Gammaretroviral vectors: Biology, technology and application

Viruses

Volumn 3, Issue 6, 2011, Pages 677-713

  Maetzig, Tobias ^a   Galla, Melanie ^a   Baum, Christopher ^a   Schambach, Axel ^a  

^a HANNOVER MEDICAL SCHOOL   (Germany)

Author keywords

Gammaretroviral; Murine leukemia virus; SIN vector; Split packaging design

Indexed keywords

ADENOSINE DEAMINASE; DRUG VEHICLE; RETROVIRUS VECTOR; SELF INACTIVATING VECTOR; UNCLASSIFIED DRUG;

3' UNTRANSLATED REGION; ACUTE LYMPHOBLASTIC LEUKEMIA; ADRENOLEUKODYSTROPHY; BIOSAFETY; BIOTECHNOLOGY; CHRONIC GRANULOMATOUS DISEASE; CLINICAL TRIAL (TOPIC); ENHANCER REGION; GENE EXPRESSION REGULATION; GENE INSERTION; GENE INTERACTION; GENE TRANSFER; GENETIC MANIPULATION; GENETIC ORGANIZATION; HEMATOPOIETIC STEM CELL; HUMAN; LIFE CYCLE; MURINE LEUKEMIA VIRUS; NONHUMAN; PROMOTER REGION; PSEUDOTYPING; REVIEW; SEVERE COMBINED IMMUNODEFICIENCY; STEM CELL GENE THERAPY; T CELL LEUKEMIA; UNSPECIFIED SIDE EFFECT; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIROLOGY; VIRUS GENOME; VIRUS INACTIVATION; VIRUS INFECTIVITY; VIRUS LIFE CYCLE; VIRUS PARTICLE; WISKOTT ALDRICH SYNDROME;

ANIMALS; GENE THERAPY; GENETIC VECTORS; HUMANS; LEUKEMIA VIRUS, MURINE;

GAMMARETROVIRUS; MURINE LEUKEMIA VIRUS;

EID: 79957792110     PISSN: None     EISSN: 19994915     Source Type: Journal    
DOI: 10.3390/v3060677     Document Type: Review

References (210)

1. Temin, H.M.; Mizutani, S. RNA-dependent DNA polymerase in virions of Rous sarcoma virus. Nature 1970, 226, 1211-1213.
2. Shimotohno, K.; Temin, H.M. Formation of infectious progeny virus after insertion of herpes simplex thymidine kinase gene into DNA of an avian retrovirus. Cell 1981, 26, 67-77.
3. Wei, C.M.; Gibson, M.; Spear, P.G.; Scolnick, E.M. Construction and isolation of a transmissible retrovirus containing the src gene of Harvey murine sarcoma virus and the thymidine kinase gene of herpes simplex virus type 1. J. Virol. 1981, 39, 935-944.
4. Gilboa, E.; Kolbe, M.; Noonan, K.; Kucherlapati, R. Construction of a mammalian transducing vector from the genome of Moloney murine leukemia virus. J. Virol. 1982, 44, 845-851.
5. Tabin, C.J.; Hoffmann, J.W.; Goff, S.P.; Weinberg, R.A. Adaptation of a retrovirus as a eucaryotic vector transmitting the herpes simplex virus thymidine kinase gene. Mol. Cell Biol. 1982, 2, 426-436.
6. Sorge, J.; Hughes, S.H. Splicing of intervening sequences introduced into an infectious retroviral vector. J. Mol. Appl. Genet. 1982, 1, 547-559.
7. Joyner, A.; Keller, G.; Phillips, R.A.; Bernstein, A. Retrovirus transfer of a bacterial gene into mouse haematopoietic progenitor cells. Nature 1983, 305, 556-558.
8. Hwang, L.H.; Gilboa, E. Expression of genes introduced into cells by retroviral infection is more efficient than that of genes introduced into cells by DNA transfection. J. Virol. 1984, 50, 417-424.
9. Eglitis, M.A.; Kantoff, P.; Gilboa, E.; Anderson, W.F. Gene expression in mice after high efficiency retroviral-mediated gene transfer. Science 1985, 230, 1395-1398.
10. Heinkelein, M.; Dressler, M.; Jármy, G.; Rammling, M.; Imrich, H.; Thurow, J.; Lindemann, D.; Rethwilm, A. Improved foamy virus vectors and packaging constructs. J. Virol. 2002, 76, 3774-3783.
11. Trobridge, G.; Josephson, N.; Vassilopoulos, G.; Mac, J.; Russell, D.W. Improved foamy virus vectors with minimal viral sequences. Mol. Ther. 2002, 6, 321-328.
12. Naldini, L.; Blomer, U.; Gallay, P.; Ory, D.; Mulligan, R.; Gage, F.H.; Verma, I.M.; Trono, D. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996, 272, 263-267.
13. Dull, T.; Zufferey, R.; Kelly, M.; Mandel, R.J.; Nguyen, M.; Trono, D.; Naldini, L. A third-generation lentivirus vector with a conditional packaging system. J. Virol. 1998, 72, 8463-8471.
14. Negre, D.; Duisit, G.; Mangeot, P.E.; Moullier, P.; Darlix, J.L.; Cosset, F.L. Lentiviral vectors derived from simian immunodeficiency virus. Curr. Top. Microbiol. Immunol. 2002, 261, 53-74.
15. Matukonis, M.; Li, M.; Molina, R.P.; Paszkiet, B.; Kaleko, M.; Luo, T. Development of second- and third-generation bovine immunodeficiency virus-based gene transfer systems. Hum. Gene Ther. 2002, 13, 1293-1303.
16. Poeschla, E.; Gilbert, J.; Li, X.; Huang, S.; Ho, A.; Wong-Staal, F. 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. 1998, 72, 6527-6536.
17. Olsen, J.C. Gene transfer vectors derived from equine infectious anemia virus. Gene Ther. 1998, 5, 1481-1487.
18. Derse, D.; Martarano, L. Construction of a recombinant bovine leukemia virus vector for analysis of virus infectivity. J. Virol. 1990, 64, 401-405.
19. Hughes, S.H. The RCAS vector system. Folia Biol. (Praha) 2004, 50, 107-119.
20. Suerth, J.D.; Maetzig, T.; Galla, M.; Baum, C.; Schambach, A. Self-inactivating alpharetroviral vectors with a split-packaging design. J. Virol. 2010, 84, 6626-6635.
21. Watanabe, S.; Temin, H.M. Encapsidation sequences for spleen necrosis virus, an avian retrovirus, are between the 5' long terminal repeat and the start of the gag gene. Proc. Natl. Acad. Sci. U. S. A. 1982, 79, 5986-5990.
22. Gunzburg, W.H.; Salmons, B. Mouse mammary tumor virus mediated transfer and expression of neomycin resistance to infected cultured cells. Virology 1986, 155, 236-248.
