoyannopoulos, G. and Emery, D. W. (2007) Extended core sequences from the cHS4 insulator are necessary for protecting retroviral vectors from silencing position effects. Hum. Gene Ther. 18, 333-343 (Pubitemid 46709345)
89. Uchida, N., Washington, K. N., Lap, C. J., Hsieh, M. M. and Tisdale, J. F. (2011) Chicken HS4 insulators have minimal barrier function among progeny of human hematopoietic cells transduced with an HIV1-based lentiviral vector. Mol. Ther. 19, 133-139
90. Zhang, F., Thornhill, S. I., Howe, S. J., Ulaganathan, M., Schambach, A., Sinclair, J., Kinnon, C., Gaspar, H. B., Antoniou, M. and Thrasher, A. J. (2007) Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 110, 1448-1457 (Pubitemid 47443959)
91. Zhang, F., Frost, A. R., Blundell, M. P., Bales, O., Antoniou, M. N. and Thrasher, A. J. (2010) A ubiquitous chromatin opening element (UCOE) confers resistance to DNA methylation-mediated silencing of lentiviral vectors. Mol. Ther. 18, 1640-1649
92. Kootstra, N. A., Munk, C., Tonnu, N., Landau, N. R. and Verma, I. M. (2003) Abrogation of postentry restriction of HIV-1-based lentiviral vector transduction in simian cells. Proc. Natl. Acad. Sci. U.S.A. 100, 1298-1303 (Pubitemid 36183996)
93. Ikeda, Y., Ylinen, L. M., Kahar-Bador, M. and Towers, G. J. (2004) Influence of gag on human immunodeficiency virus type 1 species-specific tropism. J. Virol. 78, 11816-11822 (Pubitemid 39390768)
94. Neil, S., Martin, F., Ikeda, Y. and Collins, M. (2001) Postentry restriction to human immunodeficiency virus-based vector transduction in human monocytes. J. Virol. 75, 5448-5456 (Pubitemid 32488137)
95. Mangeot, P. E., Duperrier, K., Negre, D., Boson, B., Rigal, D., Cosset, F. L. and Darlix, J. L. (2002) High levels of transduction of human dendritic cells with optimized SIV vectors. Mol. Ther. 5, 283-290 (Pubitemid 34211224)
96. Muhlebach, M. D., Wolfrum, N., Schule, S., Tschulena, U., Sanzenbacher, R., Flory, E., Cichutek, K. and Schweizer, M. (2005) Stable transduction of primary human monocytes by simian lentiviral vector PBj. Mol. Ther. 12, 1206-1216 (Pubitemid 41723055)
97. Laguette, N., Sobhian, B., Casartelli, N., Ringeard, M., Chable-Bessia, C., Segeral, E., Yatim, A., Emiliani, S., Schwartz, O. and Benkirane, M. (2011) SAMHD1 is the dendritic-and myeloid-cell-specific HIV-1 restriction factor counteracted by Vpx. Nature 474, 654-657
98. Berger, G., Durand, S., Goujon, C., Nguyen, X. N., Cordeil, S., Darlix, J. L. and Cimarelli, A. (2011) A simple, versatile and efficient method to genetically modify human monocyte-derived dendritic cells with HIV-1-derived lentiviral vectors. Nat. Protoc. 6, 806-816
99. Stitz, J., Buchholz, C. J., Engelstadter, M., Uckert, W., Bloemer, U., Schmitt, I. and Cichutek, K. (2000) Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1. Virology 273, 16-20 (Pubitemid 30602003)
100. Strang, B. L., Ikeda, Y., Cosset, F. L., Collins, M. K. and Takeuchi, Y. (2004) Characterization of HIV-1 vectors with gammaretrovirus envelope glycoproteins produced from stable packaging cells. Gene Ther. 11, 591-598 (Pubitemid 38455977)
101. Rasko, J. E., Battini, J. L., Gottschalk, R. J., Mazo, I. and Miller, A. D. (1999) The RD114/simian type D retrovirus receptor is a neutral amino acid transporter. Proc. Natl. Acad. Sci. U.S.A. 96, 2129-2134 (Pubitemid 29117879)
102. Sakuma, T., De Ravin, S. S., Tonne, J. M., Thatava, T., Ohmine, S., Takeuchi, Y., Malech, H. L. and Ikeda, Y. (2010) Characterization of retroviral and lentiviral vectors pseudotyped with xenotropic murine leukemia virus-related virus envelope glycoprotein. Hum. Gene Ther. 21, 1665-1673
103. Kahl, C. A., Marsh, J., Fyffe, J., Sanders, D. A. and Cornetta, K. (2004) Human immunodeficiency virus type 1-derived lentivirus vectors pseudotyped with envelope glycoproteins derived from Ross River virus and Semliki Forest virus. J. Virol. 78, 1421-1430 (Pubitemid 38095852)
104. Strang, B. L., Takeuchi, Y., Relander, T., Richter, J., Bailey, R., Sanders, D. A., Collins, M. K. and Ikeda, Y. (2005) Human immunodeficiency virus type 1 vectors with alphavirus envelope glycoproteins produced from stable packaging cells. J. Virol. 79, 1765-1771 (Pubitemid 40459122)
105. Beyer, W. R., Westphal, M., Ostertag, W. and von Laer, D. (2002) Oncoretrovirus and lentivirus vectors pseudotyped with lymphocytic choriomeningitis virus glycoprotein: generation, concentration, and broad host range. J. Virol. 76, 1488-1495 (Pubitemid 34066448)
