Murine leukemia following irradiation conditioning for transplantation of lentivirally-modified hematopoietic stem cells

European Journal of Haematology

Volumn 78, Issue 4, 2007, Pages 303-313

  Siapati, Elena K ^a   Bigger, Brian W ^b   Kashofer, Karl ^a   Themis, Mike ^b   Thrasher, Adrian J ^c   Bonnet, Dominique ^a  

^a IMPERIAL CANCER RESEARCH FUND   (United Kingdom)

^b IMPERIAL COLLEGE LONDON   (United Kingdom)

^c UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

Endogenous retrovirus; Gene therapy; Irradiation; Lentivirus vector; Leukemia

Indexed keywords

LENTIVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; EX VIVO STUDY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMOPHILIA B; IMMUNOSUPPRESSIVE TREATMENT; LYMPHATIC LEUKEMIA; MOUSE; MURINE LEUKEMIA; NONHUMAN; NONOBESE DIABETIC MOUSE; PRIORITY JOURNAL; SCID MOUSE;

ANIMALS; GENE THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HEMOPHILIA B; LENTIVIRUS; LEUKEMIA; MICE; MICE, INBRED C57BL; RADIATION CHIMERA; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; SENSITIVITY AND SPECIFICITY; TRANSDUCTION, GENETIC; TRANSPLANTATION CONDITIONING; VIRUS INTEGRATION;

EID: 33847636598     PISSN: 09024441     EISSN: 16000609     Source Type: Journal    
DOI: 10.1111/j.1600-0609.2006.00813.x     Document Type: Article

References (43)