23. Boris-Lawrie, K.; Temin, H.M. The retroviral vector. Replication cycle and safety considerations for retrovirus-mediated Gene therapy. Ann. New York Acad. Sci. 1994, 716, 59-70; discussion 71.
24. Baum, C.; Schambach, A.; Bohne, J.; Galla, M. Retrovirus vectors: toward the plentivirus? Mol. Ther. 2006, 13, 1050-1063.
25. Kraunus, J.; Zychlinski, D.; Heise, T.; Galla, M.; Bohne, J.; Baum, C. Murine leukemia virus regulates alternative splicing through sequences upstream of the 5' splice site. J. Biol. Chem. 2006, 281, 37381-37390.
26. Zychlinski, D.; Erkelenz, S.; Melhorn, V.; Baum, C.; Schaal, H.; Bohne, J. Limited complementarity between U1 snRNA and a retroviral 5' splice site permits its attenuation via RNA secondary structure. Nucleic Acids Res. 2009, 37, 7429-7440.
27. Freed, E.O.; Risser, R. The role of envelope glycoprotein processing in murine leukemia virus infection. J. Virol. 1987, 61, 2852-2856.
28. Sjoberg, M.; Wallin, M.; Lindqvist, B.; Garoff, H. Furin cleavage potentiates the membrane fusion-controlling intersubunit disulfide bond isomerization activity of leukemia virus Env. J. Virol. 2006, 80, 5540-5551.
29. Sommerfelt, M.A. Retrovirus receptors. J. Gen. Virol. 1999, 80, 3049-3064.
30. Overbaugh, J.; Miller, A.D.; Eiden, M.V. Receptors and entry cofactors for retroviruses include single and multiple transmembrane-spanning proteins as well as newly described glycophosphatidylinositol-anchored and secreted proteins. Microbiol. Mol. Biol. Rev. 2001, 65, 371-389, table of contents.
31. Risco, C.; Menendez-Arias, L.; Copeland, T.D.; Pinto da Silva, P.; Oroszlan, S. Intracellular transport of the murine leukemia virus during acute infection of NIH 3T3 cells: nuclear import of nucleocapsid protein and integrase. J. Cell Sci. 1995, 108, 3039-3050.
32. Fassati, A.; Goff, S.P. Characterization of intracellular reverse transcription complexes of Moloney murine leukemia virus. J. Virol. 1999, 73, 8919-8925.
33. Coffin, J.M. Structure, replication, and recombination of retrovirus genomes: some unifying hypotheses. J. Gen. Virol. 1979, 42, 1-26.
34. van Wamel, J.L.; Berkhout, B. The first strand transfer during HIV-1 reverse transcription can occur either intramolecularly or intermolecularly. Virology 1998, 244, 245-251.
35. Riviere, L.; Darlix, J.L.; Cimarelli, A. Analysis of the viral elements required in the nuclear import of HIV-1 DNA. J. Virol. 2010, 84, 729-739.
36. Yamashita, M.; Perez, O.; Hope, T.J.; Emerman, M. Evidence for direct involvement of the capsid protein in HIV infection of nondividing cells. PLoS Pathog. 2007, 3, 1502-1510.
37. Yamashita, M.; Emerman, M. Capsid is a dominant determinant of retrovirus infectivity in nondividing cells. J. Virol. 2004, 78, 5670-5678.
38. Yamashita, M.; Emerman, M. The cell cycle independence of HIV infections is not determined by known karyophilic viral elements. PLoS Pathog. 2005, 1, e18.
39. Kaushik, R.; Zhu, X.; Stranska, R.; Wu, Y.; Stevenson, M. A cellular restriction dictates the permissivity of nondividing monocytes/macrophages to lentivirus and gammaretrovirus infection. Cell Host Microbe 2009, 6, 68-80.
40. Miller, D.G.; Adam, M.A.; Miller, A.D. Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol. Cell Biol. 1990, 10, 4239-4242.
41. Roe, T.; Reynolds, T.C.; Yu, G.; Brown, P.O. Integration of murine leukemia virus DNA depends on mitosis. EMBO J. 1993, 12, 2099-2108.
42. Lewis, P.F.; Emerman, M. Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J. Virol. 1994, 68, 510-516.
43. Bowerman, B.; Brown, P.O.; Bishop, J.M.; Varmus, H.E. A nucleoprotein complex mediates the integration of retroviral DNA. Genes Dev. 1989, 3, 469-478.
44. Li, L.; Yoder, K.; Hansen, M.S.; Olvera, J.; Miller, M.D.; Bushman, F.D. Retroviral cDNA integration: stimulation by HMG I family proteins. J. Virol. 2000, 74, 10965-10974.
45. Prizan-Ravid, A.; Elis, E.; Laham-Karam, N.; Selig, S.; Ehrlich, M.; Bacharach, E. The Gag cleavage product, p12, is a functional constituent of the murine leukemia virus pre-integration complex. PLoS Pathog. 2010, 6, e1001183.
46. Lee, M.S.; Craigie, R. A previously unidentified host protein protects retroviral DNA from autointegration. Proc. Natl. Acad. Sci. U. S. A. 1998, 95, 1528-1533.
47. Suzuki, Y.; Craigie, R. Regulatory mechanisms by which barrier-to-autointegration factor blocks autointegration and stimulates intermolecular integration of Moloney murine leukemia virus preintegration complexes. J. Virol. 2002, 76, 12376-12380.
48. Schwartzberg, P.; Colicelli, J.; Goff, S.P. Construction and analysis of deletion mutations in the pol gene of Moloney murine leukemia virus: A new viral function required for productive infection. Cell 1984, 37, 1043-1052.
49. Studamire, B.; Goff, S.P. Host proteins interacting with the Moloney murine leukemia virus integrase: multiple transcriptional regulators and chromatin binding factors. Retrovirology 2008, 5, 48.
50. Lin, C.W.; Engelman, A. The barrier-to-autointegration factor is a component of functional human immunodeficiency virus type 1 preintegration complexes. J. Virol. 2003, 77, 5030-5036.
51. Hindmarsh, P.; Leis, J. Retroviral DNA integration. Microbiol. Mol. Biol. Rev. 1999, 63, 836-843, table of contents.
52. Gruter, P.; Tabernero, C.; von Kobbe, C.; Schmitt, C.; Saavedra, C.; Bachi, A.; Wilm, M.; Felber, B.K.; Izaurralde, E. TAP, the human homolog of Mex67p, mediates CTE-dependent RNA export from the nucleus. Mol. Cell 1998, 1, 649-659.
53. Bogerd, H.P.; Echarri, A.; Ross, T.M.; Cullen, B.R. Inhibition of human immunodeficiency virus Rev and human T-cell leukemia virus Rex function, but not Mason-Pfizer monkey virus constitutive transport element activity, by a mutant human nucleoporin targeted to Crm1. J. Virol. 1998, 72, 8627-8635.
54. Malim, M.H.; Hauber, J.; Le, S.Y.; Maizel, J.V.; Cullen, B.R. The HIV-1 rev trans-activator acts through a structured target sequence to activate nuclear export of unspliced viral mRNA. Nature 1989, 338, 254-257.