106. Watson, D. J., Kobinger, G. P., Passini, M. A., Wilson, J. M. and Wolfe, J. H. (2002) Targeted transduction patterns in the mouse brain by lentivirus vectors pseudotyped with VSV, Ebola, Mokola, LCMV, or MuLV envelope proteins. Mol. Ther. 5, 528-537 (Pubitemid 34546082)
107. 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 (Pubitemid 28471831)
108. Hanawa, H., Kelly, P. F., Nathwani, A. C., Persons, D. A., Vandergriff, J. A., Hargrove, P., Vanin, E. F. and Nienhuis, A. W. (2002) Comparison of various envelope proteins for their ability to pseudotype lentiviral vectors and transduce primitive hematopoietic cells from human blood. Mol. Ther. 5, 242-251 (Pubitemid 34211219)
109. + cells using amphotropic-, GALV-, or RD114-pseudotyped HIV-1-based vectors from stable producer cells. Mol. Ther. 11, 452-459
110. Sinn, P. L., Hickey, M. A., Staber, P. D., Dylla, D. E., Jeffers, S. A., Davidson, B. L., Sanders, D. A. and McCray, Jr, P. B. (2003) Lentivirus vectors pseudotyped with filoviral envelope glycoproteins transduce airway epithelia from the apical surface independently of folate receptor α. J. Virol. 77, 5902-5910 (Pubitemid 36538982)
111. Yang, L., Bailey, L., Baltimore, D. and Wang, P. (2006) Targeting lentiviral vectors to specific cell types in vivo. Proc. Natl. Acad. Sci. U.S.A. 103, 11479-11484 (Pubitemid 44182477)
112. Maurice, M., Verhoeyen, E., Salmon, P., Trono, D., Russell, S. J. and Cosset, F. L. (2002) Efficient gene transfer into human primary blood lymphocytes by surface-engineered lentiviral vectors that display a T cell-activating polypeptide. Blood 99, 2342-2350 (Pubitemid 34525419)
113. Morizono, K., Xie, Y., Ringpis, G. E., Johnson, M., Nassanian, H., Lee, B., Wu, L. and Chen, I. S. (2005) Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat. Med. 11, 346-352 (Pubitemid 40460565)
114. Funke, S., Maisner, A., Muhlebach, M. D., Koehl, U., Grez, M., Cattaneo, R., Cichutek, K. and Buchholz, C. J. (2008) Targeted cell entry of lentiviral vectors. Mol. Ther. 16, 1427-1436
115. Frecha, C., Costa, C., Negre, D., Gauthier, E., Russell, S. J., Cosset, F. L. and Verhoeyen, E. (2008) Stable transduction of quiescent T cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins. Blood 112, 4843-4852
116. Hioki, H., Kameda, H., Nakamura, H., Okunomiya, T., Ohira, K., Nakamura, K., Kuroda, M., Furuta, T. and Kaneko, T. (2007) Efficient gene transduction of neurons by lentivirus with enhanced neuron-specific promoters. Gene Ther. 14, 872-882 (Pubitemid 46780070)
117. Lopes, L., Dewannieux, M., Gileadi, U., Bailey, R., Ikeda, Y., Whittaker, C., Collin, M. P., Cerundolo, V., Tomihari, M., Ariizumi, K. and Collins, M. K. (2008) Immunization with a lentivector that targets tumor antigen expression to dendritic cells induces potent CD8+and CD4+ T-cell responses. J. Virol. 82, 86-95 (Pubitemid 350309126)
118. De Palma, M., Venneri, M. A., Roca, C. and Naldini, L. (2003) Targeting exogenous genes to tumor angiogenesis by transplantation of genetically modified hematopoietic stem cells. Nat. Med. 9, 789-795 (Pubitemid 36749231)
119. Schmeckpeper, J., Ikeda, Y., Kumar, A. H., Metharom, P., Russell, S. J. and Caplice, N. M. (2009) Lentiviral tracking of vascular differentiation in bone marrow progenitor cells. Differentiation 78, 169-176
120. Park, F., Ohashi, K. and Kay, M. A. (2003) The effect of age on hepatic gene transfer with self-inactivating lentiviral vectors in vivo. Mol. Ther. 8, 314-323 (Pubitemid 37062841)
121. Lai, E. C. (2002) Micro RNAs are complementary to 3′ UTR sequence motifs that mediate negative post-transcriptional regulation. Nat. Genet. 30, 363-364
122. Brown, B. D., Cantore, A., Annoni, A., Sergi, L. S., Lombardo, A., Della Valle, P., D'Angelo, A. and Naldini, L. (2007) A microRNA-regulated lentiviral vector mediates stable correction of hemophilia B mice. Blood 110, 4144-4152 (Pubitemid 351377775)
123. Gilham, D. E., Lie, A.L.M., Taylor, N. and Hawkins, R. E. (2010) Cytokine stimulation and the choice of promoter are critical factors for the efficient transduction of mouse T cells with HIV-1 vectors. J. Gene Med. 12, 129-136
124. Jones, S., Peng, P. D., Yang, S., Hsu, C., Cohen, C. J., Zhao, Y., Abad, J., Zheng, Z., Rosenberg, S. A. and Morgan, R. A. (2009) Lentiviral vector design for optimal T cell receptor gene expression in the transduction of peripheral blood lymphocytes and tumor-infiltrating lymphocytes. Hum. Gene Ther. 20, 630-640
