Lentiviral vectors in clinical trials: Current status

Current Opinion in Molecular Therapeutics

Volumn 11, Issue 5, 2009, Pages 554-564

  D'Costa, J ^a   Mansfield, S G ^a   Humeau, L M ^a  

^a VIRxSYS Corporation   (United States)

Author keywords

Adoptive immunotherapy; Clinical trial; Gene therapy; Lentiviral vector; Safety

Indexed keywords

AROMATIC LEVO AMINO ACID DECARBOXYLASE; B7 ANTIGEN; BETA GLOBIN; BETA GLUCURONIDASE; COMPLEMENTARY RNA; FANCONI ANEMIA GROUP A PROTEIN; GUANOSINE TRIPHOSPHATE CYCLOHYDROLASE I; INTERLEUKIN 2; LENTIGLOBIN; LENTIVIRUS VECTOR; LEVODOPA; LEXGENLEUCEL T; LG 631; MELAN A; METHYLATED DNA PROTEIN CYSTEINE METHYLTRANSFERASE; PROSAVIN; SMALL INTERFERING RNA; TEMOZOLOMIDE; THALAGEN; TYROSINE 3 MONOOXYGENASE; UNCLASSIFIED DRUG; VRX 1023; WISKOTT ALDRICH SYNDROME PROTEIN;

ACUTE GRANULOCYTIC LEUKEMIA; ADRENOLEUKODYSTROPHY; BETA THALASSEMIA; CANCER IMMUNOTHERAPY; CARCINOGENESIS; CLINICAL TRIAL; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; EX VIVO GENE TRANSFER; FANCONI ANEMIA; GENE EXPRESSION; GENETIC DISORDER; GLIOBLASTOMA; GLIOMA; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS INFECTION; INFECTION; MELANOMA; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHODGKIN LYMPHOMA; NONHUMAN; PARKINSON DISEASE; REVIEW; SICKLE CELL ANEMIA; THALASSEMIA MAJOR; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; WISKOTT ALDRICH SYNDROME; X LINKED SEVERE COMBINED IMMUNODEFICIENCY;

ANIMALS; CLINICAL TRIALS AS TOPIC; FANCONI ANEMIA; GENE THERAPY; GENETIC VECTORS; GLIOMA; HUMANS; LENTIVIRUS; LYMPHOMA, NON-HODGKIN;

EID: 70349909993     PISSN: 14648431     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Review

References (105)

1. Page KA, Landau NR, Littman DR: Construction and use of a human immunodeficiency virus vector for analysis of virus infectivity. J Virol (1990) 64(11):5270-5276. (Pubitemid 20364514)
2. Blömer U, Naldini L, Verma IM, Trono D, Gage FH: Applications of gene therapy to the CNS. Hum Mol Genet (1996) 5:1397-1404.
3. Naldini L, Blomer U, Gallay P, Ory D, Mulligan R, Gage FH, Verma IM, Trono D: In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science (1996) 272(5259):263-267. (Pubitemid 26119138)
4. Verhoeyen E, Cosset FL: Surface-engineering of lentiviral vectors. J Gene Med (2004) 6(Suppl 1):S83-S94.
5. Frecha C, Szecsi J, Cosset FL, Verhoeyen E: Strategies for targeting lentiviral vectors. Curr Gene Ther (2008) 8(6):449-460.
6. Gene therapy clinical trials worldwide: John Wiley & Sons Inc, Hoboken, NJ, USA (2009). www.wiley.co.uk/genmed/clinical • A database of worldwide gene therapy trials.
