Promoter choice for retroviral vectors: Transcriptional strength versus trans-activation potential

Human Gene Therapy

Volumn 18, Issue 9, 2007, Pages 849-860

  Weber, Erin L ^a,b   Cannon, Paula M ^a,b  

^a CHILDREN S HOSPITAL LOS ANGELES   (United States)

^b UNIVERSITY OF SOUTHERN CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA ACTIN; CHICKEN BETA ACTIN; ELONGATION FACTOR 1ALPHA; RETROVIRUS VECTOR; UNCLASSIFIED DRUG;

ARTICLE; CELL LINE; CONTROLLED STUDY; CYTOMEGALOVIRUS; ENHANCER REGION; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSCRIPTION; GENOME; HUMAN; HUMAN CELL; IMMUNE DEFICIENCY; LONG TERMINAL REPEAT; LYMPHOID CELL; NONHUMAN; PROMOTER REGION; REPORTER GENE; T LYMPHOCYTE ACTIVATION; TARGET CELL; TRANSACTIVATION; TRANSCRIPTION INITIATION; X CHROMOSOME LINKED DISORDER;

CELL LINE; ENHANCER ELEMENTS (GENETICS); FLOW CYTOMETRY; GENE DOSAGE; GENES, REPORTER; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HUMANS; JURKAT CELLS; K562 CELLS; KIDNEY; MODELS, GENETIC; MOLONEY MURINE LEUKEMIA VIRUS; PLASMIDS; PROMOTER REGIONS (GENETICS); RETROVIRIDAE; TERMINAL REPEAT SEQUENCES; TRANS-ACTIVATION (GENETICS); TRANSCRIPTION, GENETIC; TRANSFECTION;

CYTOMEGALOVIRUS;

EID: 34848851094     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2007.067     Document Type: Article

References (54)

