Hybrid lentiviral vectors

Molecular Therapy

Volumn 18, Issue 7, 2010, Pages 1263-1267

  Qasim, Waseem ^a   Vink, Conrad A ^a   Thrasher, Adrian J ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

CHEMOKINE RECEPTOR CCR5; INTERLEUKIN 2 RECEPTOR GAMMA; LENTIVIRUS VECTOR; NUCLEASE; PARVOVIRUS VECTOR; TRANSPOSASE; ZINC FINGER PROTEIN;

CELL DIVISION; CELL LINEAGE; CELL POPULATION; DNA CLEAVAGE; DNA MODIFICATION; DNA REPAIR; GENE EXPRESSION; GENE FUNCTION; GENE INSERTION; GENE SEQUENCE; GENE SILENCING; GENE TARGETING; GENETIC RECOMBINATION; GENETIC TRANSDUCTION; HUMAN; HUMAN IMMUNODEFICIENCY VIRUS 1; MITOSIS; MUTAGENESIS; NONHUMAN; REVIEW; SUICIDE GENE; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM;

GENETIC VECTORS; HIV-1; LENTIVIRUS; MODELS, GENETIC;

HARNESS; HUMAN IMMUNODEFICIENCY VIRUS 1;

EID: 77954242391     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.76     Document Type: Review

References (50)