7. Human gene transfer protocols: NIH, Bethesda, MD, USA (2009). oba.od.nih.gov/oba/rac/PROTOCOL.pdf
8. Office of Biotechnology activities: NIH, Bethesda, MD, USA (2009). oba.od.nih.gov/oba/index.html
9. Levine BL, Humeau LM, Boyer J, MacGregor RR, Rebello T, Lu X, Binder GK, Slepushkin V, Lemiale F, Mascola JR, Bushman FD et al: Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA (2006) 103(46):17372-17377. •• Published safety data of the first phase I clinical trial of LV gene therapy. (Pubitemid 44788976)
10. Annoni A, Battaglia M, Follenzi A, Lombardo A, Sergi-Sergi L, Naldini L, Roncarolo MG: The immune response to lentiviral-delivered transgene is modulated in vivo by transgene-expressing antigen-presenting cells but not by CD4+CD25+ regulatory T cells. Blood (2007) 110(6):1788-1796. (Pubitemid 47443889)
11. Kang Y, Xie L, Tran DT, Stein CS, Hickey M, Davidson BL, McCray PB Jr: Persistent expression of Factor VIII in vivo following nonprimate lentiviral gene transfer. Blood (2005) 106(5):1552-1558. (Pubitemid 41208565)
12. Morizono K, Xie Y, Ringpis GE, Johnson M, Nassanian H, Lee B, Wu L, Chen IS: Lentiviral vector retargeting to P-glycoprotein on metastatic melanoma through intravenous injection. Nat Med (2005) 11(3):346-352. (Pubitemid 40460565)
13. Guibinga GH, Friedmann T: Baculovirus GP64-pseudotyped HIV-based lentivirus vectors are stabilized against complement inactivation by codisplay of decay accelerating factor (DAF) or of a GP64-DAF fusion protein. Mol Ther (2005) 11(4):645-651.
14. Yang L, Yang H, Rideout K, Cho T, Joo KI, Ziegler L, Elliot A, Walls A, Yu D, Baltimore D, Wang P: Engineered lentivector targeting of dendritic cells for in vivo immunization. Nat Biotechnol (2008) 26(3):326-334. (Pubitemid 351355129)
15. Kimura T, Koya RC, Anselmi L, Sternini C, Wang HJ, Comin-Anduix B, Prins RM, Faure-Kumar E, Rozengurt N, Cui Y, Kasahara N et al: Lentiviral vectors with CMV or MHCII promoters administered in vivo: Immune reactivity versus persistence of expression. Mol Ther (2007) 15(7):1390-1399. (Pubitemid 46965335)
16. Di Nunzio F, Maruggi G, Ferrari S, Di Iorio E, Poletti V, Garcia M, Del Rio M, De Luca M, Larcher F, Pellegrini G, Mavilio F: Correction of laminin-5 deficiency in human epidermal stem cells by transcriptionally targeted lentiviral vectors. Mol Ther (2008) 16(12):1977-1985.
17. Brown BD, Venneri MA, Zingale A, Sergi Sergi L, Naldini L: Endogenous microRNA regulation suppresses transgene expression in hematopoietic lineages and enables stable gene transfer. Nat Med (2006) 12(5):585-591.
18. Brown BD, Sitia G, Annoni A, Hauben E, Sergi LS, Zingale A, Roncarolo MG, Guidotti LG, Naldini L: In vivo administration of lentiviral vectors triggers a type I interferon response that restricts hepatocyte gene transfer and promotes vector clearance. Blood (2007) 109(7):2797-2805. (Pubitemid 46482074)
19. Di Domenico C, Di Napoli D, Gonzalez YRE, Lombardo A, Naldini L, Di Natale P: Limited transgene immune response and long-term expression of human α-L-iduronidase in young adult mice with mucopolysaccharidosis type I by liver-directed gene therapy. Hum Gene Ther (2006) 17(11):1112-1121. (Pubitemid 44749712)
20. Pariente N, Morizono K, Virk MS, Petrigliano FA, Reiter RE, Lieberman JR, Chen IS: A novel dual-targeted lentiviral vector leads to specific transduction of prostate cancer bone metastases in vivo after systemic administration. Mol Ther (2007) 15(11):1973-1981. (Pubitemid 350011737)
21. Lemiale F, Korokhov N: Lentiviral vectors for HIV disease prevention and treatment. Vaccine (2009) 27(25-26):3443-3449. • Review on the development of LV-based vaccines for the prevention and treatment of HIV infection.
