Sustained phenotypic correction of murine hemophilia a by in vivo gene therapy

Blood

Volumn 91, Issue 9, 1998, Pages 3273-3281

  Connelly, Sheila ^b   Andrews, Julie L ^b   Gallo, Angela M ^b   Kayda, Dawn B ^b   Qian, Jiahua ^b   Hoyer, Leon ^b   Kadan, Michael J ^b   Gorziglia, Mario I ^b   Trapnell, Bruce C ^b   McClelland, Alan ^b   Kaleko, Michael ^a  

^a NOVARTIS PHARMA AG   (Switzerland)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY RESPONSE; ARTICLE; BLEEDING DISORDER; CELL SURVIVAL; CONTROLLED STUDY; DOG; GENE MUTATION; GENE THERAPY; GENE TRANSFER; HEMOPHILIA A; HUMAN TISSUE; IN VIVO STUDY; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROPHYLAXIS;

EID: 0032080179     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood.v91.9.3273     Document Type: Article

References (59)

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. Philadelphia, PA, 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. Connelly S, Kaleko M: Gene therapy for hemophilia A. Thromb Haemost 78:3l, 1997
4. Kaufman RJ: Biological regulation of factor VIII activity. Annu Rev Med 43:325, 1992
5. 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
6. Hoeben RC, Fallaux FJ, Cramer SJ, van den Wollenberg JM, Van Ormondt H, Briet 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
7. Koeberl DD, Halbert CL, Krumm A, Miller AD: Sequences within the coding regions of clotting factor VIII and CFTR block transcriptional elongation. Hum Gene Ther 6:469, 1995
8. 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
9. 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
10. 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
11. 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
12. 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
13. 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
14. Hoeben RC, van der Jagt RC, Schoute F, Van Tilburg NH, Verbeet MP, Briet 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
15. Hoeben RC, Einerhand MP, Briet 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
16. Hoeben RC, Fallaux FJ, Van Tilburg NH, Cramer SJ, Van Ormondt H, Briet 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
17. Israel DI, Kaufman RJ: Retroviral-mediated transfer and amplification of a functional human factor VIII gene. Blood 75:1074, 1990
18. 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
19. 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
20. 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
21. Connelly S, Mount J, Mauser A, Gardner JM, Kaleko M, McClelland A, Lothrop CD: Complete short-term correction of canine hemophilia A by in vivo gene therapy. Blood 88:3846, 1996
22. 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
23. Kozarsky KF, Wilson JM: Gene therapy: Adenovirus vectors. Curr Opin Genet Dev 3:499, 1993
24. 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
25. Giles AR, Tinlin S, Greenwood R: A canine model of hémophilic (factor VIII:C deficiency) bleeding. Blood 60:727, 1982
26. Bi L, Lawler AM, Antonarakis SE, High KA, Gerhart JD, Kazazian HH: Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet 10:119, 1995
27. Antonarakis SE, Kazazian HH, Tuddenham EGD: Molecular etiology of factor VIII deficiency in hemophilia A. Hum Mutat 5:1, 1995
28. 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
29. Gorziglia MI, Kadan MJ, Yei S, Lim J, Lee GM, Luthra R, Trapnell BC: Elimination of both E1 and E2a from adenovirus vectors further improves prospects for in vivo human gene therapy. J Virol 6:4173, 1996
30. Mittereder N, March KL, Trapnell BC: Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy. J Virol 70:7498, 1996
31. Rosenfeld MA, Yoshimura K, Trapnell BC, Yoneyama K, Rosenthal ER, Dalemans W, Fukayama M, Bargon J, Stier LE, Stratford Perricaudet L, Perricaudet M, Pavicani A, Lecocq JP, Crystal RG: In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68:143, 1992
32. Bi L, Sarkar R, Naas T, Lawler AM, Pain J, Shumaker S, Bedian V, Kazazian HH: Further characterization of factor VIII-deficient mice created by gene targeting: RNA and protein studies. Blood 88:3446, 1996
33. Hoyer LW: Hemophilia A. N Engl J Med 330:38, 1994
