The CD20/αCD20 'suicide' system: Novel vectors with improved safety and expression profiles and efficient elimination of CD20-transgenic T cells

Gene Therapy

Volumn 13, Issue 9, 2006, Pages 789-797

  van Meerten, T ^a   Claessen, M J ^a   Hagenbeek, A ^a   Ebeling, S B ^a  

^a UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

ADCC; CD20; cHS 4; Complement lysis; Rituximab; Suicide gene therapy; WPRE

Indexed keywords

CD20 ANTIGEN; RETROVIRUS VECTOR; RITUXIMAB; MONOCLONAL ANTIBODY;

ANTIGEN EXPRESSION; ARTICLE; CELL BASED GENE THERAPY; CELL CULTURE; CONTROLLED STUDY; FLOW CYTOMETRY; GENE EXPRESSION PROFILING; GENE VECTOR; HUMAN; HUMAN CELL; INSULATOR ELEMENT; PRIORITY JOURNAL; SAFETY; SUICIDE GENE THERAPY; T LYMPHOCYTE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS PARTICLE; ADOPTIVE TRANSFER; CELL CLONE; CELL DEATH; GENE EXPRESSION; GENE THERAPY; GENETIC ENGINEERING; GENETIC TRANSDUCTION; GENETICS; GRAFT VERSUS HOST REACTION; IMMUNOLOGY; LYMPHOCYTE DEPLETION; METABOLISM; METHODOLOGY; PATHOLOGY; RETROVIRUS; SUICIDE TRANSGENE;

MARMOTA MONAX;

ADOPTIVE TRANSFER; ANTIBODIES, MONOCLONAL; ANTIGENS, CD20; CELL DEATH; CLONE CELLS; FLOW CYTOMETRY; GENE EXPRESSION; GENE THERAPY; GENES, TRANSGENIC, SUICIDE; GENETIC ENGINEERING; GENETIC VECTORS; GRAFT VS HOST DISEASE; HUMANS; LYMPHOCYTE DEPLETION; RETROVIRIDAE; T-LYMPHOCYTES; TRANSDUCTION, GENETIC;

EID: 33645964652     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/sj.gt.3302705     Document Type: Article

References (39)