22. + T cell immunity in recombinant lentivector-mediated genetic immunization. Immunity (2006) 24(5):643-656.
23. Muul LM, Tuschong LM, Soenen SL, Jagadeesh GJ, Ramsey WJ, Long Z, Carter CS, Garabedian EK, Alleyne M, Brown M, Bernstein W et al: Persistence and expression of the adenosine deaminase gene for 12 years and immune reaction to gene transfer components: Long-term results of the first clinical gene therapy trial. Blood (2003) 101(7):2563-2569. (Pubitemid 36857614)
24. Humeau LM, Binder GK, Lu X, Slepushkin V, Merling R, Echeagaray P, Pereira M, Slepushkina T, Barnett S, Dropulic LK, Carroll R et al: Efficient lentiviral vector-mediated control of HIV-1 replication in CD4 lymphocytes from diverse HIV+ infected patients grouped according to CD4 count and viral load. Mol Ther (2004) 9(6):902-913. • Preclinical development and data for the first LV (VRX-496) approved for clinical trials. (Pubitemid 38878136)
25. Davis BM, Humeau L, Dropulic B: In vivo selection for human and murine hematopoietic cells transduced with a therapeutic MGMT lentiviral vector that inhibits HIV replication. Mol Ther (2004) 9(2):160-172. (Pubitemid 38256542)
26. Lu X, Humeau L, Slepushkin V, Binder G, Yu Q, Slepushkina T, Chen Z, Merling R, Davis B, Chang YN, Dropulic B: 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 (2004) 6(9):963-973. (Pubitemid 40228111)
27. Lu X, Yu Q, Binder GK, Chen Z, Slepushkina T, Rossi J, Dropulic B: 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 (2004) 78(13):7079-7088. (Pubitemid 38781546)
28. Manilla P, Rebello T, Afable C, Lu X, Slepushkin V, Humeau LM, Schonely K, Ni Y, Binder GK, Levine BL, MacGregor RR et al: Regulatory considerations for novel gene therapy products: A review of the process leading to the first clinical lentiviral vector. Hum Gene Ther (2005) 16(1):17-25. (Pubitemid 40250328)
29. McGarrity G, Slepushkin V, Humeau L, Rebello T, Afable C, Levine BL, June CH, Smart A: Exposure to lentiviral vector in clinical gene therapy trials: Preliminary report. 47th Annual Interscience Conference on Antimicrobial Agents and Chemotherapy (2007).
30. Cartier N, Hacein-Bey-Abina S, Veres G, Vidaud M, Dal-Cortivo L, Caccavelli L, Malhaoui N, Kiermer V, Mittlestaedt D, Simmons A, bellesme C et al: Preliminary data from the first hematopoietic stem cell gene therapy trial with lentiviral vector demonstrate expression of the therapeutic protein in high percentage of lymphocytes and monocytes in two patients with X-linked adrenoleukodystrophy. The XV Annual Congress of the European Society of Gene and Cell Therapy, Rotterdam, the Netherlands (2007). •• Preliminary data from a phase I clinical trial with an LV for the potential treatment of ALD.
31. Benhamida S, Pflumio F, Dubart-Kupperschmitt A, Zhao-Emonet JC, Cavazzana-Calvo M, Rocchiccioli F, Fichelson S, Aubourg P, Charneau P, Cartier N: Transduced CD34+ cells from adrenoleukodystrophy patients with HIV-derived vector mediate long-term engraftment of NOD/SCID mice. Mol Ther (2003) 7(3):317-324. • Preclinical studies for the first LV gene therapy clinical trials in CD34+ hematopoietic progenitor cells. (Pubitemid 36503855)
32. Cartier N, Aubourg P: Hematopoietic stem cell gene therapy in Hurler syndrome, globoid cell leukodystrophy, metachromatic leukodystrophy and X-adrenoleukodystrophy. Curr Opin Mol Ther (2008) 10(5):471-478.
