Tracking of specific integrant clones in dogs treated with foamy virus vectors

Human Gene Therapy

Volumn 22, Issue 2, 2011, Pages 217-224

  Ohmine, Ken ^a,c   Li, Yi ^a   Bauer Jr , Thomas R ^b   Hickstein, Dennis D ^b   Russell, David W ^a  

^a UNIVERSITY OF WASHINGTON   (United States)

^b NATIONAL CANCER INSTITUTE   (United States)

^c JICHI MEDICAL UNIVERSITY   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

GENOMIC DNA; RETROVIRUS VECTOR; SPUMA VIRUS VECTOR; UNCLASSIFIED DRUG;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CHROMOSOME; CLONE; EXPERIMENTAL DOG; GENE DOSAGE; LEUKOCYTE ADHESION DEFICIENCY; MOLECULAR CLONING; NONHUMAN; POLYMERASE CHAIN REACTION; PROMOTER REGION; PROTO ONCOGENE; TUMOR SUPPRESSOR GENE; VIRUS DNA CELL DNA INTERACTION;

ANIMALS; CLONE CELLS; DOGS; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; PROTO-ONCOGENES; PROVIRUSES; SPUMAVIRUS; VIRUS INTEGRATION;

ANIMALIA; CANIS FAMILIARIS; HUMAN SPUMARETROVIRUS; MURINAE;

EID: 79951845565     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2010.072     Document Type: Article

References (45)

