Unique risk factors for insertional mutagenesis in a mouse model of XSCID gene therapy

Proceedings of the National Academy of Sciences of the United States of America

Volumn 103, Issue 31, 2006, Pages 11730-11735

  Shou, Yan ^a   Ma, Zhijun ^a   Lu, Taihe ^a   Sorrentino, Brian P ^a  

^a ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

Arf; Immunodeficiency; Leukemia; Stem cells; Vector

Indexed keywords

ARF PROTEIN; GENOMIC DNA; GREEN FLUORESCENT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CARCINOGENESIS; COMBINED IMMUNODEFICIENCY; CONTROLLED STUDY; EXPRESSION VECTOR; GENE INSERTION; GENE THERAPY; GENETIC TRANSDUCTION; HEMATOPOIETIC STEM CELL; HUMAN; HUMAN CELL; INTERNAL RIBOSOME ENTRY SITE; LYMPHOCYTE TRANSFORMATION; MOUSE; MUTAGENESIS; NONHUMAN; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; PROTO ONCOGENE; RISK FACTOR; SCID MOUSE; T CELL LYMPHOMA; TUMOR SUPPRESSOR GENE; X CHROMOSOME LINKED DISORDER;

ANIMALS; BONE MARROW TRANSPLANTATION; CYCLIN-DEPENDENT KINASE INHIBITOR P16; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC DISEASES, X-LINKED; HUMANS; IMMUNOGLOBULIN GAMMA-CHAINS; LYMPHOMA, T-CELL; MICE; MICE, KNOCKOUT; MICE, SCID; MUTAGENESIS, INSERTIONAL; RISK FACTORS; SEVERE COMBINED IMMUNODEFICIENCY; TUMOR SUPPRESSOR PROTEIN P14ARF;

ANIMALIA;

EID: 33746801652     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0603635103     Document Type: Article

References (37)

