Complete short-term correction of canine hemophilia A by in vivo gene therapy

Blood

Volumn 88, Issue 10, 1996, Pages 3846-3853

  Connelly, Sheila ^b   Mount, Jane ^b   Mauser, Amy ^b   Gardner, Joann M ^b   Kaleko, Michael ^b   McClelland, Alan ^b   Lothrop Jr , Clinton D ^a  

^a AUBURN UNIVERSITY   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; BLEEDING TIME; BLOOD CLOTTING DISORDER; BLOOD CLOTTING TIME; CONTROLLED STUDY; DOG; GENE EXPRESSION; GENE THERAPY; GENETIC TRANSDUCTION; HEMOPHILIA A; NONHUMAN; PARTIAL THROMBOPLASTIN TIME; PHENOTYPE; PRIORITY JOURNAL;

EID: 0029850534     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v88.10.3846.bloodjournal88103846     Document Type: Article

References (52)

1. Sadler JE, Davie EW: Hemophilia A, hemophilia B, and von Willebrand's disease, in Nienhuis AW, Leder P, Majerus PW (eds): The Molecular Basis of Blood Diseases. New York, NY, Saunders, 1987, p 575
2. Rosendaal FR, Smit C, Briët E: Hemophilia treatment in historical perspective: A review of medical and social developments. Ann Hematol 62:5, 1991
3. Lozier JN, Brinkhous KM: Gene therapy and the hemophilias. JAMA 271:47, 1994
4. Fallaux FJ, Hoeben RC, Briët E: State and prospects of gene therapy for the hemophilias. Thromb Haemost 74:263, 1995
5. Hoeben RC: Gene therapy for the haemophilias: Current status. Biologicals 23:27, 1995
6. Zatloukal K, Cotten M, Berger M, Schmidt W, Wagner E, Birnstiel ML: In vivo production of human factor VIII in mice after intrasplenic implantation of primary fibroblasts transfected by receptor-mediated, adenovirus-augmented gene delivery. Proc Natl Acad Sci USA 91:5148, 1994
7. Dwarki VJ, Belloni P, Nijjar T, Smith J, Couto L, Rabier M, Clift S, Berns A, Cohen LK: Gene therapy for hemophilia A: Production of therapeutic levels of human factor VIII in vivo in mice. Proc Natl Acad Sci USA 92:1023, 1995
8. Kaufman RJ: Biological regulation of factor VIII activity. Annu Rev Med 43:325, 1992
9. Kaufman RJ, Wasley LC, Davies MV, Wise RJ, Israel DI, Dorner AJ: Effect of von Willebrand factor coexpression on the synthesis and secretion of factor VIII in Chinese hamster ovary cells. Mol Cell Biol 9:1233, 1989
10. Lynch CM, Israel DI, Kaufman RJ, Miller AD: Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther 4:259, 1993
11. Hoeben RC, Fallaux FJ, Cramer SJ, van den Wollenberg JM, Van Ormondt H, Briët E, Van Der Eb AJ: Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region. Blood 85:2447, 1995
12. Dorner AJ, Wasley LC, Kaufman RJ: Overexpression of GRP78 mitigates stress induction of glucose regulated proteins and blocks secretion of selective proteins in Chinese hamster ovary cells. EMBO J 11:1563, 1992
13. Chuah MKL, Vandendriessche T, Morgan RA: Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A. Hum Gene Ther 6:1363, 1995
14. Kaufman RJ, Wasley LC, Dorner AJ: Synthesis, processing, and secretion of recombinant human factor VIII expressed in mammalian cells. J Biol Chem 263:6352, 1988
15. Pittman DD, Alderman EM, Tomkinson KN, Wang JH, Giles AR, Kaufman RJ: Biochemical, Immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 81:2925, 1993
16. Israel DI, Kaufman RJ: Retroviral-mediated transfer and amplification of a functional human factor VIII gene. Blood 75:1074, 1990
17. Hoeben RC, van der Jagt RC, Schoute F, Van Tilburg NH, Verbeet MP, Briët E, Van Ormondt H, Van Der Eb AJ: Expression of functional factor VIII in primary human skin fibroblasts after retrovirus-mediated gene transfer. J Biol Chem 265:7318, 1990