1. Aiuti, A., Cassani, B., Andolfi, G., Mirolo, M., Biasco, L., Recchia, A., Urbinati, F., Valacca, C., Scaramuzza, S., Aker, M., Slavin, S., Cazzola, M., Sartori, D., Ambrosi, A., Di Serio, C., Ron-carolo, M.G., Mavilio, F., and Bordignon, C. (2007). Multi-lineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy. J. Clin. Invest. 117, 2233-2240. (Pubitemid 47219568)
2. Bauer, T.R., Jr., Allen, J.M., Hai, M., Tuschong, L.M., Khan, I.F., Olson, E.M., Adler, R.L., Burkholder, T.H., Gu, Y.C., Russell, D.W., and Hickstein, D.D. (2008). Successful treatment of canine leukocyte adhesion deficiency by foamy virus vectors. Nat. Med. 14, 93-97.
3. Bozorgmehr, F., Laufs, S., Sellers, S.E., Roeder, I., Zeller, W.J., Dunbar, C.E., and Fruehauf, S. (2007). No evidence of clonal dominance in primates up to 4 years following transplantation of multidrug resistance 1 retrovirally transduced long-term repopulating cells. Stem Cells 25, 2610-2618. (Pubitemid 47582754)
4. Brito, G.C., Fachel, A.A., Vettore, A.L., Vignal, G.M., Gimba, E.R., Campos, F.S., Barcinski, M.A., Verjovski-Almeida, S., and Reis, E.M. (2008). Identification of protein-coding and intronic noncoding RNAs down-regulated in clear cell renal carcinoma. Mol. Carcinog. 47, 757-767.
5. Busslinger, M., Klix, N., Pfeffer, P., Graninger, P.G., and Kozmik, Z. (1996). Deregulation of PAX-5 by translocation of the Emu enhancer of the IgH locus adjacent to two alternative PAX-5 promoters in a diffuse large-cell lymphoma. Proc. Natl. Acad. Sci. U.S.A. 93, 6129-6134. (Pubitemid 26190815)
6. Calmels, B., Ferguson, C., Laukkanen, M.O., Adler, R., Faulha-ber, M., Kim, H.J., Sellers, S., Hematti, P., Schmidt, M., von Kalle, C., Akagi, K., Donahue, R.E., and Dunbar, C.E. (2005). Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood 106, 2530-2533. (Pubitemid 41510830)
7. Carney, J.P., Maser, R.S., Olivares, H., Davis, E.M., Le Beau, M., Yates, J.R., 3rd, Hays, L., Morgan, W.F., and Petrini, J.H. (1998). The hMre11/hRad50 protein complex and Nijmegen breakage syndrome: linkage of double-strand break repair to the cellular DNA damage response. Cell 93, 477-486. (Pubitemid 28232091)
8. Cazzaniga, G., Daniotti, M., Tosi, S., Giudici, G., Aloisi, A., Po-gliani, E., Kearney, L., and Biondi, A. (2001). The paired box domain gene PAX5 is fused to ETV6/TEL in an acute lym-phoblastic leukemia case. Cancer Res. 61, 4666-4670. (Pubitemid 32691872)
9. Dai, Z., Kerzic, P., Schroeder, W.G., and McNiece, I.K. (2001). Deletion of the Src homology 3 domain and C-terminal pro-line-rich sequences in Bcr-Abl prevents Abl interactor 2 degradation and spontaneous cell migration and impairs leukemogenesis. J. Biol. Chem. 276, 28954-28960.
10. Deichmann, A., Hacein-Bey-Abina, S., Schmidt, M., Garrigue, A., Brugman, M.H., Hu, J., Glimm, H., Gyapay, G., Prum, B., Fraser, C.C., et al. (2007). Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy. J. Clin. Invest. 117, 2225-2232. (Pubitemid 47219567)
11. Du, Y., Jenkins, N.A., and Copeland, N.G. (2005). Insertional mutagenesis identifies genes that promote the immortalization of primary bone marrow progenitor cells. Blood 106, 3932-3939. (Pubitemid 41739034)
12. Gritti, C., Dastot, H., Soulier, J., Janin, A., Daniel, M.T., Madani, A., Grimber, G., Briand, P., Sigaux, F., and Stern, M.H. (1998). Transgenic mice for MTCP1 develop T-cell prolymphocytic leukemia. Blood 92, 368-373. (Pubitemid 28323696)
13. Grove, M., Demyanenko, G., Echarri, A., Zipfel, P.A., Quiroz, M.E., Rodriguiz, R.M., Playford, M., Martensen, S.A., Robinson, M.R., Wetsel, W.C., Maness, P.F., and Pendergast, A.M. (2004). ABI2-deficient mice exhibit defective cell migration, aberrant dendritic spine morphogenesis, and deficits in learning and memory. Mol. Cell. Biol. 24, 10905-10922. (Pubitemid 39603145)
14. 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). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302, 415-419. (Pubitemid 37296260)
15. Hai, M., Adler, R.L., Bauer, T.R., Jr., Tuschong, L.M., Gu, Y.C., Wu, X., and Hickstein, D.D. (2008). Potential genotoxicity from integration sites in CLAD dogs treated successfully with gammaretroviral vector-mediated gene therapy. Gene Ther. 15, 1067-1071. (Pubitemid 351927697)
16. Hawley, R.G., Lieu, F.H., Fong, A.Z., and Hawley, T.S. (1994). Versatile retroviral vectors for potential use in gene therapy. Gene Ther. 1, 136-138.
17. Hayakawa, J., Washington, K., Uchida, N., Phang, O., Kang, E.M., Hsieh, M.M., and Tisdale, J.F. (2009). Long-term vector integration site analysis following retroviral mediated gene transfer to hematopoietic stem cells for the treatment of HIV infection. PLoS ONE 4, e4211.
18. Hendrie, P.C., Huo, Y., Stolitenko, R.B., and Russell, D.W. (2008). A rapid and quantitative assay for measuring neighboring gene activation by vector proviruses. Mol. Ther. 16, 534-540. (Pubitemid 351314993)
19. 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. (2008). Inser-tional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J. Clin. Invest. 118, 3143-3150.
20. Iida, S., Rao, P.H., Nallasivam, P., Hibshoosh, H., Butler, M., Louie, D.C., Dyomin, V., Ohno, H., Chaganti, R.S., and Dalla-Favera, R. (1996). The t(9;14)(p13;q32) chromosomal translo-cation associated with lymphoplasmacytoid lymphoma involves the PAX-5 gene. Blood 88, 4110-4117. (Pubitemid 26399155)
