Suppression of the immune response to FVIII in hemophilia a mice by transgene modified tolerogenic dendritic cells

Molecular Therapy

Volumn 19, Issue 10, 2011, Pages 1896-1904

  Su, Rui Jun ^a   Epp, Angela ^a   Feng, Junli ^a   Roy, Jackie ^a   Latchman, Yvette ^a   Wu, Xiaoping ^a   Bolgiano, Doug ^a   Josephson, Neil C ^a,b  

^a PUGET SOUND BLOOD CENTER   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; INTERLEUKIN 10; RECOMBINANT BLOOD CLOTTING FACTOR 8; VIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY TITER; ANTIGEN SPECIFICITY; ARTICLE; BIOENGINEERING; CD4+ T LYMPHOCYTE; CELL BASED GENE THERAPY; CONTROLLED STUDY; DENDRITIC CELL; HEMOPHILIA A; IMMUNE RESPONSE; IMMUNIZATION; IMMUNOSUPPRESSIVE TREATMENT; LYMPHOCYTE PROLIFERATION; MOUSE; MULTIPLE CYCLE TREATMENT; NONHUMAN; PHENOTYPE; REGULATORY T LYMPHOCYTE; SPLEEN CELL; T LYMPHOCYTE; TOLEROGENIC DENDRITIC CELL; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRUS EXPRESSION;

HUMAN SPUMARETROVIRUS; MUS;

EID: 80053571363     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.134     Document Type: Article

References (42)