33. Williams DA: ESCGT 2008: Progress in clinical gene therapy. Mol Ther (2009) 17(1):1-2. •• A summary of the latest data from phase I clinical trials using LVs for the treatment of ALD and Parkinson's disease, presented at the 2008 European Society of Gene and Cell Therapy annual meeting.
34. Cartier-Lacave N: Hematopoietic stem cell gene therapy with lentiviral vectors in X-Adrenoleukodystrophy. American Society of Gene Therapy, San Diego, CA, USA (2009).
35. Sadelain M, Wang CH, Antoniou M, Grosveld F, Mulligan RC: Generation of a high-titer retroviral vector capable of expressing high levels of the human β-globin gene. Proc Natl Acad Sci USA (1995) 92(15):6728-6732.
36. May C, Rivella S, Callegari J, Heller G, Gaensler KM, Luzzatto L, Sadelain M: Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature (2000) 406(6791):82-86. (Pubitemid 30460215)
37. Imren S, Payen E, Westerman KA, Pawliuk R, Fabry ME, Eaves CJ, Cavilla B, Wadsworth LD, Beuzard Y, Bouhassira EE, Russell R et al: Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci USA (2002) 99(22):14380-14385. (Pubitemid 35257680)
38. Rivella S, May C, Chadburn A, Riviere I, Sadelain M: A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer. Blood (2003) 101(8):2932-2939. (Pubitemid 36857977)
39. Puthenveetil G, Scholes J, Carbonell D, Qureshi N, Xia P, Zeng L, Li S, Yu Y, Hiti AL, Yee JK, Malik P: Successful correction of the human β-thalassemia major phenotype using a lentiviral vector. Blood (2004) 104(12):3445-3453. (Pubitemid 39564412)
40. Technology and products: Genetix Pharmaceuticals Inc, Cambridge, MA, USA (2007). www.genetixpharm.com/tech-prod.html
41. Bank A, Dorazio R, Leboulch P: A phase I/II clinical trial of β-globin gene therapy for β-thalassemia. Ann NY Acad Sci (2005) 1054:308-316. •• Review of the development of LVs for the treatment of sickle cell disease and β-thalassemia.
42. Bank A: On the road to gene therapy for β-thalassemia and sickle cell anemia. Pediatr Hematol Oncol (2008) 25(1):1-4.
43. Leboulch P: Clinical benefit with clonal dominance after lentiviral gene therapy in severe human thalassemia. American Society of Gene Therapy, San Diego, CA, USA (2009).
44. Li MJ, Kim J, Li S, Zaia J, Yee JK, Anderson J, Akkina R, Rossi JJ: 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 (2005) 12(5):900-909. •• Preclinical studies of an LV-expressing combination RNAi for HIV gene therapy.
45. Li M, Li H, Rossi JJ: RNAi in combination with a ribozyme and TAR decoy for treatment of HIV infection in hematopoietic cell gene therapy. Ann NY Acad Sci (2006) 1082:172-179. (Pubitemid 44955461)
46. Bobisse S, Zanovello P, Rosato A: T-cell receptor gene transfer by lentiviral vectors in adoptive cell therapy. Expert Opin Biol Ther (2007) 7(6):893-906. (Pubitemid 46957423)
47. Chen X, Gao W, Gambotto A, Finn OJ: Lentiviral vectors encoding human MUC1-specific, MHC-unrestricted single-chain TCR and a fusion suicide gene: Potential for universal and safe cancer immunotherapy. Cancer Immunol Immunother (2009) 58(6):977-987.
48. Chhabra A, Yang L, Wang P, Comin-Anduix B, Das R, Chakraborty NG, Ray S, Mehrotra S, Yang H, Hardee CL, Hollis R et al: CD4+CD25- T cells transduced to express MHC class I-restricted epitope-specific TCR synthesize Th1 cytokines and exhibit MHC class I-restricted cytolytic effector function in a human melanoma model. J Immunol (2008) 181(2):1063-1070.
