FVIII-specific human chimeric antigen receptor T-regulatory cells suppress T- and B-cell responses to FVIII

Blood

Volumn 129, Issue 2, 2017, Pages 238-245

  Yoon, Jeongheon ^a   Schmidt, Anja ^b   Zhang, Ai Hong ^a   Königs, Christoph ^b   Kim, Yong Chan ^a   Scott, David W ^a  

^a Uniformed Services University of the Health Sciences   (United States)

^b UNIVERSITY HOSPITAL FRANKFURT   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; CHIMERIC ANTIGEN RECEPTOR; SINGLE CHAIN FRAGMENT VARIABLE ANTIBODY; LYMPHOCYTE ANTIGEN RECEPTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANTIBODY RESPONSE; ARTICLE; B LYMPHOCYTE; CELL ACTIVATION; CELL ENGINEERING; CELL PROLIFERATION; CONTROLLED STUDY; FEMALE; GENETIC TRANSDUCTION; HEMOPHILIA A; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; MALE; MOUSE; NONHUMAN; NORMAL HUMAN; PHAGE DISPLAY; PRIORITY JOURNAL; REGULATORY T LYMPHOCYTE; SPLEEN CELL; T LYMPHOCYTE; ANIMAL; FLOW CYTOMETRY; IMMUNOLOGY; IMMUNOSUPPRESSIVE TREATMENT; KNOCKOUT MOUSE; LYMPHOCYTE ACTIVATION; PROCEDURES;

ANIMALS; B-LYMPHOCYTES; CELL ENGINEERING; FACTOR VIII; FLOW CYTOMETRY; HUMANS; IMMUNOSUPPRESSION; LYMPHOCYTE ACTIVATION; MICE; MICE, KNOCKOUT; RECEPTORS, ANTIGEN, T-CELL; T-LYMPHOCYTES, REGULATORY; TRANSDUCTION, GENETIC;

EID: 85018027174     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2016-07-727834     Document Type: Article

References (21)

1. Mannucci PM, Tuddenham EG. The hemophilias–from royal genes to gene therapy. N Engl J Med. 2001;344(23):1773-1779.
2. Iorio A, Halimeh S, Holzhauer S, et al. Rate of inhibitor development in previously untreated hemophilia A patients treated with plasma-derived or recombinant factor VIII concentrates: a systematic review. J Thromb Haemost. 2010;8(6): 1256-1265.
3. Trzonkowski P, Bieniaszewska M, Ju´sci´nska J, et al. First-in-man clinical results of the treatment of patients with graft versus host disease with human ex vivo expanded CD41CD251CD127-T regulatory cells. Clin Immunol. 2009;133(1): 22-26.
4. Brunstein CG, Miller JS, Cao Q, et al. Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood. 2011;117(3): 1061-1070.
5. Brunstein CG, Blazar BR, Miller JS, et al. Adoptive transfer of umbilical cord blood-derived regulatory T cells and early viral reactivation. Biol Blood Marrow Transplant. 2013;19(8):1271-1273.
6. Kim YC, Zhang AH, Su Y, et al. Engineered antigen-specific human regulatory T cells: immunosuppression of FVIII-specific T- and B-cell responses. Blood. 2015;125(7):1107-1115.
7. Maher J, Brentjens RJ, Gunset G, Rivière I, Sadelain M. Human T-lymphocyte cytotoxicity and proliferation directed by a single chimeric TCRzeta /CD28 receptor. Nat Biotechnol. 2002; 20(1):70-75.
8. MacDonald KG, Hoeppli RE, Huang Q, et al. Alloantigen-specific regulatory T cells generated with a chimeric antigen receptor. J Clin Invest. 2016;126(4):1413-1424.
9. Naumann A, Scherger AK, Neuwirth J, et al. Selection and characterisation of FVIII-specific single chain variable fragments. Hamostaseologie. 2013;33(Suppl 1):S39-S45.
10. Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet. 1995;10(1):119-121.
11. Greeley E, Berke G, Scott DW. Receptors for antigen on lymphoid cells. I. Immunoadsorption of plaque-forming cells to poly-L-lysine-fixed antigen monolayers. J Immunol. 1974;113(6):1883-1890.
12. Lei TC, Scott DW. Induction of tolerance to factor VIII inhibitors by gene therapy with immunodominant A2 and C2 domains presented by B cells as Ig fusion proteins. Blood. 2005; 105(12):4865-4870.
13. Stockis J, Colau D, Coulie PG, Lucas S. Membrane protein GARP is a receptor for latent TGF-beta on the surface of activated human Treg. Eur J Immunol. 2009;39(12):3315-3322.
14. Healey JF, Parker ET, Barrow RT, Langley TJ, Church WR, Lollar P. The humoral response to human factor VIII in hemophilia A mice. J Thromb Haemost. 2007;5(3):512-519.
15. Trzonkowski P, Bacchetta R, Battaglia M, et al. Hurdles in therapy with regulatory T cells. Sci Transl Med. 2015;7(304):304ps18.
16. Herold KC, Vignali DA, Cooke A, Bluestone JA. Type 1 diabetes: translating mechanistic observations into effective clinical outcomes. Nat Rev Immunol. 2013;13(4):243-256.
17. Bluestone JA, Buckner JH, Fitch M, et al. Type 1 diabetes immunotherapy using polyclonal regulatory T cells. Sci Transl Med. 2015;7(315): 315ra189.
18. Thornton AM, Shevach EM. Suppressor effector function of CD41CD251 immunoregulatory T cells is antigen nonspecific. J Immunol. 2000; 164(1):183-190.
19. Sarkar D, Biswas M, Liao G, et al. Ex Vivo Expanded Autologous Polyclonal Regulatory T Cells Suppress Inhibitor Formation in Hemophilia. Mol Ther Methods Clin Dev. 2014;1:1.
20. Ellebrecht CT, Bhoj VG, Nace A, et al. Reengineering chimeric antigen receptor T cells for targeted therapy of autoimmune disease. Science. 2016;353(6295):179-184.
21. 10.1182/blood-2016-07-727834