1. BARTHOLEMEW, C., and IHLE, J.N. (1991). Retroviral insertions 90 kilobases proximal to the Evi-1 myeloid transforming gene activate transcription from the normal promoter. Mol. Cell Biol. 11, 1820-1828.
2. BELL, A.C., WEST, A.G., and FELSENFELD, G. (1999). The protein CTCF is required for the enhancer blocking activity of vertebrate insulators. Cell 98, 387-396.
3. BOGGS, S.S., GREGG, R.G., BORENSTEIN, N., and SMITHIES, O. (1986). Efficient transformation and frequent single-site, single-copy insertion of DNA can be obtained in mouse erythroleukemia cells transformed by electroporation. Exp. Hematol. 14, 988-994.
4. BUNGERT, J., TANIMOTO, K., PATEL, S., LIU, Q., FEAR, M., and ENGEL, J.D. (1999). Hypersensitive site 2 specifies a unique function within the human β-globin locus control region to stimulate globin gene transcription. Mol. Cell. Biol. 19, 3062-3072.
5. CHALLITA, P.M., SKELTON, D., EL-KHOUEIRY, A., YU, X.J., WEINBERG, K., and KOHN, D.B. (1995). 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. 69, 748-755.
6. CHECK, E. (2005). Gene therapy put on hold as third child develops cancer. Nature 433, 561.
7. CHEN, M., KASAHARA, N., KEENE, D.R., CHAN, L., HOEFFLER, W.K., FINLAY, D., BARCOVA, M., CANNON, P.M., MAZUREK, C., and WOODLEY, D.T. (2002). Restoration of type VII collagen expression and function in dystrophic epidermolysis bullosa. Nat. Genet. 32, 670-675.
8. COFFIN. J.M., HUGHES, S.H., and VARMUS, H.E. (1997). Synthesis and processing of viral RNA. In Retroviruses. (Cold Spring Harbor Laboratory Press, Woodbury, NY) pp. 205-261.
9. CORNETTA, K., MORGAN, R.A., GILLIO, A., STURM, S., BALTRUCKI, L., O'REILLY, R., and ANDERSON, W.F. (1991). No retroviremia or pathology in long-term follow-up of monkeys exposed to murine amphotropic retrovirus. Hum. Gene Ther. 2, 215-219.
10. DESGROSEILLERS, L., and JOLICOEUR, P. (1984). Mapping the viral sequences conferring the leukemogenicity and disease specificity in Moloney and amphotropic murine leukemia viruses. J. Virol. 52, 448-456.
11. DOUGHERTY, J.P., and TEMIN, H.M. (1987). A promoterless retroviral vector indicates that there are sequences in U3 required for 3′ RNA processing. Proc. Natl. Acad. Sci. U.S.A. 84, 1197-1201.
12. GREZ, M., ZORNIG, M., NOWOCK, J., and ZIEGLER, M. (1991). A single point mutation activates the Moloney murine leukemia virus long terminal repeat in embryonal stem cells. J. Virol. 65, 4691-4698.
13. HACEIN-BEY-ABINA, S., VON KALLE, C., SCHMIDT, M., McCORMACK, M.P., WULFFRAAT, N., LEBOULCH, P., LIM, A., OSBORNE, C.S., PAWLIUK, R., MORILLON, E., SORENSEN, R., FORSTER, A., FRASER, P., COHEN, J.I., DE SAINT BASILE, G., ALEXANDER, I., WINTERGERST, U., FREBOURG, T., AURIAS, A., STOPPA-LYONNET, D., ROMANA, S., RADFORD-WEISS, I., GROSS, F., VALENSI, F., DELABESSE, E., MACINTYRE, E., SIGAUX, F., SOULIER, J., LEIVA, L.E., WISSLER, M., PRINZ, C., RABBITTS, T.H., LE DEIST, F., FISCHER, A., and CAVAZZANA-CALVO, M. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419.
14. HANECAK, R., PATTENGALE, P.K., and FAN, H. (1991). Deletion of a GC-rich region flanking the enhancer element within the long terminal repeat sequences alters the disease specificity of Moloney murine leukemia virus. J. Virol. 65, 5357-5363.
15. HILDINGER, M., ECKERT, H.G., SCHILZ, A.J., JOHN, J., OSTERTAG, W., and BAUM, C. (1998). FMEV vectors: Both retroviral long terminal repeat and leader are important for high expression in transduced hematopoietic cells. Gene Ther. 5, 1575-1579.
16. KUSTIKOVA, O., FEHSE, B., MODLICH, U., YANG, M., DULLMANN, J., KAMINO, K., VON NEUHOFF, N., SCHLEGELBERGER, B., LI, Z., and BAUM, C. (2005). Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 308, 1171-1174.