49. Circosta P, Granziero L, Follenzi A, Vigna E, Stella S, Vallario A, Elia AR, Gammaitoni L, Vitaggio K, Orso F, Geuna M et al: TCR gene transfer with lentiviral vectors allows efficient redirection of tumor specificity in naive and memory T-cells without prior stimulation of endogenous TCR. Hum Gene Ther (2009): doi:10.1089/hum.2009.117
50. Stauss HJ, Thomas S, Cesco-Gaspere M, Hart DP, Xue SA, Holler A, King J, Wright G, Perro M, Pospori C, Morris E: WT1-specific T cell receptor gene therapy: Improving TCR function in transduced T cells. Blood Cells Mol Dis (2008) 40(1):113-116. (Pubitemid 350192301)
51. Yang S, Cohen CJ, Peng PD, Zhao Y, Cassard L, Yu Z, Zheng Z, Jones S, Restifo NP, Rosenberg SA, Morgan RA: Development of optimal bicistronic lentiviral vectors facilitates high-level TCR gene expression and robust tumor cell recognition. Gene Ther (2008) 15(21):1411-1423.
52. Yang S, Rosenberg SA, Morgan RA: Clinical-scale lentiviral vector transduction of PBL for TCR gene therapy and potential for expression in less-differentiated cells. J Immunother (2008) 31(9):830-839.
53. Rowe HM, Lopes L, Ikeda Y, Bailey R, Barde I, Zenke M, Chain BM, Collins MK: Immunization with a lentiviral vector stimulates both CD4 and CD8 T cell responses to an ovalbumin transgene. Mol Ther (2006) 13(2):310-319. (Pubitemid 43056896)
54. Giles FJ, Keating A, Goldstone AH, Avivi I, Willman CL, Kantarjian HM: Acute myeloid leukemia. Hematology Am Soc Hematol Educ Program (2002):73-110.
55. Galea-Lauri J: Immunological weapons against acute myeloid leukaemia. Immunology (2002) 107(1):20-27.
56. Chan L, Hardwick N, Darling D, Galea-Lauri J, Gaken J, Devereux S, Kemeny M, Mufti G, Farzaneh F: IL-2/B7.1 (CD80) fusagene transduction of AML blasts by a self-inactivating lentiviral vector stimulates T cell responses in vitro: A strategy to generate whole cell vaccines for AML. Mol Ther (2005) 11(1):120-131. (Pubitemid 40012006)
57. Ingram W, Chan L, Guven H, Darling D, Kordasti S, Hardwick N, Barber L, Mufti GJ, Farzaneh F: Human CD80/IL2 lentivirus-transduced acute myeloid leukaemia (AML) cells promote natural killer (NK) cell activation and cytolytic activity: Implications for a phase I clinical study. Br J Haematol (2009) 145(6):749-760.
58. Azzouz M, Martin-Rendon E, Barber RD, Mitrophanous KA, Carter EE, Rohll JB, Kingsman SM, Kingsman AJ, Mazarakis ND: Multicistronic lentiviral vector-mediated striatal gene transfer of aromatic L-amino acid decarboxylase, tyrosine hydroxylase, and GTP cyclohydrolase I induces sustained transgene expression, dopamine production, and functional improvement in a rat model of Parkinson's disease. J Neurosci (2002) 22(23):10302-10312. •• An EIAV-derived LV expressing three transgenes required for the synthesis of dopamine demonstrated improvements in a rat model of in Parkinson's disease.
59. Azzouz M, Mazarakis N: Non-primate EIAV-based lentiviral vectors as gene delivery system for motor neuron diseases. Curr Gene Ther (2004) 4(3):277-286. (Pubitemid 39162389)
60. Oxford Biomedica: Oxford Biomedica announces update on phase I/II study of ProSavin® in Parkinson's Disease. Press Release (2009): July 13.