1. Aiuti, A., Slavin, S., Aker, M., Ficara, F., Deola, S., Mortellaro, A., Morecki, S., Andolfi, G., Tabucchi, A., Carlucci, F., et al. (2002) Science 296, 2410-2413.
2. Cavazzana-Calvo, M., Hacein-Bey, S., De Saint, B. G., Gross, F., Yvon, E., Nusbaum, P., Selz, F., Hue, C., Certain, S., Casanova, J. L., et al. (2000) Science 288, 669-672.
3. 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. (2004) Lancet 364, 2181-2187.
4. Hacein-Bey-Abina, S., Le Deist, F., Carlier, F., Bouneaud, C., Hue, C, de Villartay, J. P., Thrasher, A. J., Wuiffraat, N., Sorensen, R., Dupuis-Girod, S., et al. (2002) N. Engl. J. Med. 346, 1185-1193.
5. Baum, C., Dullmann, J., Li, Z., Fehse, B., Meyer, J., Williams, D. A. & von Kalle, C. (2003) Blood 101, 2099-2114.
6. Fischer, A., Abina, S. H., Thrasher, A., von Kalle, C. & Cavazzana-Calvo, M. (2004) N. Engl. J. Med. 350, 2526-2527.
7. 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. (2003) N. Engl. J. Med. 348, 255-256.
8. 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., et al. (2003) Science 302, 415-419.
9. McCormack, M. P. & Rabbitts, T. H. (2004) N. Engl. J. Med. 350, 913-922.
10. Nienhuis, A. W., Dunbar, C. E. & Sorrentino, B. P. (2006) Mol. Ther. 13, 1031-1049.
11. Bonini, C., Grez, M., Traversari, C., Ciceri, F., Marktel, S., Ferrari, G., Dinauer, M., Sadat, M., Aiuti, A., Deola, S., et al. (2003) Nat. Med. 9, 367-369.
12. Ott, M. G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kuhlcke, K., Schilz, A., Kunkel, H., et al. (2006) Nat. Med. 12, 401-409.
13. Modlich, U., Kustikova, O. S., Schmidt, M., Rudolph, C., Meyer, J., Li, Z., Kamino, K., von Neuhoff, N., Schlegelberger, B., Kuehlcke, K., et al. (2005) Blood 105, 4235-4246.
14. Li, Z., Dullmann, J., Schiedlmeier, B., Schmidt, M., von Kalle, C., Meyer, J., Forster, M., Stocking, C., Wahlers, A., Frank, O., et al. (2002) Science 296, 497.
15. Kamijo, T., Bodner, S., van de Kamp, E., Randle, D. H. & Sherr, C. J. (1999) Cancer Res. 59, 2217-2222.
16. Gardie, B., Cayuela, J. M., Martini, S. & Sigaux, F. (1998) Blood 91, 1016-1020.
17. Akagi, K., Suzuki, T., Stephens, R. M., Jenkins, N. A. & Copeland, N. G. (2004) Nucleic Acids Res. 32, D523-D527.
18. Hayashi, H., Matsubara, H., Yokota, T., Kuwabara, I., Kanno, M., Koseki, H., Isono, K., Asano, T. & Taniguchi, M. (1992) J. Immunol. 149, 1223-1229.
19. Balint, I., Muller, A., Nagy, A. & Kovacs, G. (2004) Gene 332, 45-50.
20. Veillette, A., Cruz-Munoz, M. E. & Zhong, M. C. (2006) Trends Immunol. 27, 228-234.
21. Du, Y., Jenkins, N. A. & Copeland, N. G. (2005) Blood 106, 3932-3939.
22. Zhou, S., Morris, J. J., Barnes, Y., Lan, L., Schuetz, J. D. & Sorrentino, B. P. (2002) Proc. Natl. Acad. Sci. USA 99, 12339-12344.
23. Tadjali, M., Zhou, S., Rehg, J. & Sorrentino, B. P. (2006) Stem Cells 24, 1556-1563.
24. Zindy, F., Eischen, C. M., Randle, D. H., Kamijo, T., Cleveland, J. L., Sherr, C. J. & Roussel, M. F. (1998) Genes Dev. 12, 2424-2433.
25. Eischen, C. M., Weber, J. D., Roussel, M. F., Sherr, C. J. & Cleveland, J. L. (1999) Genes Dev. 13, 2658-2669.
26. Oppenheim, D. E., Roberts, S. J., Clarke, S. L., Filler, R., Lewis, J. M., Tigelaar, R. E., Girardi, M. & Hayday, A. C. (2005) Nat. Immunol. 6, 928-937.
27. Woods, N. B., Bottero, V., Schmidt, M., von, Kalle C. & Verma, I. M. (2006) Nature 440, 1123.
28. Lo, M., Bloom, M. L., Imada, K., Berg, M., Bollenbacher, J. M., Bloom, E. T., Kelsall, B. L. & Leonard, W. J. (1999) Blood 94, 3027-3036.
29. Otsu, M., Anderson, S. M., Bodine, D. M., Puck, J. M., O'Shea, J. J. & Candotti, F. (2000) Mol. Ther. 1, 145-153.
30. Soudais, C., Shiho, T., Sharara, L. I., Guy-Grand, D., Taniguchi, T., Fischer, A. & Di Santo, J. P. (2000) Blood 95, 3071-3077.
31. Kiem, H. P., Sellers, S., Thomasson, B., Morris, J. C., Tisdale, J. F., Horn, P. A., Hematti, P., Adler, R., Kuramoto, K., Calmels, B., et al. (2004) Mol. Ther. 9, 389-395.
32. Seggewiss, R., Pittaluga, S., Adler, R. L., Guenaga, F. J., Ferguson, C., Pilz, I. H., Ryu, B., Sorrentino, B. P., Young, W. S., III, Donahue, R. E., et al. (2006) Blood 107, 3865-3867.
33. Emery, D. W., Yannaki, E., Tubb, J. & Stamatoyannopoulos, G. (2000) Proc. Natl. Acad. Sci. USA 97, 9150-9155.
34. Kamijo, T., Zindy, F., Roussel, M. F., Quelle, D. E., Downing, J. R., Ashmun, R. A., Grosveld, G. & Sherr, C. J. (1997) Cell 91, 649-659.
35. Cao, X., Shores, E. W., Hu-Li, J., Anver, M. R., Kelsall, B. L., Russell, S. M., Drago, J., Noguchi, M., Grinberg, A., Bloom, E. T., et al. (1995) Immunity 2, 223-238.
36. Sawai, N., Zhou, S., Vanin, E. F., Houghton, P., Brent, T. P. & Sorrentino, B. P. (2001) Mol. Ther. 3, 78-87.
37. Schmidt, M., Zickler, P., Hoffmann, G., Haas, S., Wissler, M., Muessig, A., Tisdale, J. F., Kuramoto, K., Andrews, R. G., Wu, T., et al. (2002) Blood 100, 2737-2743.