55. Schambach, A.; Bohne, J.; Chandra, S.; Will, E.; Margison, G.P.; Williams, D.A.; Baum, C. Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O6-methylguanine-DNA methyltransferase in hematopoietic cells. Mol. Ther. 2006, 13, 391-400.
56. Schambach, A.; Mueller, D.; Galla, M.; Verstegen, M.M.; Wagemaker, G.; Loew, R.; Baum, C.; Bohne, J. Overcoming promoter competition in packaging cells improves production of self-inactivating retroviral vectors. Gene Ther. 2006, 13, 1524-1533.
57. Felsenstein, K.M.; Goff, S.P. Expression of the gag-pol fusion protein of Moloney murine leukemia virus without gag protein does not induce virion formation or proteolytic processing. J. Virol. 1988, 62, 2179-2182.
58. Yoshinaka, Y.; Katoh, I.; Copeland, T.D.; Oroszlan, S. Murine leukemia virus protease is encoded by the gag-pol gene and is synthesized through suppression of an amber termination codon. Proc. Natl. Acad. Sci. U. S. A. 1985, 82, 1618-1622.
59. Panganiban, A.T. Retroviral gag gene amber codon suppression is caused by an intrinsic cis-acting component of the viral mRNA. J. Virol. 1988, 62, 3574-3580.
60. Honigman, A.; Wolf, D.; Yaish, S.; Falk, H.; Panet, A. cis Acting RNA sequences control the gag-pol translation readthrough in murine leukemia virus. Virology 1991, 183, 313-319.
61. Rein, A.; McClure, M.R.; Rice, N.R.; Luftig, R.B.; Schultz, A.M. Myristylation site in Pr65gag is essential for virus particle formation by Moloney murine leukemia virus. Proc. Natl. Acad. Sci. U. S. A. 1986, 83, 7246-7250.
62. Schultz, A.M.; Rein, A. Unmyristylated Moloney murine leukemia virus Pr65gag is excluded from virus assembly and maturation events. J. Virol. 1989, 63, 2370-2373.
63. Beer, C.; Pedersen, L.; Wirth, M. Amphotropic murine leukaemia virus envelope protein is associated with cholesterol-rich microdomains. Virol. J. 2005, 2, 36.
64. Li, M.; Yang, C.; Tong, S.; Weidmann, A.; Compans, R.W. Palmitoylation of the murine leukemia virus envelope protein is critical for lipid raft association and surface expression. J. Virol. 2002, 76, 11845-11852.
65. Ailles, L.E.; Naldini, L. HIV-1-derived lentiviral vectors. Curr. Top. Microbiol. Immunol. 2002, 261, 31-52.
66. Hildinger, M.; Abel, K.L.; Ostertag, W.; Baum, C. Design of 5' untranslated sequences in retroviral vectors developed for medical use. J. Virol. 1999, 73, 4083-4089.
67. Yu, S.S.; Kim, J.M.; Kim, S. High efficiency retroviral vectors that contain no viral coding sequences. Gene Ther. 2000, 7, 797-804.
68. Kim, S.H.; Yu, S.S.; Park, J.S.; Robbins, P.D.; An, C.S.; Kim, S. Construction of retroviral vectors with improved safety, gene expression, and versatility. J. Virol. 1998, 72, 994-1004.
69. Baum, C.; Hegewisch-Becker, S.; Eckert, H.G.; Stocking, C.; Ostertag, W. Novel retroviral vectors for efficient expression of the multidrug-resistance (mdr-1) gene in early hemopoietic cells. J. Virol. 1995, 69, 7541-7547.
70. Robbins, P.B.; Yu, X.J.; Skelton, D.M.; Pepper, K.A.; Wasserman, R.M.; Zhu, L.; Kohn, D.B. Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells. J. Virol. 1997, 71, 9466-9474.
71. Schambach, A.; Wodrich, H.; Hildinger, M.; Bohne, J.; Krausslich, H.G.; Baum, C. Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol. Ther. 2000, 2, 435-445.
72. Mougel, M.; Tounekti, N.; Darlix, J.L.; Paoletti, J.; Ehresmann, B.; Ehresmann, C. Conformational analysis of the 5' leader and the gag initiation site of Mo-MuLV RNA and allosteric transitions induced by dimerization. Nucleic Acids Res. 1993, 21, 4677-4684.
73. Riviere, I.; Brose, K.; Mulligan, R.C. Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc. Natl. Acad. Sci. U. S. A. 1995, 92, 6733-6737.
74. Miller, A.D.; Rosman, G.J. Improved retroviral vectors for gene transfer and expression. Biotechniques 1989, 7, 980-982, 984-986, 989-990.
75. Luo, M.J.; Reed, R. Splicing is required for rapid and efficient mRNA export in metazoans. Proc. Natl. Acad. Sci. U. S. A. 1999, 96, 14937-14942.
76. Wolf, D.; Goff, S.P. TRIM28 mediates primer binding site-targeted silencing of murine leukemia virus in embryonic cells. Cell 2007, 131, 46-57.
77. Wolf, D.; Goff, S.P. Embryonic stem cells use ZFP809 to silence retroviral DNAs. Nature 2009, 458, 1201-1204.
78. Rowe, H.M.; Jakobsson, J.; Mesnard, D.; Rougemont, J.; Reynard, S.; Aktas, T.; Maillard, P.V.; Layard-Liesching, H.; Verp, S.; Marquis, J. et al. KAP1 controls endogenous retroviruses in embryonic stem cells. Nature 2010, 463, 237-240.
79. Grez, M.; Akgun, E.; Hilberg, F.; Ostertag, W. Embryonic stem cell virus, a recombinant murine retrovirus with expression in embryonic stem cells. Proc. Natl. Acad. Sci. U. S. A. 1990, 87, 9202-9206.
80. Challita, P.M.; Skelton, D.; el-Khoueiry, A.; Yu, X.J.; Weinberg, K.; Kohn, D.B. Multiple modifications in cis elements of the long terminal repeat of retroviral vectors lead to increased expression and decreased DNA methylation in embryonic carcinoma cells. J. Virol. 1995, 69, 748-755.
81. Hawley, R.G.; Lieu, F.H.; Fong, A.Z.; Hawley, T.S. Versatile retroviral vectors for potential use in Gene therapy. Gene Ther. 1994, 1, 136-138.
82. Lund, A.H.; Duch, M.; Lovmand, J.; Jorgensen, P.; Pedersen, F.S. Complementation of a primer binding site-impaired murine leukemia virus-derived retroviral vector by a genetically engineered tRNA-like primer. J. Virol. 1997, 71, 1191-1195.