61. Jarraya B, Ralph S, Lepetit H, Stratful E, Boulet S, Jan C, Bonvento G, Azzouz M, Miskin J, Gurruchaga J-M: A phase I/II trial for Parkinson's disease using a lentiviral vector (ProSavin). American Society of Gene Therapy Annual Meeting, San Diego, CA, USA (2009) 12:Abs 514.
62. Hofling AA, Devine S, Vogler C, Sands MS: Human CD34+ hematopoietic progenitor cell-directed lentiviral-mediated gene therapy in a xenotransplantation model of lysosomal storage disease. Mol Ther (2004) 9(6):856-865. (Pubitemid 38878131)
63. Badour K, Zhang J, Siminovitch KA: The Wiskott-Aldrich syndrome protein: Forging the link between actin and cell activation. Immunol Rev (2003) 192:98-112. (Pubitemid 36613326)
64. Sasahara Y, Rachid R, Byrne MJ, de la Fuente MA, Abraham RT, Ramesh N, Geha RS: Mechanism of recruitment of WASp to the immunological synapse and of its activation following TCR ligation. Mol Cell (2002) 10(6):1269-1281. (Pubitemid 36050870)
65. Marangoni F, Bosticardo M, Charrier S, Draghici E, Locci M, Scaramuzza S, Panaroni C, Ponzoni M, Sanvito F, Doglioni C, Liabeuf M et al: Evidence for long-term efficacy and safety of gene therapy for Wiskott-Aldrich syndrome in preclinical models. Mol Ther (2009) 17(6):1073-1082.
66. Zanta-Boussif MA, Charrier S, Brice-Ouzet A, Martin S, Opolon P, Thrasher AJ, Hope TJ, Galy A: 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 (2009) 16(5):605-619.
67. Charrier S, Dupré L, Scaramuzza S, Jeanson-Leh L, Blundell MP, Danos O, Cattaneo F, Aiuti A, Eckenberg R, Thrasher AJ, Roncarolo MG et al: Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients. Gene Ther (2007) 14(5):415-428. (Pubitemid 46306542)
68. Galy A, Roncarolo MG, Thrasher AJ: Development of lentiviral gene therapy for Wiskott Aldrich syndrome. Expert Opin Biol Ther (2008) 8(2):181-190. (Pubitemid 351233711)
69. Genethon Clinical Products: Genethon, Evry Cedex (2009). www.genethon.fr/index.php?id=46&no-cache=1&page=1&L=1
70. Sadelain M, Boulad F, Lisowki L, Moi P, Riviere I: Stem cell engineering for the treatment of severe hemoglobinopathies. Curr Mol Med (2008) 8(7):690-697. • Comprehensive review on the development of gene transfer strategies for hemoglobinopathies.
71. + hematopoietic progenitors at low multiplicity of infection confers significant resistance to BG/BCNU and allows selection in vitro. Mol Ther (2002) 5(4):381-387.
72. Liu L, Gerson SL: Targeted modulation of MGMT: Clinical implications. Clin Cancer Res (2006) 12(2):328-331. (Pubitemid 43166117)
73. Trials open for enrollment: Lentigen Corp, Gaithersburg, MD, USA (2008). www.lentigen.com/patients/open-trials
74. LG631: Lentigen Corp, Gaithersburg, MD, USA (2008). www.lentigen.com/ products/lg631
75. Adaptimmune Ltd: Adaptimmune engineers immune cells able to clear HIV - clinical trials planned. Press Release (2008):November 09.
76. Varela-Rohena A, Molloy PE, Dunn SM, Li Y, Suhoski MM, Carroll RG, Milicic A, Mahon T, Sutton DH, Laugel B, Moysey R et al: Control of HIV-1 immune escape by CD8 T cells expressing enhanced T-cell receptor. Nat Med (2008) 14(12):1390-1395.