34. Lollar P, Parker ET, Curtis JE, Helgerson SL, Hoyer LW, Scott ME, Scandella D: Inhibition of human factor VIIIa by anti-A2 subunit antibodies. J Clin Invest 93:2497, 1994
35. 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, Fass DN, Hewick RM: Molecular cloning of a cDNA encoding antihemophiliac factor. Nature 312:342, 1984
36. 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
37. 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
38. 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
39. Engelhardt JF, Ye X, Doranz B, Wilson JM: Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory response in mouse liver. Proc Natl Acad Sci USA 91:6196, 1994
40. Engelhardt JF, Litzky L, Wilson JM: Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum Gene Ther 5:1217, 1994
41. Yang Y, Nunes FA, Berencsi K, Gonczol E, Engelhardt JF, Wilson JM: Inactivation of E2a in recombinant adenoviruses improves the prospect for gene therapy in cystic fibrosis. Nat Genet 7:362, 1994
42. Qian J, Bi L, Kazazian HH, Hoyer LW: Inhibitor development and T cell response to human factor VIII in murine hemophilia A. Blood 88:656a, 1996 (suppl, abstr)
43. White GC, Roberts HR: The treatment of factor VIII inhibitors - a general overview. Vox Sang 70:19, 1996 (suppl 1)
44. Qian J, Scott DE, Hoyer, LW: T cell response to human factor VIII in murine hemophilia A. Blood 90:599a, 1997 (abstr, suppl 1)
45. Fang B, Wang H, Gordon G, Bellinger DA, Read MS, Brinkhous KM, Woo SL, Eisensmith RC: Lack of persistence of E1-recombinant adenoviral vectors containing a temperature-sensitive E2A mutation in immunocompetent mice and hemophilia B dogs. Gene Ther 3:217, 1996
46. Morral N, O'Neal W, Zhou H, Langston C, Beaudet A: Immune responses to reporter proteins and high viral dose limit duration of expression with adenoviral vectors: Comparison of E2a wild type and E2a deleted vectors. Hum Gene Ther 8:1275, 1997
47. Gao G, Yang Y, Wilson JM: Biology of adenovirus vectors with E1 and E4 deletions for liver-directed gene therapy. J Virol 70:8934, 1996
48. Armentano D, Zabner J, Sacks C, Sookdeo CC, Smith MP, St. George JA, Wadsworth SC, Smith AE, Gregory RJ: Effect of the E4 region on the persistence of transgene expression from adenovirus vectors. J Virol 71:2408, 1997
49. Dedieu JF, Vigne E, Torrent C, Jullien C, Mahfouz I, Caillaud JM, Aubailly N, Orsini C, Guillaume JM, Opolon P, Delaere P, Perricaudet M, Yeh P: Long-term gene delivery into the livers of immunocompetent mice with E1/E4-defective adenoviruses. J Virol 71:4626, 1997
50. Wang Q, Greenburg G, Bunch D, Farson D, Finer MH: Persistent transgene expression in mouse liver following in vivo gene transfer with a ΔE1/ΔE4 adenovirus vector. Gene Ther 4:393, 1997
51. Smith TAG, White BD, Gardner JM, Kaleko M, McClelland A: Transient immunosuppression permits successful repetitive intravenous administration of an adenovirus vector. Gene Ther 3:496, 1996
52. Yang Y, Trinchieri G, Wilson JM: Recombinant IL-12 prevents formation of blocking IgA antibodies to recombinant adenovirus and allows repeated gene therapy to mouse lung. Nat Med 1:890, 1995
53. Yang Y, Greenough K, Wilson JM: Transient immune blockade prevents formation of neutralizing antibody to recombinant adenovirus and allows repeated gene transfer to mouse liver. Gene Ther 5:412, 1996
54. Clemens PR, Kochanek S, Sunada Y, Chan S, Chen H, Campbell KP, Caskey CT: In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther 3:965, 1996
55. Fisher KJ, Choi H, Bruda J, Chen S, Wilson JM: Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 217:11, 1996
56. Haecker SE, Stedman HH, Balice-Gordon RJ, Smith DBJ, Greelish JP, Mitchell MA, Wells A, Sweeney HL, Wilson JM: In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes. Hum Gene Ther 7:1907, 1996
57. Kochanek S, Clemens PR, Mitani K, Chen H, Chan S, Caskey CT: A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and beta-galactosidase. Proc Natl Acad Sci USA 93:5731, 1996
58. Lieber A, He C, Kirillova I, Kay MA: Recombinant adenoviruses with large deletions generated by cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J Virol 70:8944, 1996
59. Hardy S, Kitamura M, Harris-Stansil T, Dai Y, Phipps ML: Construction of adenovirus vectors through Cre-lox recombination. J Virol 71:1842, 1997