17. LAZO, P.A., LEE, J.S., and TSICHLIS, P.N. (1990). Long-distance activation of the Myc protooncogene by provirus insertion in Mlvi-1 or Mlvi-4 in rat T-cell lymphomas. Proc. Natl. Acad. Sci. U.S.A. 87, 170-173.
18. LEE, J.H., HART, S.R., and SKALNIK, D.G. (2004). Histone deacetylase activity is required for embryonic stem cell differentiation. Genesis 38, 32-38.
19. LEMON, B., and TJIAN, R. (2000). Orchestrated response: A symphony of transcription factors for gene control. Genes Dev. 14, 2551-2569.
20. LI, Y., GOLEMIS, E., HARTLEY, J.W., and HOPKINS, N. (1987). Disease specificity of nondefective Friend and Moloney murine leukemia viruses is controlled by a small number of nucleotides. J. Virol. 61, 693-700.
21. LOZZIO, B.B., LOZZIO, C.B., BAMBERGER, E.G., and FELIU, A.S. (1981). A multipotential leukemia cell line (K-562) of human origin. Proc. Soc. Exp. Biol. Med. 166, 546-550.
22. LUCKOW, B., and SCHUTZ, G. (1987). CAT constructions with multiple unique restriction sites for the functional analysis of eukaryotic promoters and regulatory elements. Nucleic Acids Res. 15, 5490.
23. MANLEY, N.R., O'CONNELL, M., SUN, W., SPECK, N.A., and HOPKINS, N. (1993). Two factors that bind to highly conserved sequences in mammalian type C retroviral enhancers. J. Virol. 67, 1967-1975.
24. McKNIGHT, S.L., and KINGSBURY, R. (1982). Transcriptional control signals of a eukaryotic protein-coding gene. Science 217, 316-324.
25. MESHORER, E., and MISTELLI, T. (2006). Chromatin in pluripotent embryonic stem cells and differentiation. Nat. Rev. Mol. Cell Biol. 7, 540-546.
26. MODLICH, U., BOHNE, J., SCHMIDT, M., VON KALLE, C., KNOSS, S., SCHAMBACH, A., and BAUM, C. (2006). Cell-culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 108, 2545-2553.
27. MONTINI, E., CESANA, D., SCHMIDT, M., SANVITO, F., PONZONI, M., BARTHOLOMAE, C., SERGI SERGI L., BENEDICENTI, F., AMBROSI, A., DI SERIO, C., DOGLIONI, C., VON KALLE, C., and NALDINI, L. (2006). Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat. Biotechnol. 24, 687-696.
28. MOOLTEN, F.L., and CUPPLES, L.A. (1992). A model for predicting the risk of cancer consequent to retroviral gene therapy. Hum. Gene Ther. 3, 479-486.
29. MORISHITA, K., PARKER, D.S., MUCENSKI, M.L., JENKINS, N.A., COPELAND, N.G., and IHLE, J.N. (1988). Retroviral activation of a novel gene encoding a zinc finger protein in IL-3 dependent myeloid leukemia cell lines. Cell 54, 831-840.
30. NAM, C.H., and RABBITTS, T.H. (2006). The role of LMO2 in development and in T cell leukemia after chromosomal translocation or retroviral insertion. Mol. Ther. 13, 15-25.
31. NIELSEN, A.L., PALLISGAARD, N., PEDERSEN, F.S., and JORGENSEN, P. (1994). Basic helix-loop-helix proteins in murine type C retrovirus transcriptional regulation. J. Virol. 68, 5638-5647.
32. NIGHTINGALE, S.J., HOLLIS, R.P., PEPPER, K.A., PETERSEN, D., YU, X.J., YANG, C., BAHNER, I., and KOHN, D.B. (2006). Transient gene expression by nonintegrating lentiviral vectors. Mol. Ther. 13, 1121-1132.
33. OTT, M.G., SCHMIDT, M., SCHWARZWAELDER, K., STEIN, S., SILER, U., KOEHL, U., GLIMM, H., KUHLCKE, K., SCHILZ, A., KUNKEL, H., NAUNDORF, S., BRINKMANN, A., DEICHMANN, A., FISCHER, M., BALL, C., PILZ, I., DUNBAR, C., DU, Y., JENKINS, N.A., COPELAND, N.G., LUTHI, U., HASSAN, M., THRASHER, A.J., HOELZER, D., VON KALLE, C., SEGER, R., and GREZ, M. (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.
34. PECKHAM, I., SOBEL, S., COMER, J., JAENISCH, R., and BARKLIS, E. (1989). Retrovirus activation in embyronal carcinoma cells by cellular promoters. Genes Dev. 3, 2062-2071.
35. PERSONS, D.A., ALLAY, J.A., ALLAY, E.R., SMEYNE, R.J., ASHMUN, R.A., SORRENTINO, B.P., and NIENHUIS, A.W. (1997). Retroviral-mediated transfer of the green fluorescent protein gene into murine hematopoietic cells facilitates scoring and selection of transduced progenitors in vitro and identification of genetically modified cells in vivo. Blood 90, 1777-1786.