83. Towers, G.J.; Goff, S.P. Post-entry restriction of retroviral infections. AIDS Rev. 2003, 5, 156-164.
84. Goff, S.P. Retrovirus restriction factors. Mol. Cell 2004, 16, 849-859.
85. Hacein-Bey-Abina, S.; von Kalle, C.; Schmidt, M.; Le Deist, F.; Wulffraat, N.; McIntyre, E.; Radford, I.; Villeval, J.L.; Fraser, C.C.; Cavazzana-Calvo, M. et al. A serious adverse event after successful Gene therapy for X-linked severe combined immunodeficiency. New Engl. J. Med. 2003, 348, 255-256.
86. Howe, S.J.; Mansour, M.R.; Schwarzwaelder, K.; Bartholomae, C.; Hubank, M.; Kempski, H.; Brugman, M.H.; Pike-Overzet, K.; Chatters, S.J.; de Ridder, D. et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 2008, 118, 3143-3150.
87. Stein, S.; Ott, M.G.; Schultze-Strasser, S.; Jauch, A.; Burwinkel, B.; Kinner, A.; Schmidt, M.; Kramer, A.; Schwable, J.; Glimm, H. et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat. Med. 2010, 16, 198-204.
88. Schucht, R.; Coroadinha, A.S.; Zanta-Boussif, M.A.; Verhoeyen, E.; Carrondo, M.J.; Hauser, H.; Wirth, D. A new generation of retroviral producer cells: predictable and stable virus production by Flp-mediated site-specific integration of retroviral vectors. Mol. Ther. 2006, 14, 285-292.
89. Mavilio, F.; Pellegrini, G.; Ferrari, S.; Di Nunzio, F.; Di Iorio, E.; Recchia, A.; Maruggi, G.; Ferrari, G.; Provasi, E.; Bonini, C. et al. Correction of junctional epidermolysis bullosa by transplantation of genetically modified epidermal stem cells. Nat. Med. 2006, 12, 1397-1402.
90. Ellis, J. Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum. Gene Ther. 2005, 16, 1241-1246.
91. Zhang, F.; Thornhill, S.I.; Howe, S.J.; Ulaganathan, M.; Schambach, A.; Sinclair, J.; Kinnon, C.; Gaspar, H.B.; Antoniou, M.; Thrasher, A.J. Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cells. Blood 2007, 110, 1448-1457.
92. Albritton, L.M.; Tseng, L.; Scadden, D.; Cunningham, J.M. A putative murine ecotropic retrovirus receptor gene encodes a multiple membrane-spanning protein and confers susceptibility to virus infection. Cell 1989, 57, 659-666.
93. Miller, D.G.; Edwards, R.H.; Miller, A.D. Cloning of the cellular receptor for amphotropic murine retroviruses reveals homology to that for gibbon ape leukemia virus. Proc. Natl. Acad. Sci. U. S. A. 1994, 91, 78-82.
94. Tailor, C.S.; Nouri, A.; Lee, C.G.; Kozak, C.; Kabat, D. Cloning and characterization of a cell surface receptor for xenotropic and polytropic murine leukemia viruses. Proc. Natl. Acad. Sci. U. S. A. 1999, 96, 927-932.
95. Brenner, S.; Whiting-Theobald, N.L.; Linton, G.F.; Holmes, K.L.; Anderson-Cohen, M.; Kelly, P.F.; Vanin, E.F.; Pilon, A.M.; Bodine, D.M.; Horwitz, M.E. et al. Concentrated RD114-pseudotyped MFGS-gp91phox vector achieves high levels of functional correction of the chronic granulomatous disease oxidase defect in NOD/SCID/beta -microglobulin-/- repopulating mobilized human peripheral blood CD34+ cells. Blood 2003, 102, 2789-2797.
96. Orlic, D.; Girard, L.J.; Jordan, C.T.; Anderson, S.M.; Cline, A.P.; Bodine, D.M. The level of mRNA encoding the amphotropic retrovirus receptor in mouse and human hematopoietic stem cells is low and correlates with the efficiency of retrovirus transduction. Proc. Natl. Acad. Sci. U. S. A. 1996, 93, 11097-11102.
97. Kavanaugh, M.P.; Miller, D.G.; Zhang, W.; Law, W.; Kozak, S.L.; Kabat, D.; Miller, A.D. Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters. Proc. Natl. Acad. Sci. U. S. A. 1994, 91, 7071-7075.
98. Porter, C.D.; Collins, M.K.; Tailor, C.S.; Parkar, M.H.; Cosset, F.L.; Weiss, R.A.; Takeuchi, Y. Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors. Hum. Gene Ther. 1996, 7, 913-919.
99. Yee, J.K.; Friedmann, T.; Burns, J.C. Generation of high-titer pseudotyped retroviral vectors with very broad host range. Methods Cell Biol. 1994, 43, 99-112.
100. Sandrin, V.; Boson, B.; Salmon, P.; Gay, W.; Negre, D.; Le Grand, R.; Trono, D.; Cosset, F.L. 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 2002, 100, 823-832.
101. Stitz, J.; Buchholz, C.J.; Engelstadter, M.; Uckert, W.; Bloemer, U.; Schmitt, I.; Cichutek, K. Lentiviral vectors pseudotyped with envelope glycoproteins derived from gibbon ape leukemia virus and murine leukemia virus 10A1. Virology 2000, 273, 16-20.
102. Stitz, J.; Steidl, S.; Merget-Millitzer, H.; Konig, R.; Muller, P.; Nocken, F.; Engelstadter, M.; Bobkova, M.; Schmitt, I.; Kurth, R. et al. MLV-derived retroviral vectors selective for CD4-expressing cells and resistant to neutralization by sera from HIV-infected patients. Virology 2000, 267, 229-236.
103. Chawla, S.P.; Chua, V.S.; Fernandez, L.; Quon, D.; Blackwelder, W.C.; Gordon, E.M.; Hall, F.L. Advanced phase I/II studies of targeted gene delivery in vivo: intravenous Rexin-G for gemcitabine-resistant metastatic pancreatic cancer. Mol. Ther. 2010, 18, 435-441.
104. Chawla, S.P.; Chua, V.S.; Fernandez, L.; Quon, D.; Saralou, A.; Blackwelder, W.C.; Hall, F.L.; Gordon, E.M. Phase I/II and phase II studies of targeted gene delivery in vivo: intravenous Rexin-G for chemotherapy-resistant sarcoma and osteosarcoma. Mol. Ther. 2009, 17, 1651-1657.
105. Vongpunsawad, S.; Oezgun, N.; Braun, W.; Cattaneo, R. Selectively receptor-blind measles viruses: Identification of residues necessary for SLAM- or CD46-induced fusion and their localization on a new hemagglutinin structural model. J. Virol. 2004, 78, 302-313.
106. Nakamura, T.; Peng, K.W.; Harvey, M.; Greiner, S.; Lorimer, I.A.; James, C.D.; Russell, S.J. Rescue and propagation of fully retargeted oncolytic measles viruses. Nat. Biotechnol. 2005, 23, 209-214.
107. Buchholz, C.J.; Muhlebach, M.D.; Cichutek, K. Lentiviral vectors with measles virus glycoproteins - dream team for gene transfer? Trends Biotechnol. 2009, 27, 259-265.