125. Ni, Y., Sun, S., Oparaocha, I., Humeau, L., Davis, B., Cohen, R., Binder, G., Chang, Y. N., Slepushkin, V. and Dropulic, B. (2005) Generation of a packaging cell line for prolonged large-scale production of high-titer HIV-1-based lentiviral vector. J. Gene Med. 7, 818-834 (Pubitemid 40872867)
126. Farson, D., Witt, R., McGuinness, R., Dull, T., Kelly, M., Song, J., Radeke, R., Bukovsky, A., Consiglio, A. and Naldini, L. (2001) A new-generation stable inducible packaging cell line for lentiviral vectors. Hum. Gene Ther. 12, 981-997 (Pubitemid 32681861)
127. Pacchia, A. L., Adelson, M. E., Kaul, M., Ron, Y. and Dougherty, J. P. (2001) An inducible packaging cell system for safe, efficient lentiviral vector production in the absence of HIV-1 accessory proteins. Virology 282, 77-86 (Pubitemid 32881777)
128. Klages, N., Zufferey, R. and Trono, D. (2000) A stable system for the high-titer production of multiply attenuated lentiviral vectors. Mol. Ther. 2, 170-176
129. Kafri, T., van Praag, H., Ouyang, L., Gage, F. H. and Verma, I. M. (1999) A packaging cell line for lentivirus vectors. J. Virol. 73, 576-584 (Pubitemid 28565401)
130. Xu, K., Ma, H., McCown, T. J., Verma, I. M. and Kafri, T. (2001) Generation of a stable cell line producing high-titer self-inactivating lentiviral vectors. Mol. Ther. 3, 97-104 (Pubitemid 32509810)
131. Throm, R. E., Ouma, A. A., Zhou, S., Chandrasekaran, A., Lockey, T., Greene, M., De Ravin, S. S., Moayeri, M., Malech, H. L., Sorrentino, B. P. and Gray, J. T. (2009) Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood 113, 5104-5110
132. Reiser, J. (2000) Production and concentration of pseudotyped HIV-1-based gene transfer vectors. Gene Ther. 7, 910-913
133. VandenDriessche, T., Thorrez, L., Naldini, L., Follenzi, A., Moons, L., Berneman, Z., Collen, D. and Chuah, M. K. (2002) Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood 100, 813-822 (Pubitemid 34832606)
134. Pham, L., Ye, H., Cosset, F. L., Russell, S. J. and Peng, K. W. (2001) Concentration of viral vectors by co-precipitation with calcium phosphate. J. Gene Med. 3, 188-194 (Pubitemid 33685446)
135. Geraerts, M., Willems, S., Baekelandt, V., Debyser, Z. and Gijsbers, R. (2006) Comparison of lentiviral vector titration methods. BMC Biotechnol. 6, 34
136. Rowe, H. M., Lopes, L., Ikeda, Y., Bailey, R., Barde, I., Zenke, M., Chain, B. M. and Collins, M. K. (2006) Immunization with a lentiviral vector stimulates both CD4 and CD8 T cell responses to an ovalbumin transgene. Mol. Ther. 13, 310-319 (Pubitemid 43056896)
137. Pichlmair, A., Diebold, S. S., Gschmeissner, S., Takeuchi, Y., Ikeda, Y., Collins, M. K. and Reis e Sousa, C. (2007) Tubulovesicular structures within vesicular stomatitis virus G protein-pseudotyped lentiviral vector preparations carry DNA and stimulate antiviral responses via Toll-like receptor 9. J. Virol. 81, 539-547 (Pubitemid 46067881)
138. Naldini, L., Blomer, U., Gage, F. H., Trono, D. and Verma, I. M. (1996) 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 (Pubitemid 26347130)
139. Sandrin, V., Boson, B., Salmon, P., Gay, W., Negre, D., Le Grand, R., Trono, D. and Cosset, F. L. (2002) Lentiviral vectors pseudotyped with a modified RD114 envelope glycoprotein show increased stability in sera and augmented transduction of primary lymphocytes and CD34+ cells derived from human and nonhuman primates. Blood 100, 823-832 (Pubitemid 34832607)
140. Hacein-Bey-Abina, S., Von Kalle, C., Schmidt, M., McCormack, M. P., Wulffraat, N., Leboulch, P., Lim, A., Osborne, C. S., Pawliuk, R., Morillon, E. et al. (2003) LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419 (Pubitemid 37296260)
141. Cavazzana-Calvo, M., Hacein-Bey, S., de Saint Basile, G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J. L. et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288, 669-672 (Pubitemid 30241569)
142. Gaspar, H. B., Parsley, K. L., Howe, S., King, D., Gilmour, K. C., Sinclair, J., Brouns, G., Schmidt, M., Von Kalle, C., Barington, T. et al. (2004) Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 364, 2181-2187 (Pubitemid 40018447)
143. Schwarzwaelder, K., Howe, S. J., Schmidt, M., Brugman, M. H., Deichmann, A., Glimm, H., Schmidt, S., Prinz, C., Wissler, M., King, D. J. et al. (2007) Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo. J. Clin. Invest. 117, 2241-2249 (Pubitemid 47219569)