1. Cavazzana-Calvo M, Hacein-Bey S, de Saint Basile G, et al. Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 2000;288:669-72.
2. Gaspar HB, Parsley KL, Howe S, et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gammaretroviral vector. Lancet 2004;364:2181-7.
3. Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002;346:1185-93.
4. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med 2003;348:255-6.
5. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003;302:415-9.
6. Modlich U, Kustikova OS, Schmidt M, et al. Leukemias following retroviral transfer of multidrug resistance 1 (MDR1) are driven by combinatorial insertional mutagenesis. Blood 2005;105:4235-46.
7. Bunting KD, Galipeau J, Topham D, Benaim E, Sorrentino BP. Transduction of murine bone marrow cells with an MDR1 vector enables ex vivo stem cell expansion, but these expanded grafts cause a myeloproliferative syndrome in transplanted mice. Blood 1998;92:2269-79.
8. Li Z, Dullmann J, Schiedlmeier B, et al. Murine leukemia induced by retroviral gene marking. Science 2002;296:497.
9. Themis M, Waddington SN, Schmidt M, et al. Oncogenesis following delivery of a nonprimate lentiviral gene therapy vector to fetal and neonatal mice. Mol Ther 2005;12:763-71.
10. Coutelle C, Themis M, Waddington SN, et al. Gene therapy progress and prospects: fetal gene therapy - first proofs of concept - some adverse effects. Gene Ther 2005;12:1601-7.
11. Imren S, Fabry ME, Westerman KA, et al. High-level beta-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells. J Clin Invest 2004;114:953-62.
12. Woods NB, Muessig A, Schmidt M, et al. Lentiviral vector transduction of NOD/SCID repopulating cells results in multiple vector integrations per transduced cell: risk of insertional mutagenesis. Blood 2003;101:1284-9.
13. Mitchell RS, Beitzel BF, Schroder AR, et al. Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2004;2:E234.
14. Schroder AR, Shinn P, Chen H, et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 2002;110:521-9.
15. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science 2003;300:1749-51.
16. Bigger BW, Siapati EK, Mistry A, et al. Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther 2006;13:117-26.
17. Waddington SN, Nivsarkar MS, Mistry AR, et al. Permanent phenotypic correction of hemophilia B in immunocompetent mice by prenatal gene therapy. Blood 2004;104:2714-21.
18. Demaison C, Parsley K, Brouns G, et al. High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of immunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther 2002;13:803-13.
19. Siapati EK, Bigger BW, Miskin J, et al. Comparison of HIV- and EIAV-based vectors on their efficiency in transducing murine and human hematopoietic repopulating cells. Mol Ther 2005;12:537-46.
20. Schmidt M, Zickler P, Hoffmann G, et al. Polyclonal long-term repopulating stem cell clones in a primate model. Blood 2002;100:2737-43.
21. Schmidt M, Hoffmann G, Wissler M, et al. Detection and direct genomic sequencing of multiple rare unknown flanking DNA in highly complex samples. Hum Gene Ther 2001;12:743-9.
22. Arnold BA, Hepler RW, Keller PM. One-step fluorescent probe product-enhanced reverse transcriptase assay. Biotechniques 1998;25:98-106.
23. Kingsman SM, Mitrophanous K, Olsen JC. Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther 2005;12:3-4.
24. Dandri M, Schirmacher P, Rogler CE. Woodchuck hepatitis virus X protein is present in chronically infected woodchuck liver and woodchuck hepatocellular carcinomas which are permissive for viral replication. J Virol 1996;70:5246-54.
25. Flajolet M, Tiollais P, Buendia MA, Fourel G. Woodchuck hepatitis virus enhancer I and enhancer II are both involved in N-myc2 activation in woodchuck liver tumors. J Virol 1998;72:6175-80.
26. - bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol Ther 2000;1:566-73.
27. Drize NJ, Olshanskaya YV, Gerasimova LP, et al. Lifelong hematopoiesis in both reconstituted and sublethally irradiated mice is provided by multiple sequentially recruited stem cells. Exp Hematol 2001;29:786-94.
28. Johansson FK, Brodd J, Eklof C, et al. Identification of candidate cancer-causing genes in mouse brain tumors by retroviral tagging. Proc Natl Acad Sci USA 2004;101:11334-7.
29. Akagi K, Suzuki T, Stephens RM, Jenkins NA, Copeland NG. RTCGD: retroviral tagged cancer gene database. Nucleic Acids Res 2004;32:523-7.
30. Sastry L, Xu Y, Duffy L, et al. Product-enhanced reverse transcriptase assay for replication-competent retrovirus and lentivirus detection. Hum Gene Ther 2005;16:1227-36.
31. Cho K, Greenhalgh D. Injury-associated induction of two novel and replication-defective murine retroviral RNAs in the liver of mice. Virus Res 2003;93:189-98.
32. Seggewiss R, Pittaluga S, Adler RL, et al. Acute myeloid leukemia associated with retroviral gene transfer to hematopoietic progenitor cells of a rhesus macaque. Blood 2006;107:3865-7.
33. Kiem HP, Sellers S, Thomasson B, et al. Long-term clinical and molecular follow-up of large animals receiving retrovirally transduced stem and progenitor cells: no progression to clonal hematopoiesis or leukemia. Mol Ther 2004;9:389-95.
34. Kustikova O, Fehse B, Modlich U, et al. Clonal dominance of hematopoietic stem cells triggered by retroviral gene marking. Science 2005;308:1171-4.
35. Ellis RW, Stockert E, Fleissner E. Association of endogenous retroviruses with radiation-induced leukemias of BALB/c mice. J Virol 1980;33:652-60.
36. Lieberman M, Haran-Ghera N, Kaplan HS. Potentiation of virus leukaemogenesis in C57BL mice by X-irradiation or urethane. Nature 1964;203:420-2.
37. Pearce DJ, Ridler CM, Simpson C, Bonnet D. Multiparameter analysis of murine bone marrow side population cells. Blood 2004;103:2541-6.
38. Byfield PE, Finklestein JZ, Tittle KL, Hsi C, Imagawa DT. The role of serum factors in the acceleration by Freund's complete adjuvant of the growth of transplanted murine leukemic cells. Cancer Res 1975;35:409-14.
39. Siegel BV, Morton JI. Immunologic stimuli in relation to leukemogenesis. 1. Potentiation of Rauscher viral leukemia by Freund's adjuvants. J Natl Cancer Inst 1972;48:1681-5.
40. Brodsky I, Foley B, Haines D, et al. Expression of HERV-K proviruses in human leukocytes. Blood 1993;81:2369-74.
41. Menendez L, Benigno BB, McDonald JF. L1 and HERV-W retrotransposons are hypomethylated in human ovarian carcinomas. Mol Cancer 2004;3:12.
42. Das PM, Singal R. DNA methylation and cancer. J Clin Oncol 2004;22:4632-42.
43. Gaudet F, Hodgson JG, Eden A, et al. Induction of tumors in mice by genomic hypomethylation. Science 2003;300:489-92.