Downregulation of CD40 signal and induction of TGF-β by phosphatidylinositol mediates reduction in immunogenicity against recombinant human Factor VIII

Journal of Pharmaceutical Sciences

Volumn 101, Issue 1, 2012, Pages 48-55

  Gaitonde, Puneet ^a   Peng, Aaron ^a   Straubinger, Robert M ^a   Bankert, Richard B ^b   Balu iyer, Sathy V ^a  

^a UNIVERSITY AT BUFFALO   (United States)

^b UNIVERSITY AT BUFFALO   (United States)

Author keywords

Biotechnology; Cell culture; Co stimulatory signal; Dendritic cell; Immunogenicity; Immunology; Nanoparticle; Phospholipids; Therapeutic protein; Transforming Growth Factor

Indexed keywords

CD40 ANTIGEN; INTERLEUKIN 10; INTERLEUKIN 17; INTERLEUKIN 6; PHOSPHATIDYLINOSITOL; RECOMBINANT BLOOD CLOTTING FACTOR 8; TRANSFORMING GROWTH FACTOR BETA1;

ANIMAL EXPERIMENT; ANTIBODY RESPONSE; ARTICLE; CELL CULTURE; CONTROLLED STUDY; CYTOKINE RELEASE; DENDRITIC CELL; DOWN REGULATION; FEMALE; IMMUNOGENICITY; IN VITRO STUDY; MOUSE; NONHUMAN; PHENOTYPE;

EID: 82255175795     PISSN: 00223549     EISSN: 15206017     Source Type: Journal    
DOI: 10.1002/jps.22746     Document Type: Article

References (31)