144. Chinen, J., Davis, J., De Ravin, S. S., Hay, B. N., Hsu, A. P., Linton, G. F., Naumann, N., Nomicos, E. Y., Silvin, C., Ulrick, J. et al. (2007) Gene therapy improves immune function in preadolescents with X-linked severe combined immunodeficiency. Blood 110, 67-73 (Pubitemid 47026820)
145. Hacein-Bey-Abina, S., Hauer, J., Lim, A., Picard, C., Wang, G. P., Berry, C. C., Martinache, C., Rieux-Laucat, F., Latour, S., Belohradsky, B. H. et al. (2010) Efficacy of gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 363, 355-364
146. Wu, X., Li, Y., Crise, B. and Burgess, S. M. (2003) Transcription start regions in the human genome are favored targets for MLV integration. Science 300, 1749-1751 (Pubitemid 36712571)
147. Mitchell, R. S., Beitzel, B. F., Schroder, A. R., Shinn, P., Chen, H., Berry, C. C., Ecker, J. R. and Bushman, F. D. (2004) Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol. 2, E234
148. Schroder, A. R., Shinn, P., Chen, H., Berry, C., Ecker, J. R. and Bushman, F. (2002) HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521-529 (Pubitemid 35232295)
149. Llano, M., Saenz, D. T., Meehan, A., Wongthida, P., Peretz, M., Walker, W. H., Teo, W. and Poeschla, E. M. (2006) An essential role for LEDGF/p75 in HIV integration. Science 314, 461-464 (Pubitemid 44628966)
150. Ciuffi, A., Llano, M., Poeschla, E., Hoffmann, C., Leipzig, J., Shinn, P., Ecker, J. R. and Bushman, F. (2005) A role for LEDGF/p75 in targeting HIV DNA integration. Nat. Med. 11, 1287-1289 (Pubitemid 41752906)
151. Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Ponzoni, M., Bartholomae, C., Sergi Sergi, L., Benedicenti, F., Ambrosi, A., Di Serio, C. et al. (2006) Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. 24, 687-696 (Pubitemid 43882120)
152. Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M. H., Schambach, A., Charrier, S., Galy, A., Thrasher, A. J. et al. (2009) Insertional transformation of hematopoietic cells by self-inactivating lentiviral and gammaretroviral vectors. Mol. Ther. 17, 1919-1928
153. Montini, E., Cesana, D., Schmidt, M., Sanvito, F., Bartholomae, C. C., Ranzani, M., Benedicenti, F., Sergi, L. S., Ambrosi, A., Ponzoni, M. et al. (2009) The genotoxic potential of retroviral vectors is strongly modulated by vector design and integration site selection in a mouse model of HSC gene therapy. J. Clin. Invest. 119, 964-975
154. Woods, N. B., Bottero, V., Schmidt, M., von Kalle, C. and Verma, I. M. (2006) Gene therapy: therapeutic gene causing lymphoma. Nature 440, 1123
155. Ginn, S. L., Liao, S. H., Dane, A. P., Hu, M., Hyman, J., Finnie, J. W., Zheng, M., Cavazzana-Calvo, M., Alexander, S. I., Thrasher, A. J. and Alexander, I. E. (2010) Lymphomagenesis in SCID-X1 mice following lentivirus-mediated phenotype correction independent of insertional mutagenesis and γ c overexpression. Mol. Ther. 18, 965-976
156. Saenz, D. T., Loewen, N., Peretz, M., Whitwam, T., Barraza, R., Howell, K. G., Holmes, J. M., Good, M. and Poeschla, E. M. (2004) Unintegrated lentivirus DNA persistence and accessibility to expression in nondividing cells: analysis with class I integrase mutants. J. Virol. 78, 2906-2920 (Pubitemid 38314348)
157. Leavitt, A. D., Robles, G., Alesandro, N. and Varmus, H. E. (1996) Human immunodeficiency virus type 1 integrase mutants retain in vitro integrase activity yet fail to integrate viral DNA efficiently during infection. J. Virol. 70, 721-728 (Pubitemid 26028986)
158. Nightingale, S. J., Hollis, R. P., Pepper, K. A., Petersen, D., Yu, X. J., Yang, C., Bahner, I. and Kohn, D. B. (2006) Transient gene expression by nonintegrating lentiviral vectors. Mol. Ther. 13, 1121-1132 (Pubitemid 43796157)
159. Apolonia, L., Waddington, S. N., Fernandes, C., Ward, N. J., Bouma, G., Blundell, M. P., Thrasher, A. J., Collins, M. K. and Philpott, N. J. (2007) Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol. Ther. 15, 1947-1954 (Pubitemid 350011734)
160. Philippe, S., Sarkis, C., Barkats, M., Mammeri, H., Ladroue, C., Petit, C., Mallet, J. and Serguera, C. (2006) 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, 17684-17689 (Pubitemid 44852220)
161. Yanez-Munoz, R. J., Balaggan, K. S., MacNeil, A., Howe, S. J., Schmidt, M., Smith, A. J., Buch, P., MacLaren, R. E., Anderson, P. N., Barker, S. E. et al. (2006) Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med. 12, 348-353 (Pubitemid 43355085)