1. Maertens, G, Cherepanov, P, Pluymers, W, Busschots, K, De Clercq, E, Debyser, Z et al. (2003). LEDGF/p75 is essential for nuclear and chromosomal targeting of HIV-1 integrase in human cells. J Biol Chem 278: 33528-33539. (Pubitemid 37055807)
2. Llano, M, Saenz, DT, Meehan, A, Wongthida, P, Peretz, M, Walker, WH et al. (2006). An essential role for LEDGF/p75 in HIV integration. Science 314: 461-464.
3. Marshall, HM, Ronen, K, Berry, C, Llano, M, Sutherland, H, Saenz, D et al. (2007). Role of PSIP1/LEDGF/p75 in lentiviral infectivity and integration targeting. PLoS ONE 2: e1340.
4. Rohdewohld, H, Weiher, H, Reik, W, Jaenisch, R and Breindl, M (1987). Retrovirus integration and chromatin structure: Moloney murine leukemia proviral integration sites map near DNase I-hypersensitive sites. J Virol 61: 336-343.
5. Vijaya, S, Steffen, DL and Robinson, HL (1986). Acceptor sites for retroviral integrations map near DNase I-hypersensitive sites in chromatin. J Virol 60: 683-692.
6. Bushman, F (2002). Targeting retroviral integration? Mol Ther 6: 570-571.
7. Schröder, AR, Shinn, P, Chen, H, Berry, C, Ecker, JR and Bushman, F (2002). HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110: 521-529.
8. Lewinski, MK, Yamashita, M, Emerman, M, Ciuff, A, Marshall, H, Crawford, G et al. (2006). Retroviral DNA integration: viral and cellular determinants of target-site selection. PLoS Pathog 2: e60.
9. Hacein-Bey-Abina, S, Von Kalle, C, Schmidt, M, McCormack, MP, Wulffraat, N, Leboulch, P et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419.
10. Hacein-Bey-Abina, S, Garrigue, A, Wang, G P, Soulier, J, Lim, A, Morillon, E et al. (2008). Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 118: 3132-3142.
11. Howe, SJ, Mansour, MR, Schwarzwaelder, K, Bartholomae, C, Hubank, M, Kempski, H et al. (2008). Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 118: 3143-3150.
12. Ott, MG, Schmidt, M, Schwarzwaelder, K, Stein, S, Siler, U, Koehl, U et al. (2006). Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 12: 401-409.
13. Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Ponzoni, M, Bartholomae, 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.
14. Modlich, U, Navarro, S, Zychlinski, D, Maetzig, T, Knoess, S, Brugman, MH et al. (2009). Insertional transformation of hematopoietic cells by self-inactivating lentiviral and g-retroviral vectors. Mol Ther 17: 1919-1928.
15. Levine, BL, Humeau, LM, Boyer, J, MacGregor, RR, Rebello, T, Lu, X et al. (2006). Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA 103: 17372-17377.
16. Wang, GP, Levine, BL, Binder, GK, Berry, CC, Malani, N, McGarrity, G et al. (2009). Analysis of lentiviral vector integration in HIV+ study subjects receiving autologous infusions of gene modifed CD4+ T cells. Mol Ther 17: 844-850.
17. Cartier, N, Hacein-Bey-Abina, S, Bartholomae, CC, Veres, G, Schmidt, M, Kutschera, I et al. (2009). Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 326: 818-823.
18. Williams, DA (2009). Gene therapy continues to mature and to face challenges. Mol Ther 17: 1305-1306.
19. Wanisch, K and Yáñez-Muñoz, RJ (2009). Integration-defcient lentiviral vectors: a slow coming of age. Mol Ther 17: 1316-1332.
20. Banasik, MB and McCray, PB Jr (2010). Integrase-defective lentiviral vectors: progress and applications. Gene Ther 17: 150-157.
21. Yáñez-Muñoz, RJ, Balaggan, KS, MacNeil, A, Howe, SJ, Schmidt, M, Smith, AJ et al. (2006). Effective gene therapy with nonintegrating lentiviral vectors. Nat Med 12: 348-353.
22. Nightingale, SJ, Hollis, R P, Pepper, KA, Petersen, D, Yu, XJ, Yang, C et al. (2006). Transient gene expression by nonintegrating lentiviral vectors. Mol Ther 13: 1121-1132.
23. Kantor, B, Ma, H, Webster-Cyriaque, J, Monahan, PE and Kafri, T (2009). Epigenetic activation of unintegrated HIV-1 genomes by gut-associated short chain fatty acids and its implications for HIV infection. Proc Natl Acad Sci USA 106: 18786-18791.
24. Nakajima, N, Lu, R and Engelman, A (2001). Human immunodefciency virus type 1 replication in the absence of integrase-mediated DNA recombination: defnition of permissive and nonpermissive T-cell lines. J Virol 75: 7944-7955.
25. Moldt, B, Staunstrup, NH, Jakobsen, M, Yáñez-Muñoz, RJ and Mikkelsen, JG (2008). Genomic insertion of lentiviral DNA circles directed by the yeast Flp recombinase. BMC Biotechnol 8: 60.
26. Lombardo, A, Genovese, P, Beausejour, CM, Colleoni, S, Lee, YL, Kim, KA et al. (2007). Gene editing in human stem cells using zinc fnger nucleases and integrase-defective lentiviral vector delivery. Nat Biotechnol 25: 1298-1306.
