Tolerogenic nanoparticles to induce immunologic tolerance: Prevention and reversal of FVIII inhibitor formation

Cellular Immunology

Volumn 301, Issue , 2016, Pages 74-81

  Zhang, Ai Hong ^a   Rossi, Robert J ^a,b   Yoon, Jeongheon ^a   Wang, Hong ^a   Scott, David W ^a  

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

^b UNIVERSITY OF CONNECTICUT HEALTH CENTER   (United States)

Author keywords

FVIII protein; Hemophilia A; Human; Immune tolerance; Nanoparticles; PLGA; Rapamycin

Indexed keywords

BLOOD CLOTTING FACTOR 8 ANTIBODY; IMMUNOGLOBULIN; NANOPARTICLE; RAPAMYCIN; RECOMBINANT BLOOD CLOTTING FACTOR 8; SYNTHETIC VACCINE PARTICLE; UNCLASSIFIED DRUG; BLOOD CLOTTING FACTOR 8; IMMUNOSUPPRESSIVE AGENT; NEUTRALIZING ANTIBODY; RECOMBINANT VACCINE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY TITER; ARTICLE; CONTROLLED STUDY; DRUG EFFICACY; DRUG MEGADOSE; HEMOPHILIA A; HUMORAL IMMUNITY; IMMUNIZATION; IMMUNOLOGICAL TOLERANCE; LOW DRUG DOSE; MOUSE; NANOENCAPSULATION; NONHUMAN; PRIORITY JOURNAL; PROPHYLAXIS; ANIMAL; DISEASE MODEL; IMMUNOLOGY;

ANIMALS; ANTIBODIES, NEUTRALIZING; DISEASE MODELS, ANIMAL; FACTOR VIII; HEMOPHILIA A; IMMUNE TOLERANCE; IMMUNOSUPPRESSIVE AGENTS; MICE; NANOPARTICLES; SIROLIMUS; VACCINES, SYNTHETIC;

EID: 84959145941     PISSN: 00088749     EISSN: 10902163     Source Type: Journal    
DOI: 10.1016/j.cellimm.2015.11.004     Document Type: Article

References (30)