108. Markusic, D.M.; Kanitz, A.; Oude-Elferink, R.P.; Seppen, J. Preferential gene transfer of lentiviral vectors to liver-derived cells, using a hepatitis B peptide displayed on GP64. Hum. Gene Ther. 2007, 18, 673-679.
109. Miletic, H.; Fischer, Y.H.; Neumann, H.; Hans, V.; Stenzel, W.; Giroglou, T.; Hermann, M.; Deckert, M.; Von Laer, D. Selective transduction of malignant glioma by lentiviral vectors pseudotyped with lymphocytic choriomeningitis virus glycoproteins. Hum. Gene Ther. 2004, 15, 1091-1100.
110. Danos, O.; Mulligan, R.C. Safe and efficient generation of recombinant retroviruses with amphotropic and ecotropic host ranges. Proc. Natl. Acad. Sci. U. S. A. 1988, 85, 6460-6464.
111. Burns, J.C.; Friedmann, T.; Driever, W.; Burrascano, M.; Yee, J.K. 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. 1993, 90, 8033-8037.
112. Takeuchi, Y.; Cosset, F.L.; Lachmann, P.J.; Okada, H.; Weiss, R.A.; Collins, M.K. Type C retrovirus inactivation by human complement is determined by both the viral genome and the producer cell. J. Virol. 1994, 68, 8001-8007.
113. Funke, S.; Maisner, A.; Muhlebach, M.D.; Koehl, U.; Grez, M.; Cattaneo, R.; Cichutek, K.; Buchholz, C.J. Targeted cell entry of lentiviral vectors. Mol. Ther. 2008, 16, 1427-1436.
114. Mammano, F.; Salvatori, F.; Indraccolo, S.; De Rossi, A.; Chieco-Bianchi, L.; Gottlinger, H.G. Truncation of the human immunodeficiency virus type 1 envelope glycoprotein allows efficient pseudotyping of Moloney murine leukemia virus particles and gene transfer into CD4+ cells. J. Virol. 1997, 71, 3341-3345.
115. Sung, V.M.; Lai, M.M. Murine retroviral pseudotype virus containing hepatitis B virus large and small surface antigens confers specific tropism for primary human hepatocytes: a potential liver-specific targeting system. J. Virol. 2002, 76, 912-917.
116. Miletic, H.; Bruns, M.; Tsiakas, K.; Vogt, B.; Rezai, R.; Baum, C.; Kuhlke, K.; Cosset, F.L.; Ostertag, W.; Lother, H. et al. Retroviral vectors pseudotyped with lymphocytic choriomeningitis virus. J. Virol. 1999, 73, 6114-6116.
117. Yu, S.F.; von Ruden, T.; Kantoff, P.W.; Garber, C.; Seiberg, M.; Ruther, U.; Anderson, W.F.; Wagner, E.F.; Gilboa, E. Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl. Acad. Sci. U. S. A. 1986, 83, 3194-3198.
118. Hope, T. Improving the post-transcriptional aspects of lentiviral vectors. Curr. Top. Microbiol. Immunol. 2002, 261, 179-189.
119. Schon, U.; Diem, O.; Leitner, L.; Gunzburg, W.H.; Mager, D.L.; Salmons, B.; Leib-Mosch, C. Human endogenous retroviral long terminal repeat sequences as cell type-specific promoters in retroviral vectors. J. Virol. 2009, 83, 12643-12650.
120. Brown, B.D.; Venneri, M.A.; 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, 585-591.
121. Brown, B.D.; Gentner, B.; Cantore, A.; Colleoni, S.; Amendola, M.; Zingale, A.; Baccarini, A.; Lazzari, G.; Galli, C.; Naldini, L. Endogenous microRNA can be broadly exploited to regulate transgene expression according to tissue, lineage and differentiation state. Nat. Biotechnol. 2007, 25, 1457-1467.
122. Gentner, B.; Schira, G.; Giustacchini, A.; Amendola, M.; Brown, B.D.; Ponzoni, M.; Naldini, L. Stable knockdown of microRNA in vivo by lentiviral vectors. Nat. Methods 2009, 6, 63-66.
123. Gentner, B.; Visigalli, I.; Hiramatsu, H.; Lechman, E.; Ungari, S.; Giustacchini, A.; Schira, G.; Amendola, M.; Quattrini, A.; Martino, S. et al. Identification of hematopoietic stem cell-specific miRNAs enables gene therapy of globoid cell leukodystrophy. Sci. Transl. Med. 2010, 2, 58-84.
124. Schambach, A.; Bohne, J.; Baum, C.; Hermann, F.G.; Egerer, L.; von Laer, D.; Giroglou, T. Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther. 2006, 13, 641-645.
125. Graf, M.; Bojak, A.; Deml, L.; Bieler, K.; Wolf, H.; Wagner, R. Concerted action of multiple cis-acting sequences is required for Rev dependence of late human immunodeficiency virus type 1 gene expression. J. Virol. 2000, 74, 10822-10826.
126. Moreno-Carranza, B.; Gentsch, M.; Stein, S.; Schambach, A.; Santilli, G.; Rudolf, E.; Ryser, M.F.; Haria, S.; Thrasher, A.J.; Baum, C. et al. Transgene optimization significantly improves SIN vector titers, gp91(phox) expression and reconstitution of superoxide production in X-CGD cells. Gene Ther. 2009, 16, 111-118.
127. Zaiss, A.K.; Son, S.; Chang, L.J. RNA 3' readthrough of oncoretrovirus and lentivirus: implications for vector safety and efficacy. J. Virol. 2002, 76, 7209-7219.
128. Yang, Q.; Lucas, A.; Son, S.; Chang, L.J. Overlapping enhancer/promoter and transcriptional termination signals in the lentiviral long terminal repeat. Retrovirology 2007, 4, 4.
129. Schambach, A.; Galla, M.; Maetzig, T.; Loew, R.; Baum, C. Improving transcriptional termination of self-inactivating gamma-retroviral and lentiviral vectors. Mol. Ther. 2007, 15, 1167-1173.
130. Schambach, A.; Swaney, W.P.; Loo, J.C. Design and production of retro- and lentiviral vectors for gene expression in hematopoietic cells. Methods Mol. Biol. 2009, 506, 191-205.
131. Miller, A.D.; Garcia, J.V.; von Suhr, N.; Lynch, C.M.; Wilson, C.; Eiden, M.V. Construction and properties of retrovirus packaging cells based on gibbon ape leukemia virus. J. Virol. 1991, 65, 2220-2224.
132. Bode, J.; Schlake, T.; Iber, M.; Schubeler, D.; Seibler, J.; Snezhkov, E.; Nikolaev, L. The transgeneticist's toolbox: novel methods for the targeted modification of eukaryotic genomes. Biol. Chem. 2000, 381, 801-813.