1. Bonini C, Ferrari G, Verzeletti S, Servida P, Zappone E, Ruggieri L et al. HSV-TK gene transfer into donor lymphocytes for control of allogeneic graft-versus-leukemia. Science 1997; 276: 1719-1724.
2. Tiberghien P, Reynolds CW, Keller J, Spence S, Deschaseaux M, Certoux JM et al. Ganciclovir treatment of herpes simplex thymidine kinase-transduced primary T lymphocytes: An approach for specific in vivo donor T-cell depletion after bone marrow transplantation? Blood 1994; 84: 1333-1341.
3. Introna M, Barbui AM, Bambacioni F, Casati C, Gaipa G, Borleri G et al. Genetic modification of human T cells with CD20: A strategy to purify and lyse transduced cells with anti-CD20 antibodies. Hum Gene Ther 2000; 11: 611-620.
4. Serafini M, Manganini M, Borleri G, Bonamino M, Imberti L, Biondi A et al. Characterization of CD20-transduced T lymphocytes as an alternative suicide gene therapy approach for the treatment of graft-versus-host disease. Hum Gene Ther 2004; 15: 63-76.
5. Cartron G, Watier H, Golay J, Solal-Celigny P. From the bench to the bedside: Ways to improve rituximab efficacy. Blood 2004; 104: 2635-2642.
6. Flieger D, Renoth S, Beier I, Sauerbruch T, Schmidt-Wolf I. Mechanism of cytotoxicity induced by chimeric mouse human monoclonal antibody IDEC-C2B8 in CD20-expressing lymphoma cell lines. Cell Immunol 2000; 204: 55-63.
7. Johnson P, Glennie M. The mechanisms of action of rituximab in the elimination of tumor cells. Semin Oncol 2003; 30: 3-8.
8. Smith MR. Rituximab (monoclonal anti-CD20 antibody): Mechanisms of action and resistance. Oncogene 2003; 22: 7359-7368.
9. Frank O, Rudolph C, Heberlein C, van NN, Schrock E, Schambach A et al. Tumor cells escape suicide gene therapy by genetic and epigenetic instability. Blood 2004; 104: 3543-3549.
10. Hacein-Bey-Abina S, von KC, Schmidt M, McCormack MP, Wulffraat N, Leboulch P et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302: 415-419.
11. Golay J, Lazzari M, Facchinetti V, Bernasconi S, Borleri G, Barbui T et al. CD20 levels determine the in vitro susceptibility to rituximab and complement of B-cell chronic lymphocytec leukemia: Further regulation by CD55 and CD59. Blood 2001; 98: 3383-3389.
12. Donello JE, Loeb JE, Hope TJ. Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J Virol 1998; 72: 5085-5092.
13. Johansen J, Tornoe J, Moller A, Johansen TE. Increased in vitro and in vivo transgene expression levels mediated through cis-acting elements. J Gene Med 2003; 5: 1080-1089.
14. Zufferey R, Donello JE, Trono D, Hope TJ. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 1999; 73: 2886-2892.
15. Emery DW, Yannaki E, Tubb J, Stamatoyannopoulos G. A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl Acad Sci USA 2000; 97: 9150-9155.
16. Emery DW, Yannaki E, Tubb J, Nishino T, Li Q, Stamatoyannopoulos G. Development of virus vectors for gene therapy of beta chain hemoglobinopathies: Flanking with a chromatin insulator reduces gamma-globin gene silencing in vivo. Blood 2002; 100: 2012-2019.
17. Rivella S, Callegari JA, May C, Tan CW, Sadelain M. The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites. J Virol 2000; 74: 4679-4687.
18. Kozak M. The scanning model for translation: An update. J Cell Biol 1989; 108: 229-241.
19. Ramezani A, Hawley TS, Hawley RG. Performance- and safety-enhanced lentiviral vectors containing the human interferon-beta scaffold attachment region and the chicken beta-globin insulator. Blood 2003; 101: 4717-4724.
20. Yannaki E, Tubb J, Aker M, Stamatoyannopoulos G, Emery DW. Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors. Mol Ther 2002; 5: 589-598.
21. Kustikova OS, Wahlers A, Kuhlcke K, Stahle B, Zander AR, Baum C et al. Dose finding with retroviral vectors: Correlation of retroviral vector copy numbers in single cells with gene transfer efficiency in a cell population. Blood 2003; 102: 3934-3937.
22. Li Z, Dullmann J, Schiedlmeier B, Schmidt M, von KC, Meyer J et al. Murine leukemia induced by retroviral gene marking. Science 2002; 296: 497.
23. Cartron G, Dacheux L, Salles G, Solal-Celigny P, Bardos P, Colombat P et al. Therapeutic activity of humanized anti-CD20 monoclonal antibody and polymorphism in IgG Fc receptor FcgammaRIIIa gene. Blood 2002; 99: 754-758.
24. Clynes RA, Towers TL, Presta LG, Ravetch JV. Inhibitory Fc receptors modulate in vivo cytoxicity against tumor targets. Nat Med 2000; 6: 443-446.
25. Pfeiffer M, Stanojevic S, Feuchtinger T, Greil J, Handgretinger R, Barbin K et al. Rituximab mediates in vitro antileukemic activity in pediatric patients after allogeneic transplantation. Bone Marrow Transplant 2005; 36: 91-97.
26. Kingsman SM, Mitrophanous K, Olsen JC. Potential oncogene activity of the woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Therapy 2005; 12: 3-4.
27. Sirma H, Giannini C, Poussin K, Paterlini F, Kremsdorf D, Brechot C. Hepatitis B virus X mutants, present in hepatocellular carcinoma tissue abrogate both the antiproliferative and transactivation effects of HBx. Oncogene 1999; 18: 4848-4859.
28. Poussin K, Dienes H, Sirma H, Urban S, Beaugrand M, Franco D et al. Expression of mutated hepatitis B virus X genes in human hepatocellular carcinomas. Int J Cancer 1999; 80: 497-505.
29. Engels B, Cam H, Schuler T, Indraccolo S, Gladow M, Baum C et al. Retroviral vectors for high-level transgene expression in T lymphocytes. Hum Gene Ther 2003; 14: 1155-1168.
30. Serafini M, Bonamino M, Golay J, Introna M. Elongation factor 1 (EF1alpha) promoter in a lentiviral backbone improves expression of the CD20 suicide gene in primary T lymphocytes allowing efficient rituximab-mediated lysis. Haematologica 2004; 89: 86-95.
31. Kohn DB, Sadelain M, Glorioso JC. Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 2003; 3: 477-488.
32. Baum C, von KC, Staal FJ, Li Z, Fehse B, Schmidt M et al. Chance or necessity? Insertional mutagenesis in gene therapy and its consequences. Mol Ther 2004; 9: 5-13.
33. Kohn DB, Sadelain M, Dunbar C, Bodine D, Kiem HP, Candotti F et al. American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 2003; 8: 180-187.
34. Walters MC, Fiering S, Bouhassira EE, Scalzo D, Goeke S, Magis W et al. The chicken beta-globin 5′HS4 boundary element blocks enhancer-mediated suppression of silencing. Mol Cell Biol 1999; 19: 3714-3726.
35. Schambach A, Wodrich H, Hildinger M, Bohne J, Krausslich HG, Baum C. Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol Ther 2000; 2: 435-445.
36. Auten J, Agarwal M, Chen J, Sutton R, Plavec I. Effect of scaffold attachment region on transgene expression in retrovirus vector-transduced primary T cells and macrophages. Hum Gene Ther 1999; 10: 1389-1399.
37. Kessels HW, van dB, Spits H, Hooijberg E, Schumacher TN. Changing T cell specificity by retroviral T cell receptor display. Proc Natl Acad Sci USA 2000; 97: 14578-14583.
38. Weijtens M, van SA, Hagenbeek A, Braakman E, Martens A. Reduced graft-versus-host disease-inducing capacity of T cells after activation, culturing, and magnetic cell sorting selection in an allogeneic bone marrow transplantation model in rats. Hum Gene Ther 2002; 13: 187-198.
39. Jedema I, van der Werff NM, Barge RM, Willemze R, Falkenburg JH. New CFSE-based assay to determine susceptibility to lysis by cytotoxic T cells of leukemic precursor cells within a heterogeneous target cell population. Blood 2004; 103: 2677-2682.