Naked DNA transfer of factor VIII induced transgene-specific, species-independent immune response in hemophilia a mice

Molecular Therapy

Volumn 10, Issue 1, 2004, Pages 117-126

  Ye, Peiqing ^a   Thompson, Arthur R ^a,b   Sarkar, Rita ^c   Shen, Zhenping ^a   Lillicrap, David P ^d   Kaufman, Randal J ^e   Ochs, Hans D ^a   Rawlings, David J ^a   Miao, Carol H ^a  

^a UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

^b PUGET SOUND BLOOD CENTER   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

^d QUEEN S UNIVERSITY   (Canada)

^e UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

Author keywords

Gene therapy; Hemophilia; Immune response; Inhibitory antibody; Naked DNA transfer

Indexed keywords

ANTIBODY; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; COMPLEMENTARY DNA; CYCLOPHOSPHAMIDE; IMMUNOGLOBULIN G; NAKED DNA; PLASMID VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ANTIBODY TITER; ARTICLE; CONTROLLED STUDY; CORRELATION ANALYSIS; DOG; DOSE RESPONSE; GENE TRANSFER; HEMOPHILIA A; HUMORAL IMMUNITY; IMMUNE RESPONSE; IMMUNOSUPPRESSIVE TREATMENT; MOLECULAR CLONING; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; SIGNAL TRANSDUCTION; TH2 CELL; TRANSGENE;

ANIMALS; ANTIBODY FORMATION; CYCLOPHOSPHAMIDE; DNA; DOGS; FACTOR VIII; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA A; HUMANS; IMMUNOGLOBULIN G; LIVER; MICE; PEPTIDE FRAGMENTS; PLASMIDS; TRANSGENES;

MURINAE;

EID: 3042815553     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.04.009     Document Type: Article

References (43)