77. Recombinant DNA advisory committee - Minutes of meeting: NIH, Bethesda, MD, USA (2009). oba.od.nih.gov/oba/RAC/meetings/mar2009/March%202009%20minutes. pdf
78. Lemiale F, Korokhov N: Lentiviral vectors for HIV disease prevention and treatment. Vaccine (2009) 27(25-26):3443-3449.
79. Dullaers M, Thielemans K: From pathogen to medicine: HIV-1-derived lentiviral vectors as vehicles for dendritic cell based cancer immunotherapy. J Gene Med (2006) 8(1):3-17. (Pubitemid 43169376)
80. Palmowski MJ, Lopes L, Ikeda Y, Salio M, Cerundolo V, Collins MK: Intravenous injection of a lentiviral vector encoding NY-ESO-1 induces an effective CTL response. J Immunol (2004) 172(3):1582-1587. (Pubitemid 38116804)
81. Iglesias MC, Mollier K, Beignon AS, Souque P, Adotevi O, Lemonnier F, Charneau P: Lentiviral vectors encoding HIV-1 polyepitopes induce broad CTL responses in vivo. Mol Ther (2007) 15(6):1203-1210. (Pubitemid 46813625)
82. Beignon AS, Mollier K, Liard C, Coutant F, Munier S, Rivière J, Souque P, Charneau P: A Lentiviral vector-based prime/boost vaccination against AIDS: A pilot study shows protection against SIVmac251 challenge in macaques. J Virol (2009): doi:10.1128/JVI.01284-09.
83. Lopes L, Dewannieux M, Gileadi U, Bailey R, Ikeda Y, Whittaker C, Collin MP, Cerundolo V, Tomihari M, Ariizumi K, Collins MK: Immunization with a lentivector that targets tumor antigen expression to dendritic cells induces potent CD8+ and CD4+ T-cell responses. J Virol (2008) 82(1):86-95. (Pubitemid 350309126)
84. Negri DR, Michelini Z, Baroncelli S, Spada M, Vendetti S, Buffa V, Bona R, Leone P, Klotman ME, Cara A: Successful immunization with a single injection of non-integrating lentiviral vector. Mol Ther (2007) 15(9):1716-1723. (Pubitemid 47289016)
85. Coutant F, Frenkiel MP, Despres P, Charneau P: Protective antiviral immunity conferred by a nonintegrative lentiviral vector-based vaccine. PLoS ONE (2008) 3(12):e3973.
86. Board of the European Society of Gene and Cell Therapy: Case of leukaemia associated with X-linked severe combined immunodeficiency gene therapy trial in London. Hum Gene Ther (2008) 19(1):3-4.
87. Hacein-Bey-Abina S, Fischer A, Cavazzana-Calvo M: Gene therapy of X-linked severe combined immunodeficiency. Int J Hematol (2002) 76(4):295-298. (Pubitemid 43014090)
88. Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E, Clappier E, Caccavelli L, Delabesse E, Beldjord K, Asnafi V et al: Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest (2008) 118(9):3132-3142.
89. Hacein-Bey-Abina S, Von Kalle C, Schmidt M, McCormack MP, Wulffraat N, Leboulch P, Lim A, Osborne CS, Pawliuk R, Morillon E, Sorensen R et al: LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science (2003) 302(5644):415-419. (Pubitemid 37296260)
90. Aiuti A, Cattaneo F, Galimberti S, Benninghoff U, Cassani B, Callegaro L, Scaramuzza S, Andolfi G, Mirolo M, Brigida I, Tabucchi A et al: Gene therapy for immunodeficiency due to adenosine deaminase deficiency. N Engl J Med (2009) 360(5):447-458.
91. Wu X, Li Y, Crise B, Burgess SM: Transcription start regions in the human genome are favored targets for MLV integration. Science (2003) 300(5626):1749-1751. (Pubitemid 36712571)
92. Schröder AR, Shinn P, Chen H, Berry C, Ecker JR, Bushman F: HIV-1 integration in the human genome favors active genes and local hotspots. Cell (2002) 110(4):521-529. •• Analysis of 903 MLV and 379 HIV-1 integrations in the human genome demonstrated that HIV-1 did not have preferred integration sites.
