Site-specific gene insertion mediated by a Cre-loxP-carrying lentiviral vector

Molecular Therapy

Volumn 18, Issue 10, 2010, Pages 1814-1821

  Michel, Gilles ^a   Yu, Yin ^a   Chang, Tammy ^a   Yee, Jiing Kuan ^a  

^a CITY OF HOPE NATIONAL MEDICAL CENTER   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

CRE RECOMBINASE; LENTIVIRUS VECTOR;

ARTICLE; CONTROLLED STUDY; ENZYME ACTIVATION; GENE INSERTION; GENE SEQUENCE; HUMAN; HUMAN CELL; LOXP SITE; NONHUMAN; SITE DIRECTED MUTAGENESIS; VIRAL GENE DELIVERY SYSTEM;

EID: 77957608958     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2010.150     Document Type: Article

References (44)

1. Hacein-Bey-Abina, S, von Kalle, C, Schmidt, M, Le Deist, F, Wulffraat, N, McIntyre, E et al. (2003). A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 348: 255-256.
2. 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.
3. Stein, S, Ott, MG, Schultze-Strasser, S, Jauch, A, Burwinkel, B, Kinner, A et al. (2010). Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 16: 198-204.
4. Wu, X, Li, Y, Crise, B and Burgess, SM (2003). Transcription start regions in the human genome are favored targets for MLV integration. Science 300: 1749-1751.
5. Akagi, K, Suzuki, T, Stephens, RM, Jenkins, NA and Copeland, NG (2004). RTCGD: retroviral tagged cancer gene database. Nucleic Acids Res 32(Database issue): D523-D527.
6. Schroder, 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.
7. Montini, E, Cesana, D, Schmidt, M, Sanvito, F, Bartholomae, CC, Ranzani, 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.
8. Kaiser, J (2009). Gene therapy. β-thalassemia treatment succeeds, with a caveat. Science 326: 1468-1469.
9. Inoue, N, Izui-Sarumaru, T, Murakami, Y, Endo, Y, Nishimura, J, Kurokawa, K et al. (2006). Molecular basis of clonal expansion of hematopoiesis in 2 patients with paroxysmal nocturnal hemoglobinuria (PNH). Blood 108: 4232-4236.
10. Hess, JL and Kossev, P (2002). Molecular genetics of benign tumors. Cancer Invest 20: 362-372.
11. Fedele, M, Battista, S, Manfioletti, G, Croce, CM, Giancotti, V and Fusco, A (2001). Role of the high mobility group A proteins in human lipomas. Carcinogenesis 22: 1583-1591.
12. Odero, MD, Grand, FH, Iqbal, S, Ross, F, Roman, JP, Vizmanos, JL et al. (2005). Disruption and aberrant expression of HMGA2 as a consequence of diverse chromosomal translocations in myeloid malignancies. Leukemia 19: 245-252.
13. Katz, RA, Merkel, G and Skalka, AM (1996). Targeting of retroviral integrase by fusion to a heterologous DNA binding domain: in vitro activities and incorporation of a fusion protein into viral particles. Virology 217: 178-190.
14. Holmes-Son, ML, Appa, RS and Chow, SA (2001). Molecular genetics and target site specificity of retroviral integration. Adv Genet 43: 33-69.
15. Holmes-Son, ML and Chow, SA (2000). Integrase-lexA fusion proteins incorporated into human immunodeficiency virus type 1 that contains a catalytically inactive integrase gene are functional to mediate integration. J Virol 74: 11548-11556.
16. Tan, W, Zhu, K, Segal, DJ, Barbas, CF 3rd and Chow, SA (2004). Fusion proteins consisting of human immunodeficiency virus type 1 integrase and the designed polydactyl zinc finger protein E2C direct integration of viral DNA into specific sites. J Virol 78: 1301-1313.
17. Sauer, B and Henderson, N (1988). Site-specific DNA recombination in mammalian cells by the Cre recombinase of bacteriophage P1. Proc Natl Acad Sci USA 85: 5166-5170.
18. Sauer, B and Henderson, N (1990). Targeted insertion of exogenous DNA into the eukaryotic genome by the Cre recombinase. New Biol 2: 441-449.
19. Thyagarajan, B, Guimaraes, MJ, Groth, AC and Calos, MP (2000). Mammalian genomes contain active recombinase recognition sites. Gene 244: 47-54.
20. Popov, S, Rexach, M, Zybarth, G, Reiling, N, Lee, MA, Ratner, L et al. (1998). Viral protein R regulates nuclear import of the HIV-1 pre-integration complex. EMBO J 17: 909-917.
21. Vodicka, MA, Koepp, DM, Silver, PA and Emerman, M (1998). HIV-1 Vpr interacts with the nuclear transport pathway to promote macrophage infection. Genes Dev 12: 175-185.
22. Gasmi, M, Glynn, J, Jin, MJ, Jolly, DJ, Yee, JK and Chen, ST (1999). Requirements for efficient production and transduction of human immunodeficiency virus type 1-based vectors. J Virol 73: 1828-1834. (Pubitemid 29098082)