1. Scott D.W., Pratt K.P., Miao C.H. Progress toward inducing immunologic tolerance to factor VIII. Blood 2013, 121:4449-4456.
2. Skupsky J., Saltis M., Song C., Rossi R., Nelson D., Scott D.W. Gene therapy for tolerance and vice versa: a case for hemophilia. Curr. Opin. Mol. Ther. 2010, 12:509-518.
3. Banugaria S.G., Patel T.T., Kishnani P.S. Immune modulation in Pompe disease treated with enzyme replacement therapy. Expert Rev. Clin. Immunol. 2012, 8:497-499.
4. Nechansky A., Kircheis R. Immunogenicity of therapeutics: a matter of efficacy and safety. Expert Opin. Drug Discov. 2010, 5:1067-1079.
5. Hoyer L.W., Hemophilia A. N. Engl. J. Med. 1994, 330:38-47.
6. DiMichele D.M. Immune tolerance in haemophilia: the long journey to the fork in the road. Br. J. Haematol. 2012, 159:123-134.
7. Bluestone J.A., Bour-Jordan H. Current and future immunomodulation strategies to restore tolerance in autoimmune diseases. Cold Spring Harbor Perspect. Biol. 2012, 4.
8. Getts D.R., Turley D.M., Smith C.E., Harp C.T., McCarthy D., Feeney E.M., Getts M.T., Martin A.J., Luo X., Terry R.L., King N.J., Miller S.D. Tolerance induced by apoptotic antigen-coupled leukocytes is induced by PD-L1+ and IL-10-producing splenic macrophages and maintained by T regulatory cells. J. Immunol. 2011, 187:2405-2417.
9. Steinman R.M., Hawiger D., Nussenzweig M.C. Tolerogenic dendritic cells. Annu. Rev. Immunol. 2003, 21:685-711.
10. Maldonado R.A., von Andrian U.H. How tolerogenic dendritic cells induce regulatory T cells. Adv. Immunol. 2010, 108:111-165.
11. Getts D.R., Martin A.J., McCarthy D.P., Terry R.L., Hunter Z.N., Yap W.T., Getts M.T., Pleiss M., Luo X., King J.N., Shea L.D., Miller S.D. Microparticles bearing encephalitogenic peptides induce T-cell tolerance and ameliorate experimental autoimmune encephalomyelitis. Nat. Biotechnol. 2012, 30:1217-1224.
12. Getts D.R., Terry R.L., Getts M.T., Deffrasnes C., Muller M., van Vreden C., Ashhurst T.M., Chami B., McCarthy D., Wu H., Ma J., Martin A., Shae L.D., Witting P., Kansas G.S., Kühn J., Hafezi W., Campbell I.L., Reilly D., Say J. Therapeutic inflammatory monocyte modulation using immune-modifying microparticles. Sci. Transl. Med. 2014, 6:219ra7.
13. Weiner H.L., da Cunha A.P., Quintana F., Wu H. Oral tolerance. Immunol. Rev. 2011, 241:241-259.
14. Lei T.C., Scott D.W. 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, 105:4865-4870.
15. Joseph A., Munroe K., Housman M., Garman R., Richards S. Immune tolerance induction to enzyme-replacement therapy by co-administration of short-term, low-dose methotrexate in a murine Pompe disease model. Clin. Exp. Immunol. 2008, 152:138-146.
16. Selecta Biosciences, Inc., Safety and Pharmacodynamics of SEL-068 Vaccine in Smokers and Non-Smokers. In: ClinicalTrials.gov, 2011.
17. Irvine D.J., Swartz M.A., Szeto G.L. Engineering synthetic vaccines using cues from natural immunity. Nat. Mater. 2013, 12:978-990.
18. Metcalfe S.M., Fahmy T.M. Targeted nanotherapy for induction of therapeutic immune responses. Trends Mol. Med. 2012, 18:72-80.
19. Bachmann M.F., Jennings G.T. Vaccine delivery: a matter of size, geometry, kinetics and molecular patterns. Nat. Rev. Immunol. 2010, 10:787-796.
20. Maldonado R.A., LaMothe R.A., Ferrari J.D., Zhang A.H., Rossi R.J., Griset A.P., O'Neil C., Altreuter D.H., Browning E., Johnston L., Farokhzad O.C., Langer R., Scott D.W., von Andrian U.H., Kishimoto T.K. Polymeric synthetic nanoparticles for the induction of antigen-specific immunological tolerance. Proc. Natl. Acad. Sci. USA 2015, 112. E156-65.
21. Taner T., Hackstein H., Wang Z., Morelli A.E., Thomson A.W. Rapamycin-treated, alloantigen-pulsed host dendritic cells induce ag-specific T cell regulation and prolong graft survival. Am. J. Transplant. 2005, 5:228-236.
22. null mice. Mol. Ther. 2014, 22:169-177.
23. Kappler J.W., Hoffmann M., Dutton R.W. Regulation of the immune response. I. Differential effect of passively administered antibody on the thymus-derived and bone marrow-derived lymphocytes. J. Exp. Med. 1971, 134:577-587.
24. Qian J., Collins M., Sharpe A.H., Hoyer L.W. Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood 2000, 95:1324-1329.
25. Skupsky J., Zhang A.H., Su Y., Scott D.W. A role for thrombin in the initiation of the immune response to therapeutic factor VIII. Blood 2009, 114:4741-4748.
26. Jeejeebhoy H.F. Immunological studies on the rat thymectomized in adult life. Immunology 1965, 9:417-425.
27. Bi L., Lawler A.M., Antonarakis S.E., High K.A., Gearhart J.D., Kazazian H.H. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat. Genet. 1995, 10:119-121.
28. Danhier F., Ansorena E., Silva J.M., Coco R., Le Breton A., Preat V. PLGA-based nanoparticles: an overview of biomedical applications. J. Control. Release 2012, 161:505-522.
29. Moghimi B., Sack B.K., Nayak S., Markusic D.M., Mah C.S., Herzog R.W. Tolerance induction to Factor VIII by transient co-administration with rapamycin. J. Thromb. Haemost. 2011, 9:1524-1533.
30. Nayak S., Cao O., Hoffman B.E., Cooper M., Zhou S., Atkinson M.A., Herzog R.W. Prophylactic immune tolerance induced by changing the ratio of antigen-specific effector to regulatory T cells. J. Thromb. Haemost. 2009, 7:1523-1532.