133. Wirth, D.; Gama-Norton, L.; Riemer, P.; Sandhu, U.; Schucht, R.; Hauser, H. Road to precision: recombinase-based targeting technologies for genome engineering. Curr. Opin. Biotechnol. 2007, 18, 411-419.
134. Loew, R.; Meyer, Y.; Kuehlcke, K.; Gama-Norton, L.; Wirth, D.; Hauser, H.; Stein, S.; Grez, M.; Thornhill, S.; Thrasher, A. et al. A new PG13-based packaging cell line for stable production of clinical-grade self-inactivating gamma-retroviral vectors using targeted integration. Gene Ther. 2010, 17, 272-280.
135. Philpott, N.J.; Thrasher, A.J. Use of nonintegrating lentiviral vectors for gene therapy. Hum. Gene Ther. 2007, 18, 483-489.
136. 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. Effective gene therapy with nonintegrating lentiviral vectors. Nat. Med. 2006, 12, 348-353.
137. Nightingale, S.J.; Hollis, R.P.; Pepper, K.A.; Petersen, D.; Yu, X.J.; Yang, C.; Bahner, I.; Kohn, D.B. Transient gene expression by nonintegrating lentiviral vectors. Mol. Ther. 2006, 13, 1121-1132.
138. Lai, L.; Liu, H.; Wu, X.; Kappes, J.C. Moloney murine leukemia virus integrase protein augments viral DNA synthesis in infected cells. J. Virol. 2001, 75, 11365-11372.
139. Maetzig, T.; Galla, M.; Brugman, M.; Loew, R.; Baum, C.; Schambach, A. Mechanisms controlling titer and expression of bidirectional lentiviral and g-retroviral vectors. Gene Ther. 2009.
140. Galla, M.; Schambach, A.; Towers, G.J.; Baum, C. Cellular restriction of retrovirus particle-mediated mRNA transfer. J. Virol. 2008, 82, 3069-3077.
141. Galla, M.; Will, E.; Kraunus, J.; Chen, L.; Baum, C. Retroviral pseudotransduction for targeted cell manipulation. Mol. Cell 2004, 16, 309-315.
142. Galla, M.; Schambach, A.; Falk, C.; Maetzig, T.; Kuehle, J.; Lange, K.; Zychlinski, D.; Heinz, N.; Brugman, M.; Göhring, G. et al. Avoiding cytotoxicity of transposases by dose-controlled mRNA delivery. Nucleic Acids Res. 2011, in press.
143. Voelkel, C.; Galla, M.; Maetzig, T.; Warlich, E.; Kuehle, J.; Zychlinski, D.; Bode, J.; Cantz, T.; Schambach, A.; Baum, C. Protein transduction from retroviral Gag precursors. Proc. Natl. Acad. Sci. U. S. A. 2010, 107, 7805-7810.
144. Auerbach, M.R.; Brown, K.R.; Singh, I.R. Mutational analysis of the N-terminal domain of Moloney murine leukemia virus capsid protein. J. Virol. 2007, 81, 12337-12347.
145. Auerbach, M.R.; Shu, C.; Kaplan, A.; Singh, I.R. Functional characterization of a portion of the Moloney murine leukemia virus gag gene by genetic footprinting. Proc. Natl. Acad. Sci. U. S. A. 2003, 100, 11678-11683.
146. Muller, B.; Daecke, J.; Fackler, O.T.; Dittmar, M.T.; Zentgraf, H.; Krausslich, H.G. Construction and characterization of a fluorescently labeled infectious human immunodeficiency virus type 1 derivative. J. Virol. 2004, 78, 10803-10813.
147. Metzner, C.; Salmons, B.; Gunzburg, W.H.; Dangerfield, J.A. Rafts, anchors and viruses-A role for glycosylphosphatidylinositol anchored proteins in the modification of enveloped viruses and viral vectors. Virology 2008, 382, 125-131.
148. Kueng, H.J.; Leb, V.M.; Haiderer, D.; Raposo, G.; Thery, C.; Derdak, S.V.; Schmetterer, K.G.; Neunkirchner, A.; Sillaber, C.; Seed, B. et al. General strategy for decoration of enveloped viruses with functionally active lipid-modified cytokines. J. Virol. 2007, 81, 8666-8676.
149. Kueng, H.J.; Manta, C.; Haiderer, D.; Leb, V.M.; Schmetterer, K.G.; Neunkirchner, A.; Byrne, R.A.; Scheinecker, C.; Steinberger, P.; Seed, B. et al. Fluorosomes: a convenient new reagent to detect and block multivalent and complex receptor-ligand interactions. FASEB J. 2010, 24, 1572-1582.
150. Verhoeyen, E.; Wiznerowicz, M.; Olivier, D.; Izac, B.; Trono, D.; Dubart-Kupperschmitt, A.; Cosset, F.L. Novel lentiviral vectors displaying early-acting cytokines selectively promote survival and transduction of NOD/SCID repopulating human hematopoietic stem cells. Blood 2005, 106, 3386-3395.
151. Frecha, C.; Costa, C.; Negre, D.; Gauthier, E.; Russell, S.J.; Cosset, F.L.; Verhoeyen, E. Stable transduction of quiescent T-cells without induction of cycle progression by a novel lentiviral vector pseudotyped with measles virus glycoproteins. Blood 2008, 112, 4843-4852.
152. Gene Therapy Clinical Trials Worldwide. Available online: http://www.wiley.com/legacy/wileychi/genmed/clinical/ (accessed on 9 May 2011).
153. Kondo, M.; Wagers, A.J.; Manz, M.G.; Prohaska, S.S.; Scherer, D.C.; Beilhack, G.F.; Shizuru, J.A.; Weissman, I.L. Biology of hematopoietic stem cells and progenitors: implications for clinical application. Annu. Rev. Immunol. 2003, 21, 759-806.
154. Wilson, A.; Laurenti, E.; Oser, G.; van der Wath, R.C.; Blanco-Bose, W.; Jaworski, M.; Offner, S.; Dunant, C.F.; Eshkind, L.; Bockamp, E. et al. Hematopoietic stem cells reversibly switch from dormancy to self-renewal during homeostasis and repair. Cell 2008, 135, 1118-1129.
155. Essers, M.A.; Offner, S.; Blanco-Bose, W.E.; Waibler, Z.; Kalinke, U.; Duchosal, M.A.; Trumpp, A. IFNalpha activates dormant haematopoietic stem cells in vivo. Nature 2009, 458, 904-908.
156. Orford, K.W.; Scadden, D.T. Deconstructing stem cell self-renewal: genetic insights into cell-cycle regulation. Nat. Rev. Genet. 2008, 9, 115-128.
157. Park, Y.; Gerson, S.L. DNA repair defects in stem cell function and aging. Annu. Rev. Med. 2005, 56, 495-508.
158. Verfaillie, C.M. Hematopoietic stem cells for transplantation. Nat. Immunol. 2002, 3, 314-317.
159. Hacein-Bey-Abina, S.; Le Deist, F.; Carlier, F.; Bouneaud, C.; Hue, C.; De Villartay, J.P.; Thrasher, A.J.; Wulffraat, N.; Sorensen, R.; Dupuis-Girod, S. et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. New Engl. J. Med. 2002, 346, 1185-1193.