162. Pfeifer, A., Brandon, E. P., Kootstra, N., Gage, F. H. and Verma, I. M. (2001) Delivery of the Cre recombinase by a self-deleting lentiviral vector: efficient gene targeting in vivo. Proc. Natl. Acad. Sci. U.S.A. 98, 11450-11455 (Pubitemid 32928749)
163. Ventura, A., Meissner, A., Dillon, C. P., McManus, M., Sharp, P. A., Van Parijs, L., Jaenisch, R. and Jacks, T. (2004) Cre-lox-regulated conditional RNA interference from transgenes. Proc. Natl. Acad. Sci. U.S.A. 101, 10380-10385 (Pubitemid 38924934)
164. Miller, J. C., Holmes, M. C., Wang, J., Guschin, D. Y., Lee, Y. L., Rupniewski, I., Beausejour, C. M., Waite, A. J., Wang, N. S., Kim, K. A. et al. (2007) An improved zinc-finger nuclease architecture for highly specific genome editing. Nat. Biotechnol. 25, 778-785
165. Lombardo, A., Genovese, P., Beausejour, C. M., Colleoni, S., Lee, Y. L., Kim, K. A., Ando, D., Urnov, F. D., Galli, C., Gregory, P. D. et al. (2007) Gene editing in human stem cells using zinc finger nucleases and integrase-defective lentiviral vector delivery. Nat. Biotechnol. 25, 1298-1306
166. Malik, P., Arumugam, P. I., Yee, J. K. and Puthenveetil, G. (2005) Successful correction of the human Cooley's anemia β-thalassemia major phenotype using a lentiviral vector flanked by the chicken hypersensitive site 4 chromatin insulator. Ann. N.Y. Acad. Sci. 1054, 238-249 (Pubitemid 43031030)
167. Pawliuk, R., Westerman, K. A., Fabry, M. E., Payen, E., Tighe, R., Bouhassira, E. E., Acharya, S. A., Ellis, J., London, I. M., Eaves, C. J. et al. (2001) Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294, 2368-2371 (Pubitemid 33140563)
168. May, C., Rivella, S., Callegari, J., Heller, G., Gaensler, K. M., Luzzatto, L. and Sadelain, M. (2000) Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature 406, 82-86 (Pubitemid 30460215)
169. Adjali, O., Marodon, G., Steinberg, M., Mongellaz, C., Thomas-Vaslin, V., Jacquet, C., Taylor, N. and Klatzmann, D. (2005) In vivo correction of ZAP-70 immunodeficiency by intrathymic gene transfer. J. Clin. Invest. 115, 2287-2295 (Pubitemid 41134170)
170. Lo Bianco, C., Schneider, B. L., Bauer, M., Sajadi, A., Brice, A., Iwatsubo, T. and Aebischer, P. (2004) Lentiviral vector delivery of parkin prevents dopaminergic degeneration in an α-synuclein rat model of Parkinson's disease. Proc. Natl. Acad. Sci. U.S.A. 101, 17510-17515 (Pubitemid 39657434)
171. Wang, G., Slepushkin, V., Zabner, J., Keshavjee, S., Johnston, J. C., Sauter, S. L., Jolly, D. J., Dubensky, Jr. T.W., Davidson, B. L. and McCray, Jr, P. B. (1999) Feline immunodeficiency virus vectors persistently transduce nondividing airway epithelia and correct the cystic fibrosis defect. J. Clin. Invest. 104, R55-R62 (Pubitemid 30002376)
172. Azzouz, M., Le, T., Ralph, G. S., Walmsley, L., Monani, U. R., Lee, D. C., Wilkes, F., Mitrophanous, K. A., Kingsman, S. M., Burghes, A. H. and Mazarakis, N. D. (2004) Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy. J. Clin. Invest. 114, 1726-1731 (Pubitemid 40385528)
173. Yu, X., Zhan, X., D'Costa, J., Tanavde, V. M., Ye, Z., Peng, T., Malehorn, M. T., Yang, X., Civin, C. I. and Cheng, L. (2003) Lentiviral vectors with two independent internal promoters transfer high-level expression of multiple transgenes to human hematopoietic stem-progenitor cells. Mol. Ther. 7, 827-838 (Pubitemid 36790260)
174. Amendola, M., Venneri, M. A., Biffi, A., Vigna, E. and Naldini, L. (2005) Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat. Biotechnol. 23, 108-116 (Pubitemid 41724633)
175. Zhu, Y., Feuer, G., Day, S. L., Wrzesinski, S. and Planelles, V. (2001) Multigene lentiviral vectors based on differential splicing and translational control. Mol. Ther. 4, 375-382 (Pubitemid 33040524)
176. Licursi, M., Christian, S. L., Pongnopparat, T. and Hirasawa, K. (2011) In vitro and in vivo comparison of viral and cellular internal ribosome entry sites for bicistronic vector expression. Gene Ther. 18, 631-636
177. Szymczak, A. L., Workman, C. J., Wang, Y., Vignali, K. M., Dilioglou, S., Vanin, E. F. and Vignali, D. A. (2004) Correction of multi-gene deficiency in vivo using a single 'self-cleaving' 2A peptide-based retroviral vector. Nat. Biotechnol. 22, 589-594 (Pubitemid 38585911)
178. Yang, S., Cohen, C. J., Peng, P. D., Zhao, Y., Cassard, L., Yu, Z., Zheng, Z., Jones, S., Restifo, N. P., Rosenberg, S. A. and Morgan, R. A. (2008) Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition. Gene Ther. 15, 1411-1423