23. Yam, PY, Li, S, Wu, J, Hu, J, Zaia, JA and Yee, JK (2002). Design of HIV vectors for efficient gene delivery into human hematopoietic cells. Mol Ther 5: 479-484.
24. Mochizuki, H, Schwartz, JP, Tanaka, K, Brady, RO 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.
25. Pfeifer, A, Brandon, EP, Kootstra, N, Gage, FH and Verma, IM (2001). Delivery of the Cre recombinase by a self-deleting lentiviral vector: efficient gene targeting in vivo. Proc Natl Acad Sci USA 98: 11450-11455.
26. Silver, DP and Livingston, DM (2001). Self-excising retroviral vectors encoding the Cre recombinase overcome Cre-mediated cellular toxicity. Mol Cell 8: 233-243.
27. Nolden, L, Edenhofer, F, Haupt, S, Koch, P, Wunderlich, FT, Siemen, H et al. (2006). Site-specific recombination in human embryonic stem cells induced by cell-permeant Cre recombinase. Nat Methods 3: 461-467.
28. BouHamdan, M, Kulkosky, J, Duan, LX and Pomerantz, RJ (2000). Inhibition of HIV-1 replication and infectivity by expression of a fusion protein, VPR-anti-integrase singlechain variable fragment (SFv): intravirion molecular therapies. J Hum Virol 3: 6-15.
29. Yao, XJ, Kobinger, G, Dandache, S, Rougeau, N and Cohen, E (1999). HIV-1 Vprchloramphenicol acetyltransferase fusion proteins: sequence requirement for virion incorporation and analysis of antiviral effect. Gene Ther 6: 1590-1599.
30. Cavrois, M, De Noronha, C and Greene, WC (2002). A sensitive and specific enzymebased assay detecting HIV-1 virion fusion in primary T lymphocytes. Nat Biotechnol 20: 1151-1154.
31. Muller, B, Tessmer, U, Schubert, U and Krausslich, HG (2000). Human immunodeficiency virus type 1 Vpr protein is incorporated into the virion in significantly smaller amounts than gag and is phosphorylated in infected cells. J Virol 74: 9727-9731.
32. Yao, XJ, Subbramanian, RA, Rougeau, N, Boisvert, F, Bergeron, D and Cohen, EA (1995). Mutagenic analysis of human immunodeficiency virus type 1 Vpr: role of a predicted N-terminal α-helical structure in Vpr nuclear localization and virion incorporation. J Virol 69: 7032-7044.
33. Nunes-Duby, SE, Kwon, HJ, Tirumalai, RS, Ellenberger, T and Landy, A (1998). Similarities and differences among 105 members of the Int family of site-specific recombinases. Nucleic Acids Res 26: 391-406.
34. Esposito, D and Scocca, JJ (1997). The integrase family of tyrosine recombinases: evolution of a conserved active site domain. Nucleic Acids Res 25: 3605-3614.
35. Zhao, HL, Yao, XQ, Xue, C, Wang, Y, Xiong, XH and Liu, ZM (2008). Increasing the homogeneity, stability and activity of human serum albumin and interferon-α2b fusion protein by linker engineering. Protein Expr Purif 61: 73-77.
36. Cao, Y, Marks, JD, Marks, JW, Cheung, LH, Kim, S and Rosenblum, MG (2009). Construction and characterization of novel, recombinant immunotoxins targeting the Her2/neu oncogene product: in vitro and in vivo studies. Cancer Res 69: 8987-8995.
37. Buchholz, F and Stewart, AF (2001). Alteration of Cre recombinase site specificity by substrate-linked protein evolution. Nat Biotechnol 19: 1047-1052.
38. Thomson, JG, Rucker, EB 3rd and Piedrahita, JA (2003). Mutational analysis of loxP sites for efficient Cre-mediated insertion into genomic DNA. Genesis 36: 162-167.
39. Araki, K, Araki, M and Yamamura, K (2002). Site-directed integration of the cre gene mediated by Cre recombinase using a combination of mutant lox sites. Nucleic Acids Res 30: e103.
40. Thorpe, HM and Smith, MC (1998). In vitro site-specific integration of bacteriophage DNA catalyzed by a recombinase of the resolvase/invertase family. Proc Natl Acad Sci USA 95: 5505-5510.
41. Thorpe, HM, Wilson, SE and Smith, MC (2000). Control of directionality in the sitespecific recombination system of the Streptomyces phage phiC31. Mol Microbiol 38: 232-241.
42. Burns, JC, Friedmann, T, Driever, W, Burrascano, M and Yee, JK (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 USA 90: 8033-8037.
43. Friedrich, G and Soriano, P (1991). Promoter traps in embryonic stem cells: a genetic screen to identify and mutate developmental genes in mice. Genes Dev 5: 1513-1523.
44. Sauer, B (1993). Manipulation of transgenes by site-specific recombination: use of Cre recombinase. Meth Enzymol 225: 890-900.