36. + cells: Implications for leukemogenesis in gene therapy. Leukemia 21, 754-763.
37. RAMALHO-SANTOS, M., YOON, S., MATSUZAKI, Y., MULLIGAN, R.C., and MELTON, D.A. (2002). "Sternness": Transcriptional profiling of embryonic and adult stem cells. Science 298, 597-600.
38. RAMEZANI, A., HAWLEY, T.S., and HAWLEY, R.G. (2000). Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol. Ther. 2, 458-469.
39. REISMAN, D. (1990). Nuclear factor-1 (NF-1) binds to multiple sites with the transcriptional enhancer of Moloney murine leukemia virus. FEBS Lett. 277, 209-211.
40. ROBBINS, P.B., YU, X.J., SKELTON, D.M., PEPPER, K.A., WASSERMAN, R.M., ZHU, J., and KOHN, D.B. (1997). Increased probability of expression from modified retroviral vectors in embryonal stem cells and embryonal carcinoma cells. J. Virol. 71, 9466-9474.
41. ROYER-POKORA, B., LOOS, U., and LUDWIG, W.D. (1991). TTG-2, a new gene encoding a cysteine-rich protein with the LIM motif, is overexpressed in acute T-cell leukaemia with the t(11;14) (p13;q11). Oncogene 6, 1887-1893.
42. SEGGEWISS, R., PITTALUGA, S., ADLER, R.L., GUENAGA, J., FERGUSON, C., PILZ, I.H., RYU, B., SORRENTINO, B.P., YOUNG, W.S., III, DONAHUE, R.E., VON KALLE, C., NIENHUIS, A.W., and DUNBAR, C.E. (2006). Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Gene Ther. 107, 3865-3867.
43. SHINNICK, T.M., LERNER, R.A., and SUTCLIFFE, J.G. (1981). Nucleotide sequence of Moloney murine leukaemia virus. Nature. 293, 543-548.
44. SHOU, Y., MA, Z., LU, T., and SORRENTINO, B.P. (2006). Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy. Proc. Natl. Acad. Sci. U.S.A. 103, 11730-11735.
45. SKOTZKO, M., WU, L., ANDERSON, W.F., GORDON, E.M., and HALL, F.L. (1995). Retroviral vector-mediated gene transfer of antisense cyclin G1 (CYCG1) inhibits proliferation of human osteogenic sarcoma cells. Cancer Res. 55, 5493-5498.
46. SUN, W., GRAVES, B.J., and SPECK, N.A. (1995). Transactivation of the Moloney murine leukemia virus and T-cell receptor β-chain enhancers by cbf and ets requires intact binding sites for both proteins. J. Virol. 69, 4941-4949.
47. TONEGUZZO, F., KEATING, A., GLYNN, S., and McDONALD, K. (1988). Electric field-mediated gene transfer: characterization of DNA transfer and patterns of integration in lymphoid cells. Nucleic Acids Res. 16, 5515-5532.
48. TROBRIDGE, G., JOSEPHSON, N., VASSILOPOULOS, G., MAC, J., and RUSSELL, D.W. (2002). Improved foamy virus vectors with minimal viral sequences. Mol. Ther. 6, 321-328.
49. WAHLERS, A., ZIPFEL, P.F., SCHWIEGER, M., OSTERTAG, W., and BAUM, C. (2002a). In vivo analysis of retroviral enhancer mutations in hematopoietic cells: SP1/EGR1 and ETS/GATA motifs contribute to long terminal repeat specificity. J. Virol. 76, 303-312.
50. WAHLERS, A., KUSTIKOVA, O., ZIPFEL, P.F., ITOH, K., KOESTER, M., HEBERLEIN, C., LI, Z., SCHIEDLMEIER, B., SKERKA, C., FEHSE, B., and BAUM, C. (2002b). Upstream conserved sequences of mouse leukemia viruses are important for high transgene expression. Genes Dev. 16, 2583-2592.
51. WU, X., LI, Y., CRISE, B., and BURGESS, S.M. (2003). Transcription start regions in the human genome are favored targets for MLV integration. Science 300, 1749-1751.
52. WURTELE, H., LITTLE, K.C.E., and CHARTRAND, P. (2003). Illegitimate DNA integration in mammalian cells. Gene Ther. 10, 1791-1799.
53. YU, S.F., VON RUDEN, T., KANTOFF, P.W., GARBER, C., SEILBERG, M., RUTHER, U., ANDERSON, W.F., WAGNER, E.F., and GILBOA, E. (1986). Self-inactivating retroviral vectors designed for transfer of whole genes into mammalian cells. Proc. Natl. Acad. Sci. U.S.A. 83, 3194-3198.
54. ZUFFEREY, R., DULL, T., MANDEL, R.J., BUKOVSKY, A., QUIROZ, D., NALDINI, L., and TRONO, D. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J. Virol. 72, 9873-9880.