179. Ibrahimi, A., Vande Velde, G., Reumers, V., Toelen, J., Thiry, I., Vandeputte, C., Vets, S., Deroose, C., Bormans, G., Baekelandt, V. et al. (2009) Highly efficient multicistronic lentiviral vectors with peptide 2A sequences. Hum. Gene Ther. 20, 845-860
180. Fire, A., Xu, S., Montgomery, M. K., Kostas, S. A., Driver, S. E. and Mello, C. C. (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391, 806-811 (Pubitemid 28099681)
181. Hammond, S. M., Bernstein, E., Beach, D. and Hannon, G. J. (2000) An RNA-directed nuclease mediates post-transcriptional gene silencing in Drosophila cells. Nature 404, 293-296 (Pubitemid 30163547)
182. Paddison, P. J., Caudy, A. A., Bernstein, E., Hannon, G. J. and Conklin, D. S. (2002) Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev. 16, 948-958 (Pubitemid 34408544)
183. Stewart, S. A., Dykxhoorn, D. M., Palliser, D., Mizuno, H., Yu, E. Y., An, D. S., Sabatini, D. M., Chen, I. S., Hahn, W. C., Sharp, P. A. et al. (2003) Lentivirus-delivered stable gene silencing by RNAi in primary cells. RNA 9, 493-501 (Pubitemid 36356735)
184. Singer, O. and Verma, I. M. (2008) Applications of lentiviral vectors for shRNA delivery and transgenesis. Curr. Gene Ther. 8, 483-488
185. Li, M. J., Kim, J., Li, S., Zaia, J., Yee, J. K., Anderson, J., Akkina, R. and Rossi, J. J. (2005) 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. Mol. Ther. 12, 900-909 (Pubitemid 41614486)
186. Vandekerckhove, L., Christ, F., Van Maele, B., De Rijck, J., Gijsbers, R., Van den Haute, C., Witvrouw, M. and Debyser, Z. (2006) Transient and stable knockdown of the integrase cofactor LEDGF/p75 reveals its role in the replication cycle of human immunodeficiency virus. J. Virol. 80, 1886-1896 (Pubitemid 43228728)
187. Raoul, C., Abbas-Terki, T., Bensadoun, J. C., Guillot, S., Haase, G., Szulc, J., Henderson, C. E. and Aebischer, P. (2005) Lentiviral-mediated silencing of SOD1 through RNA interference retards disease onset and progression in a mouse model of ALS. Nat. Med. 11, 423-428 (Pubitemid 40562333)
188. Pfeifer, A., Eigenbrod, S., Al-Khadra, S., Hofmann, A., Mitteregger, G., Moser, M., Bertsch, U. and Kretzschmar, H. (2006) Lentivector-mediated RNAi efficiently suppresses prion protein and prolongs survival of scrapie-infected mice. J. Clin. Invest. 116, 3204-3210 (Pubitemid 44878634)
189. + cells after lentiviral vector-mediated gene transfer. Blood 117, 2817-2826
190. Bridge, A. J., Pebernard, S., Ducraux, A., Nicoulaz, A. L. and Iggo, R. (2003) Induction of an interferon response by RNAi vectors in mammalian cells. Nat. Genet. 34, 263-264 (Pubitemid 36792856)
191. Gentner, B., Schira, G., Giustacchini, A., Amendola, M., Brown, B. D., Ponzoni, M. and Naldini, L. (2009) Stable knockdown of microRNA in vivo by lentiviral vectors. Nat. Methods 6, 63-66
192. Gossen, M., Freundlieb, S., Bender, G., Muller, G., Hillen, W. and Bujard, H. (1995) Transcriptional activation by tetracyclines in mammalian cells. Science 268, 1766-1769
193. Kafri, T., van Praag, H., Gage, F. H. and Verma, I. M. (2000) Lentiviral vectors: regulated gene expression. Mol. Ther. 1, 516-521
194. Vigna, E., Cavalieri, S., Ailles, L., Geuna, M., Loew, R., Bujard, H. and Naldini, L. (2002) Robust and efficient regulation of transgene expression in vivo by improved tetracycline-dependent lentiviral vectors. Mol. Ther. 5, 252-261 (Pubitemid 34211220)
195. Wiznerowicz, M. and Trono, D. (2003) Conditional suppression of cellular genes: lentivirus vector-mediated drug-inducible RNA interference. J. Virol. 77, 8957-8961 (Pubitemid 36936105)
196. Perl, A. K., Zhang, L. and Whitsett, J. A. (2009) Conditional expression of genes in the respiratory epithelium in transgenic mice: cautionary notes and toward building a better mouse trap. Am. J. Respir. Cell Mol. Biol. 40, 1-3
197. Morimoto, M. and Kopan, R. (2009) rtTA toxicity limits the usefulness of the SP-C-rtTA transgenic mouse. Dev. Biol. 325, 171-178
198. Benabdellah, K., Cobo, M., Munoz, P., Toscano, M. G. and Martin, F. (2011) Development of an all-in-one lentiviral vector system based on the original TetR for the easy generation of Tet-ON cell lines. PLoS ONE 6, e23734
199. Pfeifer, A., Ikawa, M., Dayn, Y. and Verma, I. M. (2002) Transgenesis by lentiviral vectors: lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos. Proc. Natl. Acad. Sci. U.S.A. 99, 2140-2145 (Pubitemid 34169028)