1. Hoyer L.W. Scandella D. Factor VIII inhibitors: structure and function in autoantibody and hemophilia A patients Semin. Hematol. 31 1994 1-5
2. Briet E. Mauser-Bunschoten E.P. Revision consensus hemophilia: treatment and responsibility. Nederlandse Vereniging van Hemophilia Patients. Ned. Tijdschr. Geneeskd. 141 1997 2566-2571
3. + T-cell clones specific for wild-type factor VIII: a molecular mechanism responsible for a higher incidence of inhibitor formation in mild/moderate hemophilia A Blood 101 2003 1351-1358
4. Thompson A.R. Structure and function of the factor VIII gene and protein Semin. Thromb. Hemostasis 29 2003 11-22
5. Scandella D. et al. Some factor VIII inhibitor antibodies recognize a common epitope corresponding to C2 domain amino acids 2248 through 2312, which overlap a phospholipid-binding site Blood 86 1995 1811-1819
6. Jacquemin M.G. et al. Mechanism and kinetics of factor VIII inactivation: study with an IgG4 monoclonal antibody derived from a hemophilia A patient with inhibitor Blood 92 1998 496-506
7. Lacroix-Desmazes S. Misra N. Bayry J. Villard S. Kazatchkine M. Kaveri S. Antibodies with hydrolytic activity towards factor VIII in patients with hemophilia A J. Immunol. Methods 269 2002 251
8. Bi L. Lawler A.M. Antonarakis S.E. High K.A. Gearhart J.D. Kazazian H.H. Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A Nat. Genet. 10 1995 119-121
9. Sarkar R. Gao G.P. Chirmule N. Tazelaar J. Kazazian H.H.J. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII Hum. Gene. Ther. 11 2000 881-894
10. Qian J. Collins M. Sharpe A.H. Hoyer L.W. Prevention and treatment of factor VIII inhibitors in murine hemophilia A Blood 95 2000 1324-1329
11. Wu H. et al. Mechanism of the immune response to human factor VIII in murine hemophilia A Thromb. Haemostasis 85 2001 125-133
12. Miao C.H. Ye X. Thompson A.R. High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors Hum. Gene Ther. 14 2003 1297-1305
13. Toole J.J. Pittman D.D. Orr E.C. Murtha P. Wasley L.C. Kaufman R.J. A large region (approximately equal to 95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity Proc. Natl. Acad. Sci. USA 83 1986 5939-5942
14. Notley C. Killoran A. Cameron C. Wynd K. Hough C. Lillicrap D. The canine factor VIII 3′-untranslated region and a concatemeric hepatocyte nuclear factor 1 regulatory element enhance factor VIII transgene expression in vivo Hum. Gene Ther. 13 2002 1583-1593
15. Ye X. Loeb K.R. Stafford D.W. Thompson A.R. Miao C.H. Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid J. Thromb. Haemostasis 1 2003 103-111
16. Liu F. Song Y. Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA Gene Ther. 6 1999 1258-1266
17. Zhang G. Budkar V. Wolff J.A. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA Hum. Gene Ther. 10 1999 1735-1737
18. Miao C.H. et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro Mol. Ther. 1 2000 522-532
19. Kasper C.K. et al. Proceedings: a more uniform measurement of factor VIII inhibitors Thromb. Diath. Haemorrh. 34 1975 612
20. Miao C.H. Thompson A.R. Loeb K. Ye X. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo Mol. Ther. 3 2001 947-957
21. Balague C. et al. Sustained high-level expression of full-length human factor VIII and restoration of clotting activity in hemophilic mice using a minimal adenovirus vector Blood 95 2000 820-828
22. Andrews J.L. Kadan M.J. Gorziglia M.I. Kaleko M. Connelly S. Generation and characterization of E1/E2a/E3/E4-deficient adenoviral vectors encoding human factor VIII Mol. Ther. 3 2001 329-336
23. Brown B.D. Shi C.X. Powell S. Hurlbut D. Graham F.L. Lillicrap D. Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A Blood 103 2004 804-810
24. + T cells specific for factor VIII as a target for specific suppression of inhibitor production Adv. Exp. Med. Biol. 489 2001 119-134
25. Herzog R.W. Mount J.D. Arruda V.R. High K.A. Lothrop C.D. Jr. Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation Mol. Ther. 4 2001 192-200
26. Fields P.A. et al. Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9 Mol. Ther. 4 2001 201-210
27. Fields P.A. et al. Role of vector in activation of T cell subsets in immune responses against the secreted transgene product factor IX Mol. Ther. 1 2000 225-235
28. Herzog R.W. et al. Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy Hum. Gene Ther. 13 2002 1281-1291
29. VandenDriessche T. Collen D. Chuah M.K. Gene therapy for the hemophilias J. Thromb. Haemostasis 1 2003 1550-1558
30. VandenDriessche T. et al. Long-term expression of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VIII-deficient mice Proc. Natl. Acad. Sci. USA 96 1999 10379-10384
31. Chao H. Mao L. Bruce A.T. Walsh C.E. Sustained expression of human factor VIII in mice using a parvovirus-based vector Blood 95 2000 1594-1599
32. Park F. Ohashi K. Kay M.A. Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver Blood 96 2000 1173-1176
33. Chuah M.K. et al. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors Blood 101 2003 1734-1743
34. Kay M.A. et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs Proc. Natl. Acad. Sci. USA 91 1994 2353-2367
35. Dai Y. Schwarz E.M. Gu D. Zhang W.W. Sarvetnick N. Verma I.M. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: tolerization of factor IX and vector antigens allows for long-term expression Proc. Natl. Acad. Sci. USA 92 1995 1401-1405
36. Snyder R.O. Miao C. et al. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors Nat. Med. 5 1999 64-70
37. Miao C.H. et al. Non-random transduction of recombinant adeno-associated viral vectors in mouse hepatocytes in vivo J. Virol. 74 2000 3793-3803
38. Greengard J.S. Jolly P.J. Animal testing of retroviral-mediated gene therapy for factor VIII deficiency Thromb. Haemostasis 82 1999 555-561
39. Stein C.S. et al. In vivo treatment of hemophilia A and mucopolysaccharidosis type VII using nonprimate lentiviral vectors Mol. Ther. 3 2001 850-856
40. Gallo-Penn A.M. et al. Systemic delivery of an adenoviral vector encoding canine factor VIII results in short-term phenotypic correction, inhibitor development, and biphasic liver toxicity in hemophilia A dogs Blood 97 2001 107-113
41. Reddy P.S. et al. Sustained human factor VIII expression in hemophilia A mice following systemic delivery of a gutless adenoviral vector Mol Ther. 5 2002 63-73
42. Rock K.L. Rothstein L. Gamble S. Fleischacker C. Characterization of antigen-presenting cells that present exogenous antigens in association with class I MHC molecules J. Immunol. 150 1993 438-446
43. Carbone F.R. Kurts C. Bennett S.R. Miller J.F. Heath W.R. Cross-presentation: a general mechanism for CTL immunity and tolerance Immunol. Today 19 1998 368-373