27. Cornu, TI and Cathomen, T (2007). Targeted genome modifcations using integrase-defcient lentiviral vectors. Mol Ther 15: 2107-2113.
28. Bayer, M, Kantor, B, Cockrell, A, Ma, H, Zeithaml, B, Li, X et al. (2008). A large U3 deletion causes increased in vivo expression from a nonintegrating lentiviral vector. Mol Ther 16: 1968-1976.
29. Redondo, P, Prieto, J, Muñoz, IG, Alibés, A, Stricher, F, Serrano, L et al. (2008). Molecular basis of xeroderma pigmentosum group C DNA recognition by engineered meganucleases. Nature 456: 107-111.
30. Urnov, FD, Miller, JC, Lee, YL, Beausejour, CM, Rock, JM, Augustus, S et al. (2005). Highly effcient endogenous human gene correction using designed zinc-fnger nucleases. Nature 435: 646-651.
31. Perez, EE, Wang, J, Miller, JC, Jouvenot, Y, Kim, KA, Liu, O et al. (2008). Establishment of HIV-1 resistance in CD4+ T cells by genome editing using zinc-fnger nucleases. Nat Biotechnol 26: 808-816.
32. Lombardo A, Cesana D, Genovese P, Provasi E, Maruggi G, Binini C et al. (2009). Characterization of potential genomic 'safe harbor' for effcient targeted gene addition with zinc fnger nucleases. Mol Ther 17: S168.
33. Cornu, TI, Thibodeau-Beganny, S, Guhl, E, Alwin, S, Eichtinger, M, Joung, JK et al. (2008). DNA-binding specifcity is a major determinant of the activity and toxicity of zinc-fnger nucleases. Mol Ther 16: 352-358.
34. Szczepek, M, Brondani, V, Büchel, J, Serrano, L, Segal, DJ and Cathomen, T (2007). Structure-based redesign of the dimerization interface reduces the toxicity of zinc-fnger nucleases. Nat Biotechnol 25: 786-793.
35. Miller, JC, Holmes, MC, Wang, J, Guschin, DY, Lee, YL, Rupniewski, I et al. (2007). An improved zinc-fnger nuclease architecture for highly specifc genome editing. Nat Biotechnol 25: 778-785.
36. Ivics, Z, Hackett, PB, Plasterk, RH and Izsvák, Z (1997). Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fsh, and its transposition in human cells. Cell 91: 501-510.
37. Izsvák, Z, Khare, D, Behlke, J, Heinemann, U, Plasterk, RH and Ivics, Z (2002). Involvement of a bifunctional, paired-like DNA-binding domain and a transpositional enhancer in Sleeping Beauty transposition. J Biol Chem 277: 34581-34588.
38. Yant, SR and Kay, MA (2003). Nonhomologous-end-joining factors regulate DNA repair fdelity during Sleeping Beauty element transposition in mammalian cells. Mol Cell Biol 23: 8505-8518.
39. Izsvák, Z, Stüwe, EE, Fiedler, D, Katzer, A, Jeggo, PA and Ivics, Z (2004). Healing the wounds inficted by sleeping beauty transposition by double-strand break repair in mammalian somatic cells. Mol Cell 13: 279-290.
40. Yant, SR, Wu, X, Huang, Y, Garrison, B, Burgess, SM and Kay, MA (2005). High-resolution genome-wide mapping of transposon integration in mammals. Mol Cell Biol 25: 2085-2094.
41. Berry, C, Hannenhalli, S, Leipzig, J and Bushman, FD (2006). Selection of target sites for mobile DNA integration in the human genome. PLoS Comput Biol 2: e157.
42. Vink, CA, Gaspar, HB, Gabriel, R, Schmidt, M, McIvor, RS, Thrasher, AJ et al. (2009). Sleeping beauty transposition from nonintegrating lentivirus. Mol Ther 17: 1197-1204.
43. Staunstrup, NH, Moldt, B, Mátés, L, Villesen, P, Jakobsen, M, Ivics, Z et al. (2009). Hybrid lentivirus-transposon vectors with a random integration profle in human cells. Mol Ther 17: 1205-1214.
44. Garrison, BS, Yant, SR, Mikkelsen, JG and Kay, MA (2007). Postintegrative gene silencing within the Sleeping Beauty transposition system. Mol Cell Biol 27: 8824-8833.
45. Dalsgaard, T, Moldt, B, Sharma, N, Wolf, G, Schmitz, A, Pedersen, FS et al. (2009). Shielding of sleeping beauty DNA transposon-delivered transgene cassettes by heterologous insulators in early embryonal cells. Mol Ther 17: 121-130.
46. Ivics, Z, Katzer, A, Stüwe, EE, Fiedler, D, Knespel, S and Izsvák, Z (2007). Targeted Sleeping Beauty transposition in human cells. Mol Ther 15: 1137-1144.
47. Ding, S, Wu, X, Li, G, Han, M, Zhuang, Y and Xu, T (2005). Effcient transposition of the piggyBac (PB) transposon in mammalian cells and mice. Cell 122: 473-483.
48. Liang, Q, Kong, J, Stalker, J and Bradley, A (2009). Chromosomal mobilization and reintegration of Sleeping Beauty and PiggyBac transposons. Genesis 47: 404-408.
49. Apolonia, L, Waddington, SN, Fernandes, C, Ward, NJ, Bouma, G, Blundell, MP et al. (2007). Stable gene transfer to muscle using non-integrating lentiviral vectors. Mol Ther 15: 1947-1954.
50. Philippe, S, Sarkis, C, Barkats, M, Mammeri, H, Ladroue, C, Petit, C et al. (2006). Lentiviral vectors with a defective integrase allow effcient and sustained transgene expression in vitro and in vivo. Proc Natl Acad Sci USA 103: 17684-17689.