21. Josephson, N.C., Vassilopoulos, G., Trobridge, G.D., Priestley, G.V., Wood, B.L., Papayannopoulou, T., and Russell, D.W. (2002). Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors. Proc. Natl. Acad. Sci. U.S.A. 99, 8295-8300. (Pubitemid 34651046)
22. Josephson, N.C., Trobridge, G., and Russell, D.W. (2004). Transduction of long-term and mobilized peripheral blood-derived NOD/SCID repopulating cells by foamy virus vectors. Hum. Gene Ther. 15, 87-92. (Pubitemid 38147191)
23. Kiem, H.P., Allen, J., Trobridge, G., Olson, E., Keyser, K., Peterson, L., and Russell, D.W. (2007). Foamy virus-mediated gene transfer to canine repopulating cells. Blood 109, 65-70. (Pubitemid 46053045)
24. 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. (Pubitemid 40696434)
25. Leurs, C., Jansen, M., Pollok, K.E., Heinkelein, M., Schmidt, M., Wissler, M., Lindemann, D., Von Kalle, C., Rethwilm, A., Williams, D.A., and Hanenberg, H. (2003). Comparison of three retroviral vector systems for transduction of nonobese diabetic/severe combined immunodeficiency mice re-populating human CD34(\+) cord blood cells. Hum. Gene Ther. 14, 509-519. (Pubitemid 36442706)
26. Li, Z., Dü llmann, J., Schiedlmeier, B., Schmidt, M., von Kalle, C., Meyer, J., Forster, M., Stocking, C., Wahlers, A., Frank, O., Ostertag, W., Kü hlcke, K., Eckert, H.G., Fehse, B., and Baum, C. (2002). Murine leukemia induced by retroviral gene marking. Science 296, 497. (Pubitemid 34408663)
27. Li, Z.D., Wu, Y., Bao, Y.L., Yu, C.L., Guan, L.L., Wang, Y.Z., Meng, X.Y., and Li, Y.X. (2009). Identification and characterization of human ARIP2 and its relation to breast cancer. Cytokine 46, 251-259.
28. Lin, T.Y., Huang, C.H., Chou, W.G., and Juang, J.L. (2004). Abi enhances Abl-mediated CDC2 phosphorylation and inactiva-tion. J. Biomed. Sci. 11, 902-910.
29. Metais, J.Y., and Dunbar, C.E. (2008). The MDS1-EVI1 gene complex as a retrovirus integration site: impact on behavior of hematopoietic cells and implications for gene therapy. Mol. Ther. 16, 439-449. (Pubitemid 351314981)
30. 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.
31. 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. (Pubitemid 43882120)
32. Morishita, K., Parganas, E., William, C.L., Whittaker, M.H., Drabkin, H., Oval, J., Taetle, R., Valentine, M.B., and Ihle, J.N. (1992). Activation of EVI1 gene expression in human acute myelogenous leukemias by translocations spanning 300-400 kilobases on chromosome band 3q26. Proc. Natl. Acad. Sci. U.S.A. 89, 3937-3941.
33. Nagy, K.Z., Laufs, S., Gentner, B., Naundorf, S., Kuehlcke, K., Topaly, J., Buss, E.C., Zeller, W.J., and Fruehauf, S. (2004). Clonal analysis of individual marrow-repopulating cells after experimental peripheral blood progenitor cell transplantation. Stem Cells 22, 570-579. (Pubitemid 39014572)
34. Ott, M.G., Schmidt, M., Schwarzwaelder, K., Stein, S., Siler, U., Koehl, U., Glimm, H., Kuhlcke, K., Schilz, A., Kunkel, H., 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.
35. Pasqualucci, L., Neumeister, P., Goossens, T., Nanjangud, G., Chaganti, R.S., Kuppers, R., and Dalla-Favera, R. (2001). Hy-permutation of multiple protooncogenes in B-cell diffuse large-cell lymphomas. Nature 412, 341-346. (Pubitemid 32691848)
36. Resnick, I.B., Kondratenko, I., Pashanov, E., Maschan, A.A., Kar-achunsky, A., Togoev, O., Timakov, A., Polyakov, A., Tvers-kaya, S., Evgrafov, O., and Roumiantsev, A.G. (2003). 657del5 mutation in the gene for Nijmegen breakage syndrome (NBS1) in a cohort of Russian children with lymphoid tissue malignancies and controls. Am. J. Med. Genet. A. 120A, 174-179. (Pubitemid 37063793)
37. Stein, S., Ott, M.G., Schultze-Strasser, S., Jauch, A., Burwinkel, B., Kinner, A., Schmidt, M., Kramer, A., Schwable, J., Glimm, H., 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.
38. Stern, M.H., Soulier, J., Rosenzwajg, M., Nakahara, K., Canki-Klain, N., Aurias, A., Sigaux, F., and Kirsch, I.R. (1993). MTCP-1: a novel gene on the human chromosome Xq28 translocated to the T cell receptor alpha/delta locus in mature T cell proliferations. Oncogene 8, 2475-2483. (Pubitemid 23252377)
39. 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. (Pubitemid 36305333)
40. Trobridge, G.D., Miller, D.G., Jacobs, M.A., Allen, J.M., Kiem, H.P., Kaul, R., and Russell, D.W. (2006). Foamy virus vector integration sites in normal human cells. Proc. Natl. Acad. Sci. U.S.A. 103, 1498-1503. (Pubitemid 43191194)
41. Trobridge, G.D., Allen, J.M., Peterson, L., Ironside, C.G., Russell, D., and Kiem, H.P. (2009). Foamy and lentiviral vectors transduce canine long-term repopulating cells at similar efficiency. Hum. Gene Ther. 20, 519-523.
42. Varon, R., Reis, A., Henze, G., von Einsiedel, H.G., Sperling, K., and Seeger, K. (2001). Mutations in the Nijmegen Breakage Syndrome gene (NBS1) in childhood acute lymphoblastic leukemia (ALL). Cancer Res. 61, 3570-3572. (Pubitemid 32694962)
43. Vassilopoulos, G., Trobridge, G., Josephson, N.C., and Russell, D.W. (2001). Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors. Blood 98, 604-609.
44. Woods, N.B., Bottero, V., Schmidt, M., von Kalle, C., and Verma, I.M. (2006). Gene therapy: therapeutic gene causing lym-phoma. Nature 440, 1123.
45. Zychlinski, D., Schambach, A., Modlich, U., Maetzig, T., Meyer, J., Grassman, E., Mishra, A., and Baum, C. (2008). Physiological promoters reduce the genotoxic risk of integrating gene vectors. Mol. Ther. 16, 718-725. (Pubitemid 351426173)