1. Peng A, Straubinger RM, Balu-Iyer SV. 2010. Phosphatidylinositol containing lipidic particles reduces immunogenicity and catabolism of factor VIII in hemophilia a mice. AAPS J 12(3):473-481.
2. Foster PA, Zimmerman TS. 1989. Factor VIII structure and function. Blood Rev 3(3):180-191.
3. Fay PJ. 1993. Factor VIII structure and function. Thromb Haemost 70(1):63-67.
4. Klinge J, Ananyeva NM, Hauser CA, Saenko EL. 2002. Hemophilia A-From basic science to clinical practice. Semin Thromb Hemost 28(3):309-322.
5. Fijnvandraat K, Bril WS, Voorberg J. 2003. Immunobiology of inhibitor development in hemophilia A. Semin Thromb Hemost 29(1):61-68.
6. Lollar P. 2002. Molecular characterization of the immune response to factor VIII. Vox Sang 83 (Suppl 1):403-408.
7. Ferraiolo BL, Mohler MA, Gloff CA. 1992. Protein pharmacokinetics and metabolism. New York: Plenum.
8. Schellekens H. 2002. Immunogenicity of therapeutic proteins: Clinical implications and future prospects. Clin Ther 24(11):1720-1740.
9. Braeckman R. 2000. Pharmacokinetics and pharmacodynamics of protein therapeutics. In Peptide and protein drug analysis; Reid RE, Ed. New York: Marcel Dekker, Inc., pp 633-669.
10. Braun A, Kwee L, Labow MA, Alsenz J. 1997. Protein aggregates seem to play a key role among the parameters influencing the antigenicity of interferon alpha (IFN-alpha) in normal and transgenic mice. Pharm Res 14(10):1472-1478.
11. Schernthaner G. 1993. Immunogenicity and allergenic potential of animal and human insulins. Diabetes Care. 16(Suppl 3):155-165.
12. Tang L, Persky AM, Hochhaus G, Meibohm B. 2004. Pharmacokinetic aspects of biotechnology products. J Pharm Sci 93(9):2184-2204.
13. Chirino AJ, Ary ML, Marshall SA. 2004. Minimizing the immunogenicity of protein therapeutics. Drug Discov Today 9(2):82-90.
14. Bartlett GR. 1959. Phosphorus assay in column chromatography. J Biol Chem 234(3):466-468.
15. Lutz MB, Kukutsch N, Ogilvie AL, Rossner S, Koch F, Romani N, Schuler G. 1999. An advanced culture method for generating large quantities of highly pure dendritic cells from mouse bone marrow. J Immunol Methods 223(1):77-92.
16. Straubinger RM, Papahadjopoulos D, Hong KL. 1990. Endocytosis and intracellular fate of liposomes using pyranine as a probe. Biochemistry 29(20):4929-4939.
17. Gabizon A, Shiota R, Papahadjopoulos D. 1989. Pharmacokinetics and tissue distribution of doxorubicin encapsulated in stable liposomes with long circulation times. J Natl Cancer Inst 81(19):1484-1488.
18. Dasgupta S, Navarrete AM, Andre S, Wootla B, Delignat S, Repesse Y, Bayry J, Nicoletti A, Saenko EL, d'Oiron R, Jacquemin M, Saint-Remy JM, Kaveri SV, Lacroix-Desmazes S. 2008. Factor VIII bypasses CD91/LRP for endocytosis by dendritic cells leading to T-cell activation. Haematologica 93(1):83-89.
19. Foged C, Arigita C, Sundblad A, Jiskoot W, Storm G, Frokjaer S. 2004. Interaction of dendritic cells with antigen-containing liposomes: Effect of bilayer composition. Vaccine 22(15-16):1903-1913.
20. Waters B, Lillicrap D. 2009. The molecular mechanisms of immunomodulation and tolerance induction to factor VIII. J Thromb Haemost 7(9):1446-1456.
21. Skupsky J, Zhang AH, Su Y, Scott DW. 2009. A role for thrombin in the initiation of the immune response to therapeutic Factor VIII. Blood 114:4741-4748.
22. Qadura M, Othman M, Waters B, Chegeni R, Walker K, Labelle A, Ozelo M, Hough C, Lillicrap D. 2008. Reduction of the immune response to factor VIII mediated through tolerogenic factor VIII presentation by immature dendritic cells. J Thromb Haemost 6(12):2095-2104.
23. Rossi G, Sarkar J, Scandella D. 2001. Long-term induction of immune tolerance after blockade of CD40-CD40L interaction in a mouse model of hemophilia A. Blood 97(9):2750-2757.
24. Ewenstein BM, Hoots WK, Lusher JM, DiMichele D, White GC, 2nd, Adelman B, Nadeau K.. 2000. Inhibition of CD40 ligand (CD154) in the treatment of factor VIII inhibitors. Haematologica 85(10 Suppl):35-39.
25. Qadura M, Waters B, Burnett E, Chegeni R, Hough C, Othman M, Lillicrap D.. 2010. Immunoglobulin isotypes and functional anti-FVIII antibodies in response to FVIII treatment in Balb/c and C57BL/6 haemophilia A mice. Haemophilia 17:288-295.
26. Li MO, Flavell RA. 2008. TGF-beta: A master of all T cell trades. Cell 134(3):392-404.
27. Chen X, Doffek K, Sugg SL, Shilyansky J. 2004. Phosphatidylserine regulates the maturation of human dendritic cells. J Immunol 173(5):2985-2994.
28. Caselli E, Baricordi OR, Melchiorri L, Bellini F, Ponzin D, Bruni A. 1992. Inhibition of DNA synthesis in peripheral blood mononuclear cells treated with phosphatidylserines containing unsaturated acyl chains. Immunopharmacology 23(3):205-213.
29. Morelli AE, Thomson AW. 2007. Tolerogenic dendritic cells and the quest for transplant tolerance. Nat Rev Immunol 7(8):610-621.
30. Wan YY, Flavell RA. 2008. TGF-beta and regulatory T cell in immunity and autoimmunity. J Clin Immunol 28(6):647-659.
31. Horwitz DA, Zheng SG, Gray JD. 2008. Natural and TGF-beta-induced Foxp3(+)CD4(+) CD25(+) regulatory T cells are not mirror images of each other. Trends Immunol 29(9):429-435.