160. Gaspar, H.B.; Bjorkegren, E.; Parsley, K.; Gilmour, K.C.; King, D.; Sinclair, J.; Zhang, F.; Giannakopoulos, A.; Adams, S.; Fairbanks, L.D. et al. Successful reconstitution of immunity in ADA-SCID by stem cell gene therapy following cessation of PEG-ADA and use of mild preconditioning. Mol. Ther. 2006, 14, 505-513.
161. Ott, M.G.; Schmidt, M.; Schwarzwaelder, K.; Stein, S.; Siler, U.; Koehl, U.; Glimm, H.; Kuhlcke, K.; Schilz, A.; Kunkel, H. et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat. Med. 2006, 12, 401-409.
162. Boztug, K.; Schmidt, M.; Schwarzer, A.; Banerjee, P.P.; Diez, I.A.; Dewey, R.A.; Bohm, M.; Nowrouzi, A.; Ball, C.R.; Glimm, H. et al. Stem-cell gene therapy for the Wiskott-Aldrich syndrome. New Engl. J. Med. 2010, 363, 1918-1927.
163. Zhang, C.C.; Kaba, M.; Ge, G.; Xie, K.; Tong, W.; Hug, C.; Lodish, H.F. Angiopoietin-like proteins stimulate ex vivo expansion of hematopoietic stem cells. Nat. Med. 2006, 12, 240-245.
164. Zhang, C.C.; Kaba, M.; Iizuka, S.; Huynh, H.; Lodish, H.F. Angiopoietin-like 5 and IGFBP2 stimulate ex vivo expansion of human cord blood hematopoietic stem cells as assayed by NOD/SCID transplantation. Blood 2008, 111, 3415-3423.
165. Zhang, C.C.; Lodish, H.F. Murine hematopoietic stem cells change their surface phenotype during ex vivo expansion. Blood 2005, 105, 4314-4320.
166. Boitano, A.E.; Wang, J.; Romeo, R.; Bouchez, L.C.; Parker, A.E.; Sutton, S.E.; Walker, J.R.; Flaveny, C.A.; Perdew, G.H.; Denison, M.S. et al. Aryl hydrocarbon receptor antagonists promote the expansion of human hematopoietic stem cells. Science 2010, 329, 1345-1348.
167. Deakin, C.T.; Alexander, I.E.; Kerridge, I. The ethics of gene therapy: Balancing the risks. Curr. Opin. Mol. Ther. 2010, 12, 578-585.
168. Aiuti, A.; Roncarolo, M.G. Ten years of gene therapy for primary immune deficiencies. Hematology Am. Soc. Hematol. Educ. Program 2009, 682-689.
169. Blaese, R.M.; Culver, K.W.; Miller, A.D.; Carter, C.S.; Fleisher, T.; Clerici, M.; Shearer, G.; Chang, L.; Chiang, Y.; Tolstoshev, P. et al. T lymphocyte-directed gene therapy for ADA- SCID: initial trial results after 4 years. Science 1995, 270, 475-480.
170. Bordignon, C.; Notarangelo, L.D.; Nobili, N.; Ferrari, G.; Casorati, G.; Panina, P.; Mazzolari, E.; Maggioni, D.; Rossi, C.; Servida, P. et al. Gene therapy in peripheral blood lymphocytes and bone marrow for ADA- immunodeficient patients. Science 1995, 270, 470-475.
171. Aiuti, A.; Slavin, S.; Aker, M.; Ficara, F.; Deola, S.; Mortellaro, A.; Morecki, S.; Andolfi, G.; Tabucchi, A.; Carlucci, F. et al. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science 2002, 296, 2410-2413.
172. Aiuti, A.; Cattaneo, F.; Galimberti, S.; Benninghoff, U.; Cassani, B.; Callegaro, L.; Scaramuzza, S.; Andolfi, G.; Mirolo, M.; Brigida, I. et al. Gene therapy for immunodeficiency due to adenosine deaminase deficiency. New Engl. J. Med. 2009, 360, 447-458.
173. Thrasher, A.J.; Hacein-Bey-Abina, S.; Gaspar, H.B.; Blanche, S.; Davies, E.G.; Parsley, K.; Gilmour, K.; King, D.; Howe, S.; Sinclair, J. et al. Failure of SCID-X1 gene therapy in older patients. Blood 2005, 105, 4255-4257.
174. Hacein-Bey-Abina, S.; Garrigue, A.; Wang, G.P.; Soulier, J.; Lim, A.; Morillon, E.; Clappier, E.; Caccavelli, L.; Delabesse, E.; Beldjord, K. et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J. Clin. Invest. 2008, 118, 3132-3142.
175. Grez, M.; Reichenbach, J.; Schwable, J.; Seger, R.; Dinauer, M.C.; Thrasher, A.J. Gene therapy of chronic granulomatous disease: the engraftment dilemma. Mol. Ther. 2010, 19, 28-35.
176. Porter, C.C. The Promise of Gene Therapy That WAS is Now. Online Commentary. Available online: http://www.hematology.org/Publications/ASH-News-Daily/2010/6199.aspx (accessed on 9 May 2011).
177. 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. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009, 326, 818-823.
178. De Luca, M.; Pellegrini, G.; Mavilio, F. Gene therapy of inherited skin adhesion disorders: A critical overview. Br. J. Dermatol. 2009, 161, 19-24.
179. Jolly, D.; Perez, O.; Burnett, R.; Gunzburg, W.; Ibanez, C.; Chen, C.; Pettersson, P.; Lin, A.; Gruber, H.; Pertschuk, D. et al. Replicating Retrovirus Encoding a Prodrug Activator (Toca 511) Enters Clinic for the Treatment of Glioblastoma Multiforme (GBM). Hum. Gene Ther. 2010, 21, 1375.
180. 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. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004, 364, 2181-2187.
181. Aiuti, A.; Cassani, B.; Andolfi, G.; Mirolo, M.; Biasco, L.; Recchia, A.; Urbinati, F.; Valacca, C.; Scaramuzza, S.; Aker, M. et al. Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 2007, 117, 2233-2240.
182. Cavazzana-Calvo, M.; Payen, E.; Negre, O.; Wang, G.; Hehir, K.; Fusil, F.; Down, J.; Denaro, M.; Brady, T.; Westerman, K. et al. Transfusion independence and HMGA2 activation after gene therapy of human beta-thalassaemia. Nature 2010, 467, 318-322.
183. Dudley, M.E.; Wunderlich, J.R.; Robbins, P.F.; Yang, J.C.; Hwu, P.; Schwartzentruber, D.J.; Topalian, S.L.; Sherry, R.; Restifo, N.P.; Hubicki, A.M. et al. Cancer regression and autoimmunity in patients after clonal repopulation with antitumor lymphocytes. Science 2002, 298, 850-854.