1. Lusher, J, Abildgaard, C, Arkin, S, Mannucci, PM, Zimmermann, R, Schwartz, L et al. (2004). Human recombinant DNA-derived antihemophilic factor in the treatment of previously untreated patients with hemophilia A: final report on a hallmark clinical investigation. J Thromb Haemost 2: 574-583. (Pubitemid 40185680)
2. Scharrer, I, Bray, GL and Neutzling, O (1999). Incidence of inhibitors in haemophilia A patients-a review of recent studies of recombinant and plasma-derived factor VIII concentrates. Haemophilia 5: 145-154. (Pubitemid 29326763)
3. Morfini, M, Haya, S, Tagariello, G, Pollmann, H, Quintana, M, Siegmund, B et al. (2007). European study on orthopaedic status of haemophilia patients with inhibitors. Haemophilia 13: 606-612. (Pubitemid 47338004)
4. Colowick, AB, Bohn, RL, Avorn, J and Ewenstein, BM (2000). Immune tolerance induction in hemophilia patients with inhibitors: costly can be cheaper. Blood 96: 1698-1702. (Pubitemid 30661051)
5. Darby, SC, Keeling, DM, Spooner, RJ, Wan Kan, S, Giangrande, PL, Collins, PW et al.; UK Haemophilia Centre Doctors' Organisation. (2004). The incidence of factor VIII and factor IX inhibitors in the hemophilia population of the UK and their effect on subsequent mortality, 1977-99. J Thromb Haemost 2: 1047-1054.
6. Dimichele, D (2007). Immune tolerance therapy for factor VIII inhibitors: moving from empiricism to an evidence-based approach. J Thromb Haemost 5 Suppl 1: 143-150. (Pubitemid 46958827)
7. Probst, HC, Lagnel, J, Kollias, G and van den Broek, M (2003). Inducible transgenic mice reveal resting dendritic cells as potent inducers of CD8+ T cell tolerance. Immunity 18: 713-720. (Pubitemid 36588587)
8. Steinman, RM, Hawiger, D and Nussenzweig, MC (2003). Tolerogenic dendritic cells. Annu Rev Immunol 21: 685-711. (Pubitemid 37174545)
9. Joffre, O, Nolte, MA, Spörri, R and Reis e Sousa, C (2009). Inflammatory signals in dendritic cell activation and the induction of adaptive immunity. Immunol Rev 227: 234-247.
10. Morelli, AE and Thomson, AW (2007). Tolerogenic dendritic cells and the quest for transplant tolerance. Nat Rev Immunol 7: 610-621. (Pubitemid 47123548)
11. Doan, T, McNally, A, Thomas, R and Steptoe, RJ (2009). Steady-state dendritic cells continuously inactivate T cells that escape thymic negative selection. Immunol Cell Biol 87: 615-622.
12. Hagymasi, AT, Slaiby, AM, Mihalyo, MA, Qui, HZ, Zammit, DJ, Lefrancois, L et al. (2007). Steady state dendritic cells present parenchymal self-antigen and contribute to, but are not essential for, tolerization of naive and Th1 effector CD4 cells. J Immunol 179: 1524-1531.
13. Coates, PT, Duncan, FJ, Colvin, BL, Wang, Z, Zahorchak, AF, Shufesky, WJ et al. (2004). In vivo-mobilized kidney dendritic cells are functionally immature, subvert alloreactive T cell responses, and prolong organ allograft survival. Transplantation 77: 1080-1089. (Pubitemid 38508927)
14. Eto, M, Hackstein, H, Kaneko, K, Nomoto, K and Thomson, AW (2002). Promotion of skin graft tolerance across MHC barriers by mobilization of dendritic cells in donor hemopoietic cell infusions. J Immunol 169: 2390-2396. (Pubitemid 34920981)
15. Fu, F, Li, Y, Qian, S, Lu, L, Chambers, F, Starzl, TE et al. (1996). Costimulatory molecule-deficient dendritic cell progenitors (MHC class II+, CD80dim, CD86-) prolong cardiac allograft survival in nonimmunosuppressed recipients. Transplantation 62: 659-665. (Pubitemid 26313912)
16. Lutz, MB, Suri, RM, Niimi, M, Ogilvie, AL, Kukutsch, NA, Rössner, S et al. (2000). Immature dendritic cells generated with low doses of GM-CSF in the absence of IL-4 are maturation resistant and prolong allograft survival in vivo. Eur J Immunol 30: 1813-1822. (Pubitemid 30456177)
17. Rastellini, C, Lu, L, Ricordi, C, Starzl, TE, Rao, AS and Thomson, AW (1995). Granulocyte/macrophage colony-stimulating factor-stimulated hepatic dendritic cell progenitors prolong pancreatic islet allograft survival. Transplantation 60: 1366-1370. (Pubitemid 26005300)
18. Bériou, G, Pêche, H, Guillonneau, C, Merieau, E and Cuturi, MC (2005). Donorspecific allograft tolerance by administration of recipient-derived immature dendritic cells and suboptimal immunosuppression. Transplantation 79: 969-972. (Pubitemid 40586872)
19. Hadeiba, H, Sato, T, Habtezion, A, Oderup, C, Pan, J and Butcher, EC (2008). CCR9 expression defines tolerogenic plasmacytoid dendritic cells able to suppress acute graft-versus-host disease. Nat Immunol 9: 1253-1260.
20. Sato, K, Eizumi, K, Fukaya, T, Fujita, S, Sato, Y, Takagi, H et al. (2009). Naturally occurring regulatory dendritic cells regulate murine cutaneous chronic graft-versushost disease. Blood 113: 4780-4789.