18. Hoeben RC, Einerhand MP, Briët E, Van Ormondt H, Valerio D, Van Der Eb AJ: Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 67:341, 1992
19. Hoeben RC, Fallaux FJ, Van Tilburg NH, Cramer SJ, Van Ormondt H, Briët E, Van Der Eb AJ: Toward gene therapy for hemophilia A: Long-term persistence of factor VIII-secreting fibroblasts after transplantation into immunodeficient mice. Hum Gene Ther 4:179, 1993
20. Connelly S, Smith TAG, Dhir G, Gardner JM, Mehaffey MG, Zaret KS, McClelland A, Kaleko M: In vivo gene delivery and expression of physiological levels of functional human factor VIII in mice. Hum Gene Ther 6:185, 1995
21. Connelly S, Gardner JM, McClelland A, Kaleko M: High level tissue-specific expression of functional human FVIII in mice. Hum Gene Ther 7:183, 1996
22. Connelly S, Gardner JM, Lyons RM, McClelland A, Kaleko M: Sustained expression of therapeutic levels of human factor VIII in mice. Blood 87:4671, 1996
23. Jaffe HA, Danel C, Longenecker G, Metzger M, Setoguchi Y, Rosenfeld MA, Gant TW, Thorgeirsson SS, Stratford Perricaudet LD, Perricaudet M, Pavicani A, Lecocq JP, Crystal RG: Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat Genet 1:372, 1992
24. Smith TAG, Mehaffey MG, Kayda DB, Saunders JM, Yei S, Trapnell BC, McClelland A, Kaleko M: Adenovirus-mediated expression of therapeutic plasma levels of human factor IX in mice. Nat Genet 5:397, 1993
25. Graham JB, Buckwalter JA, Hartley LJ, Brinkhous KM: Canine hemophilia: Observations on the course, the clotting anomaly, and the effects of blood transfusion. J Exp Med 90:97, 1949
26. Giles AR, Tinlin S, Greenwood R: A canine model of hemophilic (factor VIII:C deficiency) bleeding. Blood 60:727, 1982
27. Kingdon HS, Hassell TM: Hemophilic dog model for evaluating therapeutic effectiveness of plasma protein fractions. Blood 58:969, 1981
28. Brinkhous KM, Sandberg H, Garris JB, Mattson C, Palm M, Griggs T, Read MS: Purified human factor VIII procoagulant protein: Comparative hemostatic response after infusions into hemophilic and von Willebrand disease dogs. Proc Natl Acad Sci USA 82:8752, 1985
29. Giles AR, Tinlin S, Hoogendoorn H, Fournel MA, Ng P, Pancham N: In vivo characterization of recombinant factor VIII in a canine model of hemophilia A (factor VIII deficiency). Blood 72:335, 1988
30. Nichols T, Bellinger DA, Reddick RL, Smith SV, Koch GG, Davis K, Sigman J, Brinkhous KM, Griggs TR, Read MS: The roles of von Willebrand factor and factor VIII in arterial thrombosis: Studies in canine von Willebrand disease and hemophilia A. Blood 81:2644, 1993
31. Littlewood JD, Barrowcliffe TW: The development and characterisation of antibodies to human factor VIII in haemophilic dogs. Thromb Haemost 57:314, 1987
32. Tinlin S, Webster S, Giles AR: The development of homologous (canine/anti-canine) antibodies in dogs with haemophilia A (factor VIII deficiency): A ten-year longitudinal study. Thromb Haemost 69:21, 1993
33. Aledort L: Inhibitors in hemophilia patients: Current status and management. Am J Hematol 47:208, 1994
34. Mount JM: Gene therapy of canine hemophilia A. Masters thesis, Auburn University, Auburn, AL, 1995
35. Tolstoshev P, Mittereder N, Yei S, Trapnell BC: Adenovirus vectors for gene therapy, in Cancer Chemotherapy: Challenges for the Future. Tokyo, Japan, Excerpta Medica, 1994, p 23
36. 1-antitrypsin in dogs after autologous transplantation of retroviral transduced hepatocytes. Proc Natl Acad Sci USA 89:89, 1992