184. Morgan, R.A.; Dudley, M.E.; Wunderlich, J.R.; Hughes, M.S.; Yang, J.C.; Sherry, R.M.; Royal, R.E.; Topalian, S.L.; Kammula, U.S.; Restifo, N.P. et al. Cancer regression in patients after transfer of genetically engineered lymphocytes. Science 2006, 314, 126-129.
185. Ciceri, F.; Bonini, C.; Stanghellini, M.T.; Bondanza, A.; Traversari, C.; Salomoni, M.; Turchetto, L.; Colombi, S.; Bernardi, M.; Peccatori, J. et al. Infusion of suicide-gene-engineered donor lymphocytes after family haploidentical haemopoietic stem-cell transplantation for leukaemia (the TK007 trial): a non-randomised phase I-II study. Lancet Oncol. 2009, 10, 489-500.
186. 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. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc. Natl. Acad. Sci. U. S. A. 2006, 103, 17372-17377.
187. Knudson, A.G. Two genetic hits (more or less) to cancer. Nat. Rev. Cancer 2001, 1, 157-162.
188. Baum, C.; Dullmann, J.; Li, Z.; Fehse, B.; Meyer, J.; Williams, D.A.; von-Kalle, C. Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 2003, 101, 2099-2114.
189. Fehse, B.; Kustikova, O.S.; Bubenheim, M.; Baum, C. Pois(s)on-It's a question of dose?! Gene Ther. 2004, 11, 879-881.
190. Kustikova, O.S.; Fehse, B.; Modlich, U.; Yang, M.; Düllmann, J.; Kamino, K.; von Neuhoff, N.; Schlegelberger, B.; Li, Z.; Baum, C. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 2005, 308, 1171-1174.
191. Schmidt, M.; Carbonaro, D.A.; Speckmann, C.; Wissler, M.; Bohnsack, J.; Elder, M.; Aronow, B.J.; Nolta, J.A.; Kohn, D.B.; von Kalle, C. Clonality analysis after retroviral-mediated gene transfer to CD34+ cells from the cord blood of ADA-deficient SCID neonates. Nat. Med. 2003, 9, 463-468.
192. Bushman, F.; Lewinski, M.; Ciuffi, A.; Barr, S.; Leipzig, J.; Hannenhalli, S.; Hoffmann, C. Genome-wide analysis of retroviral DNA integration. Nat. Rev. Microbiol. 2005, 3, 848-858.
193. Derse, D.; Crise, B.; Li, Y.; Princler, G.; Lum, N.; Stewart, C.; McGrath, C.F.; Hughes, S.H.; Munroe, D.J.; Wu, X. Human T-cell leukemia virus type 1 integration target sites in the human genome: comparison with those of other retroviruses. J. Virol. 2007, 81, 6731-6741.
194. Lewinski, M.K.; Yamashita, M.; Emerman, M.; Ciuffi, A.; Marshall, H.; Crawford, G.; Collins, F.; Shinn, P.; Leipzig, J.; Hannenhalli, S. et al. Retroviral DNA integration: viral and cellular determinants of target-site selection. PLoS Pathog. 2006, 2, e60.
195. Mitchell, R.S.; Beitzel, B.F.; Schroder, A.R.; Shinn, P.; Chen, H.; Berry, C.C.; Ecker, J.R.; Bushman, F.D. Retroviral DNA Integration: ASLV, HIV, and MLV Show Distinct Target Site Preferences. PLoS Biol. 2004, 2, E234.
196. Schroder, A.R.; Shinn, P.; Chen, H.; Berry, C.; Ecker, J.R.; Bushman, F. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 2002, 110, 521-529.
197. Wu, X.; Li, Y.; Crise, B.; Burgess, S.M. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003, 300, 1749-1751.
198. Uren, A.G.; Kool, J.; Berns, A.; van Lohuizen, M. Retroviral insertional mutagenesis: past, present and future. Oncogene 2005, 24, 7656-7672.
199. West, A.G.; Fraser, P. Remote control of gene transcription. Hum. Mol. Genet. 2005, 14, R101-R111.
200. Wotton, S.; Stewart, M.; Blyth, K.; Vaillant, F.; Kilbey, A.; Neil, J.C.; Cameron, E.R. Proviral insertion indicates a dominant oncogenic role for Runx1/AML-1 in T-cell lymphoma. Cancer Res. 2002, 62, 7181-7185.
201. Martin-Hernandez, J.; Sorensen, A.B.; Pedersen, F.S. Murine leukemia virus proviral insertions between the N-ras and unr genes in B-cell lymphoma DNA affect the expression of N-ras only. J. Virol. 2001, 75, 11907-11912.
202. Modlich, U.; Bohne, J.; Schmidt, M.; von Kalle, C.; Knoss, S.; Schambach, A.; Baum, C. Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 2006, 108, 2545-2553.
203. Zychlinski, D.; Schambach, A.; Modlich, U.; Maetzig, T.; Meyer, J.; Grassman, E.; Mishra, A.; Baum, C. Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol. Ther. 2008, 16, 718-725.
204. Montini, E.; Cesana, D.; Schmidt, M.; Sanvito, F.; Bartholomae, C.C.; Ranzani, M.; Benedicenti, F.; Sergi, L.S.; Ambrosi, A.; Ponzoni, M. et al. 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. 2009, 119, 964-975.
205. Maetzig, T.; Brugman, M.H.; Bartels, S.; Heinz, N.; Kustikova, O.S.; Modlich, U.; Li, Z.; Galla, M.; Schiedlmeier, B.; Schambach, A. et al. Polyclonal fluctuation of lentiviral vector-transduced and expanded murine hematopoietic stem cells. Blood 2011, 117, 3053-3064.
206. Bokhoven, M.; Stephen, S.L.; Knight, S.; Gevers, E.F.; Robinson, I.C.; Takeuchi, Y.; Collins, M.K. Insertional gene activation by lentiviral and gammaretroviral vectors. J. Virol. 2009, 83, 283-294.
207. Thornhill, S.I.; Schambach, A.; Howe, S.J.; Ulaganathan, M.; Grassman, E.; Williams, D.; Schiedlmeier, B.; Sebire, N.J.; Gaspar, H.B.; Kinnon, C. et al. Self-inactivating gammaretroviral vectors for gene therapy of X-linked severe combined immunodeficiency. Mol. Ther. 2008, 16, 590-598.
208. Arumugam, P.I.; Urbinati, F.; Velu, C.S.; Higashimoto, T.; Grimes, H.L.; Malik, P. The 3' region of the chicken hypersensitive site-4 insulator has properties similar to its core and is required for full insulator activity. PLoS ONE 2009, 4, e6995.
209. Durand, S.; Cimarelli, A. The Inside Out of Lentiviral Vectors. Viruses 2011, 3, 132-159.
210. Lindemann, D.; Rethwilm, A. Foamy virus biology and its application for vector development. Viruses 2011, 3, 561-585.