21. Turnquist, HR, Raimondi, G, Zahorchak, AF, Fischer, RT, Wang, Z and Thomson, AW (2007). Rapamycin-conditioned dendritic cells are poor stimulators of allogeneic CD4+ T cells, but enrich for antigen-specific Foxp3+ T regulatory cells and promote organ transplant tolerance. J Immunol 178: 7018-7031. (Pubitemid 46847428)
22. Delgado, M, Gonzalez-Rey, E and Ganea, D (2005). The neuropeptide vasoactive intestinal peptide generates tolerogenic dendritic cells. J Immunol 175: 7311-7324. (Pubitemid 41713427)
23. Penna, G, Amuchastegui, S, Giarratana, N, Daniel, KC, Vulcano, M, Sozzani, S et al. (2007). 1,25-Dihydroxyvitamin D3 selectively modulates tolerogenic properties in myeloid but not plasmacytoid dendritic cells. J Immunol 178: 145-153. (Pubitemid 46033595)
24. Wakkach, A, Fournier, N, Brun, V, Breittmayer, JP, Cottrez, F and Groux, H (2003). Characterization of dendritic cells that induce tolerance and T regulatory 1 cell differentiation in vivo. Immunity 18: 605-617. (Pubitemid 36588578)
25. Miao, HZ, Sirachainan, N, Palmer, L, Kucab, P, Cunningham, MA, Kaufman, RJ et al. (2004). Bioengineering of coagulation factor VIII for improved secretion. Blood 103: 3412-3419. (Pubitemid 38525672)
26. Tang, Q and Bluestone, JA (2008). The Foxp3+ regulatory T cell: a jack of all trades, master of regulation. Nat Immunol 9: 239-244. (Pubitemid 351267321)
27. Vignali, DA, Collison, LW and Workman, CJ (2008). How regulatory T cells work. Nat Rev Immunol 8: 523-532.
28. Su, RJ, Epp, A, Latchman, Y, Bolgiano, D, Pipe, SW and Josephson, NC (2010). Suppression of FVIII inhibitor formation in hemophilic mice by delivery of transgene modified apoptotic fibroblasts. Mol Ther 18: 214-222.
29. Qadura, M, Othman, M, Waters, B, Chegeni, R, Walker, K, Labelle, A et al. (2008). Reduction of the immune response to factor VIII mediated through tolerogenic factor VIII presentation by immature dendritic cells. J Thromb Haemost 6: 2095-2104.
30. Ragni, MV, Wu, W, Liang, X, Hsieh, CC, Cortese-Hassett, A and Lu, L (2009). Factor VIII-pulsed dendritic cells reduce anti-factor VIII antibody formation in the hemophilia A mouse model. Exp Hematol 37: 744-754.
31. Saraiva, M and O'Garra, A (2010). The regulation of IL-10 production by immune cells. Nat Rev Immunol 10: 170-181.
32. Kim, SH, Kim, S, Oligino, TJ and Robbins, PD (2002). Effective treatment of established mouse collagen-induced arthritis by systemic administration of dendritic cells genetically modified to express FasL. Mol Ther 6: 584-590. (Pubitemid 35363690)
33. Morita, Y, Yang, J, Gupta, R, Shimizu, K, Shelden, EA, Endres, J et al. (2001). Dendritic cells genetically engineered to express IL-4 inhibit murine collagen-induced arthritis. J Clin Invest 107: 1275-1284. (Pubitemid 32494544)
34. Terness, P, Bauer, TM, Röse, L, Dufter, C, Watzlik, A, Simon, H et al. (2002). Inhibition of allogeneic T cell proliferation by indoleamine 2,3-dioxygenase-expressing dendritic cells: mediation of suppression by tryptophan metabolites. J Exp Med 196: 447-457.
35. Sato, K, Yamashita, N, Yamashita, N, Baba, M and Matsuyama, T (2003). Regulatory dendritic cells protect mice from murine acute graft-versus-host disease and leukemia relapse. Immunity 18: 367-379. (Pubitemid 36351516)
36. Woltman, AM, de Fijter, JW, Kamerling, SW, Paul, LC, Daha, MR and van Kooten, C (2000). The effect of calcineurin inhibitors and corticosteroids on the differentiation of human dendritic cells. Eur J Immunol 30: 1807-1812. (Pubitemid 30456176)
37. Miller, G, Pillarisetty, VG, Shah, AB, Lahrs, S and DeMatteo, RP (2003). Murine Flt3 ligand expands distinct dendritic cells with both tolerogenic and immunogenic properties. J Immunol 170: 3554-3564. (Pubitemid 36359222)
38. Josephson, NC, Vassilopoulos, G, Trobridge, GD, Priestley, GV, Wood, BL, Papayannopoulou, T et al. (2002). Transduction of human NOD/SCID-repopulating cells with both lymphoid and myeloid potential by foamy virus vectors. Proc Natl Acad Sci USA 99: 8295-8300. (Pubitemid 34651046)
39. Trobridge, GD, Miller, DG, Jacobs, MA, Allen, JM, Kiem, HP, Kaul, R et al. (2006). Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci USA 103: 1498-1503. (Pubitemid 43191194)
40. Wu, H, Reding, M, Qian, J, Okita, DK, Parker, E, Lollar, P et al. (2001). Mechanism of the immune response to human factor VIII in murine hemophilia A. Thromb Haemost 85: 125-133. (Pubitemid 32050954)
41. Yang, L, Yang, H, Rideout, K, Cho, T, Joo, KI, Ziegler, L et al. (2008). Engineered lentivector targeting of dendritic cells for in vivo immunization. Nat Biotechnol 26: 326-334. (Pubitemid 351355129)
42. Kasper, CK, Aledort, L, Aronson, D, Counts, R, Edson, JR, van Eys, J et al. (1975). Proceedings: A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 34: 612.