37. Tvedten H: Hemostatic abnormalities, in Willard MD, Tvedten H, Turnwald GH (eds): Small Animal Clinical Diagnosis by Laboratory Methods. Philadelphia, PA, Saunders, 1994, p 81
38. Coles EH: Hemostasis and coagulation of blood, in Pedersen D, Dick E (eds): Veterinary Clinical Pathology. Philadelphia, PA, Saunders, 1986, p 106
39. Vehar GA, Lawn RM, Tuddenham EGD, Wood WI: Factor VIII and factor V: Biochemistry and pathophysiology, in Albertini A, Lenfant CL, Mannucci PM, Sixma JJ (eds): Biotechnology of Plasma Proteins. Current Studies in Hematology and Blood Transfusion. Basel, Switzerland, Karger, 1991, p 2155
40. Hoyer LW, Wyshock EG, Colman RW: Coagulation cofactors: Factors V and VIII, in Colman RW, Hirsh J, Marder VJ, Salzman EW (eds): Hemostasis and Thrombosis: Basic Principles and Clinical Practice. Philadelphia, PA, Lippincott, 1994, p 109
41. Kasper CK, Aledort LM, Counts RB, Edson JR, Fratantoni J, Green D, Hampton JW, Hilgartner MW, Lazerson J, Levine PH, McMillan CW, Pool JG, Shapiro SS, Shulman NR, Van Eys J: A more uniform measurement of factor VIII inhibitors. Thromb Diath Haemorrh 34:869, 1975
42. Toole JJ, Knopf JL, Wozney JM, Sultzman PA, Buecker J, Pittman DD, Kaufman RJ, Brown E, Shoemaker C, Orr EC, Amphlett GW, Foster WB, Coe ML, Knutson GJ, Pass DN, Hewick RM: Molecular cloning of a cDNA encoding antihemophiliac factor. Nature 312:342, 1984
43. Wood WI, Capon DJ, Simonsen CC, Eaton DL, Gitschier J, Keyt B, Seeburg PH, Smith DH, Hollingshead P, Wion KL, Delwart E, Tuddenham EGD, Vehar GA, Lawn RM: Expression of active human factor VIII from recombinant DNA cloned. Nature 312:330, 1984
44. Toole JJ, Pittman DD, Orr EC, Murtha P, Wasley LC, Kaufman RJ: A large region (≈95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc Natl Acad Sci USA 83:5939, 1986
45. Eaton DL, Wood WI, Eaton D, Hass PE, Hollingshead P, Wion K, Mather J, Lawn RM, Vehar GA, Gorman C: Construction and characterization of an active factor VIII variant lacking the central one-third of the molecule. Biochemistry 25:1, 1986
46. Yang Y, Nunes FA, Berencsi K, Furth EE, Gönczöl E, Wilson JM: Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc Natl Acad Sci USA 91:4407, 1994
47. Yang Y, Ertl HCJ, Wilson JM: MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunology 1:433, 1994
48. Yang Y, Wilson JM: Clearance of adenovirus-infected hepatocytes by MHC class I-restricted CD4+ CTLs in vivo. J Immunol 155:2564, 1995
49. Fang B, Eisensmith RC, Wang H, Kay MA, Cross RE, Landen CN, Gordon G, Bellinger DA, Read MS, Hu PC, Brinkhous KM, Woo SLC: Gene therapy for hemophilia B: Host immunosuppression prolongs the therapeutic effect of adenovirus-mediated factor IX expression. Hum Gene Ther 6:1039, 1995
50. Kay MA, Rothenberg SR, Landen CN, Bellinger DA, Leland F, Toman C, Finegold M, Thompson AR, Read MS, Brinkhous KM, Woo SLC: In vivo gene therapy of hemophilia B: Sustained partial correction in factor IX-deficient dogs. Science 262:117, 1993
51. Kay MA, Landen CN, Rothenberg SR, Taylor LA, Leland F, Wiehle S, Fang B, Bellinger D, Finegold M, Thompson AR, Read M, Brinkhous KM, Woo SL: In vivo hepatic gene therapy: Complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc Natl Acad Sci USA 91:2353, 1994
52. Mahajan PB, Thompson EA: Cyclosporine A inhibits the activity of a TATA box-binding protein that is required for transcription from the adenovirus major late promoter. J Biol Chem 268:16693, 1993