200. McGrew, M. J., Sherman, A., Ellard, F. M., Lillico, S. G., Gilhooley, H. J., Kingsman, A. J., Mitrophanous, K. A. and Sang, H. (2004) Efficient production of germline transgenic chickens using lentiviral vectors. EMBO Rep. 5, 728-733 (Pubitemid 39173010)
201. van den Brandt, J., Wang, D., Kwon, S. H., Heinkelein, M. and Reichardt, H. M. (2004) Lentivirally generated eGFP-transgenic rats allow efficient cell tracking in vivo. Genesis 39, 94-99 (Pubitemid 38924656)
202. Wongsrikeao, P., Saenz, D., Rinkoski, T., Otoi, T. and Poeschla, E. (2011) Antiviral restriction factor transgenesis in the domestic cat. Nat. Methods 8, 853-859
203. Lois, C., Hong, E. J., Pease, S., Brown, E. J. and Baltimore, D. (2002) Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science 295, 868-872 (Pubitemid 34118370)
204. Hofmann, A., Kessler, B., Ewerling, S., Weppert, M., Vogg, B., Ludwig, H., Stojkovic, M., Boelhauve, M., Brem, G., Wolf, E. and Pfeifer, A. (2003) Efficient transgenesis in farm animals by lentiviral vectors. EMBO Rep. 4, 1054-1060 (Pubitemid 37527423)
205. Tiscornia, G., Singer, O., Ikawa, M. and Verma, I. M. (2003) A general method for gene knockdown in mice by using lentiviral vectors expressing small interfering RNA. Proc. Natl. Acad. Sci. U.S.A. 100, 1844-1848 (Pubitemid 36254536)
206. Herold, M. J., van den Brandt, J., Seibler, J. and Reichardt, H. M. (2008) Inducible and reversible gene silencing by stable integration of an shRNA-encoding lentivirus in transgenic rats. Proc. Natl. Acad. Sci. U.S.A. 105, 18507-18512
207. Palmowski, M. J., Lopes, L., Ikeda, Y., Salio, M., Cerundolo, V. and Collins, M. K. (2004) Intravenous injection of a lentiviral vector encoding NY-ESO-1 induces an effective CTL response. J. Immunol. 172, 1582-1587 (Pubitemid 38116804)
208. Esslinger, C., Chapatte, L., Finke, D., Miconnet, I., Guillaume, P., Levy, F. and MacDonald, H. R. (2003) In vivo administration of a lentiviral vaccine targets DCs and induces efficient CD8+ T cell responses. J. Clin. Invest. 111, 1673-1681 (Pubitemid 38057749)
209. Bobisse, S., Rondina, M., Merlo, A., Tisato, V., Mandruzzato, S., Amendola, M., Naldini, L., Willemsen, R. A., Debets, R., Zanovello, P. and Rosato, A. (2009) Reprogramming T lymphocytes for melanoma adoptive immunotherapy by T-cell receptor gene transfer with lentiviral vectors. Cancer Res. 69, 9385-9394
210. Iglesias, M. C., Mollier, K., Beignon, A. S., Souque, P., Adotevi, O., Lemonnier, F. and Charneau, P. (2007) Lentiviral vectors encoding HIV-1 polyepitopes induce broad CTL responses in vivo. Mol. Ther. 15, 1203-1210 (Pubitemid 46813625)
211. Dai, B., Yang, L., Yang, H., Hu, B., Baltimore, D. and Wang, P. (2009) HIV-1 Gag-specific immunity induced by a lentivector-based vaccine directed to dendritic cells. Proc. Natl. Acad. Sci. U.S.A. 106, 20382-20387
212. Lemiale, F., Asefa, B., Ye, D., Chen, C., Korokhov, N. and Humeau, L. (2010) An HIV-based lentiviral vector as HIV vaccine candidate: immunogenic characterization. Vaccine 28, 1952-1961
213. Hu, B., Yang, H., Dai, B., Tai, A. and Wang, P. (2009) Nonintegrating lentiviral vectors can effectively deliver ovalbumin antigen for induction of antitumor immunity. Hum. Gene Ther. 20, 1652-1664
214. Yang, H. G., Hu, B. L., Xiao, L. and Wang, P. (2011) Dendritic cell-directed lentivector vaccine induces antigen-specific immune responses against murine melanoma. Cancer Gene Ther. 18, 370-380
215. Takahashi, K., Tanabe, K., Ohnuki, M., Narita, M., Ichisaka, T., Tomoda, K. and Yamanaka, S. (2007) Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131, 861-872 (Pubitemid 350138099)
216. Yu, J., Vodyanik, M. A., Smuga-Otto, K., Antosiewicz-Bourget, J., Frane, J. L., Tian, S., Nie, J., Jonsdottir, G. A., Ruotti, V., Stewart, R. et al. (2007) Induced pluripotent stem cell lines derived from human somatic cells. Science 318, 1917-1920
217. Ohmine, S., Dietz, A. B., Deeds, M. C., Hartjes, K. A., Miller, D. R., Thatava, T., Sakuma, T., Kudva, Y. C. and Ikeda, Y. (2011) Induced pluripotent stem cells from GMP-grade hematopoietic progenitor cells and mononuclear myeloid cells. Stem Cell Res. Ther. 2, 46
218. Thatava, T., Armstrong, A. S., De Lamo, J. G., Edukulla, R., Khan, Y. K., Sakuma, T., Ohmine, S., Sundsbak, J. L., Harris, P. C., Kudva, Y. C. and Ikeda, Y. (2011) Successful disease-specific induced pluripotent stem cell generation from patients with kidney transplantation. Stem Cell Res. Ther. 2, 48