93. Mitchell RS, Beitzel BF, Schroder AR, Shinn P, Chen H, Berry CC, Ecker JR, Bushman FD: Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol (2004) 2(8):E234. (Pubitemid 39245268)
94. Hacker CV, Vink CA, Wardell TW, Lee S, Treasure P, Kingsman SM, Mitrophanous KA, Miskin JE: The integration profile of EIAV-based vectors. Mol Ther (2006) 14(4):536-545. • Reports that EIAV vectors, similar to LVs, did not have preferred integration sites in the human genome.
95. Hacker CV, Ralph GS, Kingsman SM, Mitrophanous KA, Miskin JE: Determination of the profile and site preference of lentiviral vector integration in primary astrocyte cultures. Blood Cells Mol Dis (2008) 40(2):269-269
96. Faschinger A, Rouault F, Sollner J, Lukas A, Salmons B, Gunzburg W, Indik S: Mouse mammary tumor virus integration site selection in human and mouse genomes. J Virol (2008) 82(3):1360-1367.
97. Narezkina A, Taganov KD, Litwin S, Stoyanova R, Hayashi J, Seeger C, Skalka AM, Katz RA: Genome-wide analyses of avian sarcoma virus integration sites. J Virol (2004) 78(21):11656-11663. (Pubitemid 39390752)
98. Hargrove PW, Kepes S, Hanawa H, Obenauer JC, Pei D, Cheng C, Gray JT, Neale G, Persons DA: Globin lentiviral vector insertions can perturb the expression of endogenous genes in β-thalassemic hematopoietic cells. Mol Ther (2008) 16(3):525-533.
99. Montini E, Cesana D, Schmidt M, Sanvito F, Ponzoni M, Bartholomae C, Sergi Sergi L, Benedicenti F, Ambrosi A, Di Serio C, Doglioni C et al: Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol (2006) 24(6):687-696. (Pubitemid 43882120)
100. Bauer G, Dao MA, Case SS, Meyerrose T, Wirthlin L, Zhou P, Wang X, Herrbrich P, Arevalo J, Csik S, Skelton DC et al: In vivo biosafety model to assess the risk of adverse events from retroviral and lentiviral vectors. Mol Ther (2008) 16(7):1308-1315. (Pubitemid 351866063)
101. Newrzela S, Cornils K, Li Z, Baum C, Brugman MH, Hartmann M, Meyer J, Hartmann S, Hansmann ML, Fehse B, von Laer D: Resistance of mature T cells to oncogene transformation. Blood (2008) 112(6):2278-2286.
102. Themis M, Waddington SN, Schmidt M, von Kalle C, Wang Y, Al-Allaf F, Gregory LG, Nivsarkar M, Themis M, Holder MV, Buckley SM et al: Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther (2005) 12(4):763-771. (Pubitemid 41350158)
103. Wang GP, Levine BL, Binder GK, Berry CC, Malani N, McGarrity G, Tebas P, June CH, Bushman FD: Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modified CD4+ T cells. Mol Ther (2009) 17(5):844-850.
104. Ni Y, Sun S, Oparaocha I, Humeau L, Davis B, Cohen R, Binder G, Chang YN, Slepushkin V, Dropulic B: Generation of a packaging cell line for prolonged large-scale production of high-titer HIV-1-based lentiviral vector. J Gene Med (2005) 7(6):818-834. (Pubitemid 40872867)
105. Throm RE, Ouma AA, Zhou S, Chandrasekaran A, Lockey T, Greene M, De Ravin SS, Moayeri M, Malech HL, Sorrentino BP, Gray JT: Efficient construction of producer cell lines for a SIN lentiviral vector for SCID-X1 gene therapy by concatemeric array transfection. Blood (2009) 113(21):5104-5110.