219. Papapetrou, E. P. and Sadelain, M. (2011) Generation of transgene-free human induced pluripotent stem cells with an excisable single polycistronic vector. Nat. Protoc. 6, 1251-1273
220. Carey, B. W., Markoulaki, S., Hanna, J., Saha, K., Gao, Q., Mitalipova, M. and Jaenisch, R. (2009) Reprogramming of murine and human somatic cells using a single polycistronic vector. Proc. Natl. Acad. Sci. U.S.A. 106, 157-162
221. Subramanyam, D., Lamouille, S., Judson, R. L., Liu, J. Y., Bucay, N., Derynck, R. and Blelloch, R. (2011) Multiple targets of miR-302 and miR-372 promote reprogramming of human fibroblasts to induced pluripotent stem cells. Nat. Biotechnol. 29, 443-448
222. Huang, P., He, Z., Ji, S., Sun, H., Xiang, D., Liu, C., Hu, Y., Wang, X. and Hui, L. (2011) Induction of functional hepatocyte-like cells from mouse fibroblasts by defined factors. Nature 475, 386-389
223. Ieda, M., Fu, J. D., Delgado-Olguin, P., Vedantham, V., Hayashi, Y., Bruneau, B. G. and Srivastava, D. (2010) Direct reprogramming of fibroblasts into functional cardiomyocytes by defined factors. Cell 142, 375-386
224. Pfeifer, A., Kessler, T., Yang, M., Baranov, E., Kootstra, N., Cheresh, D. A., Hoffman, R. M. and Verma, I. M. (2001) Transduction of liver cells by lentiviral vectors: analysis in living animals by fluorescence imaging. Mol. Ther. 3, 319-322 (Pubitemid 32514647)
225. Iyer, M., Salazar, F. B., Lewis, X., Zhang, L., Carey, M., Wu, L. and Gambhir, S. S. (2004) Noninvasive imaging of enhanced prostate-specific gene expression using a two-step transcriptional amplification-based lentivirus vector. Mol. Ther. 10, 545-552 (Pubitemid 39186115)
226. Deroose, C. M., Reumers, V., Gijsbers, R., Bormans, G., Debyser, Z., Mortelmans, L. and Baekelandt, V. (2006) Noninvasive monitoring of long-term lentiviral vector-mediated gene expression in rodent brain with bioluminescence imaging. Mol. Ther. 14, 423-431 (Pubitemid 44184969)
227. Ray, P., De, A., Min, J. J., Tsien, R. Y. and Gambhir, S. S. (2004) Imaging tri-fusion multimodality reporter gene expression in living subjects. Cancer Res. 64, 1323-1330 (Pubitemid 38235600)
228. Chen, W., Wu, X., Levasseur, D. N., Liu, H., Lai, L., Kappes, J. C. and Townes, T. M. (2000) Lentiviral vector transduction of hematopoietic stem cells that mediate long-term reconstitution of lethally irradiated mice. Stem Cells 18, 352-359
229. Gallo, P., Grimaldi, S., Latronico, M. V., Bonci, D., Pagliuca, A., Gallo, P., Ausoni, S., Peschle, C. and Condorelli, G. (2008) A lentiviral vector with a short troponin-I promoter for tracking cardiomyocyte differentiation of human embryonic stem cells. Gene Ther. 15, 161-170 (Pubitemid 351150040)
230. Luker, K. E., Mihalko, L. A., Schmidt, B. T., Lewin, S. A., Ray, P., Shcherbo, D., Chudakov, D. M. and Luker, G. D. (2011) In vivo imaging of ligand receptor binding with Gaussia luciferase complementation. Nat. Med. 18, 172-177
231. Manilla, P., Rebello, T., Afable, C., Lu, X., Slepushkin, V., Humeau, L. M., Schonely, K., Ni, Y., Binder, G. K., Levine, B. L. et al. (2005) Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector. Hum. Gene Ther. 16, 17-25 (Pubitemid 40250328)
232. Lu, X., Humeau, L., Slepushkin, V., Binder, G., Yu, Q., Slepushkina, T., Chen, Z., Merling, R., Davis, B., Chang, Y. N. and Dropulic, B. (2004) Safe two-plasmid production for the first clinical lentivirus vector that achieves >99% transduction in primary cells using a one-step protocol. J. Gene Med. 6, 963-973 (Pubitemid 40228111)
233. Levine, B. L., Humeau, L. M., Boyer, J., MacGregor, R. R., Rebello, T., Lu, X., Binder, G. K., Slepushkin, V., Lemiale, F., Mascola, J. R. et al. (2006) Gene transfer in humans using a conditionally replicating lentiviral vector. Proc. Natl. Acad. Sci. U.S.A. 103, 17372-17377 (Pubitemid 44788976)
234. Cartier, N., Hacein-Bey-Abina, S., Bartholomae, C. C., Veres, G., Schmidt, M., Kutschera, I., Vidaud, M., Abel, U., Dal-Cortivo, L., Caccavelli, L. et al. (2009) Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326, 818-823
235. Bank, A., Dorazio, R. and Leboulch, P. (2005) A phase I/II clinical trial of β-globin gene therapy for β-thalassemia. Ann. N.Y. Acad. Sci. 1054, 308-316 (Pubitemid 43031038)
236. Cavazzana-Calvo, M., Payen, E., Negre, O., Wang, G., Hehir, K., Fusil, F., Down, J., Denaro, M., Brady, T., Westerman, K. et al. (2010) Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia. Nature 467, 318-322