Evaluation of an adenoviral vector encoding full-length human factor VIII in hemophiliac mice

Thrombosis and Haemostasis

Volumn 81, Issue 2, 1999, Pages 234-239

  Connelly, Sheila ^b   Andrews, Julie L ^b   Gallo Penn, Angela M ^b   Tagliavacca, Luigina ^a   Kaufman, Randal J ^a   Kaleko, Michael ^b  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b NOVARTIS PHARMA AG   (Switzerland)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLEEDING DISORDER; CONTROLLED STUDY; GENE THERAPY; HEMOPHILIA; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; LIVER; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL;

EID: 0001322026     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1055/s-0037-1614449     Document Type: Article

References (36)

1. Connelly S, Kaleko M. Gene therapy for hemophilia A. Thromb Haemost 1997; 78: 31-6.
2. Connelly S, Kaleko M. Haemophilia A gene therapy. Haemophilia 1998; 4: 380-8.
3. Smith TAG. Gene therapy for haemophilia. Exp Opin Invest Drugs 1997; 6: 1685-90.
4. 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 1995; 6: 185-93.
5. Connelly S, Gardner JM, Lyons RM, McClelland A, Kaleko M. Sustained expression of therapeutic levels of human factor VIII in mice. Blood 1996: 87: 4671-7.
6. Connelly S, Gardner JM, McClelland, A, Kaleko M. High level tissue-specific expression of functional human factor VIII in mice. Hum Gene Ther 1996; 7: 183-95.
7. Connelly S, Andrews J, Gallo AM, Qian J, Hoyer L, Kadan MJ, Gorziglia MI, Trapnell BC, McClelland A, Kaleko M. Sustained phenotypic correction of murine hemophilia A by in vivo gene therapy. Blood 1998; 91: 3273-81.
8. 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 1996; 88: 3846-53.
9. Berkner KL. Development of adenovirus vectors for the expression of heterologous genes. Biotechniques 1988; 6: 616-29.
10. 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 1996; 6: 4173-8.
11. 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 1994; 91: 6196-200.
12. Engelhardt JF, Litzky L, Wilson JM. Prolonged transgene expression in cotton rat lung with recombinant adenoviruses defective in E2a. Hum Gene Ther 1994; 5: 1217-29.
13. 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 1994; 7: 362-9.
14. 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 1997; 8: 1275-86.
15. 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 Therapy 1996; 3: 217-22.
16. 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. Biochem 1986; 25: 1-2.
17. 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 1986; 83: 5939-42.
18. Meulien P, Faure T, Mischler F, Harrer H, Ulrich P, Bouderbala B, Dott K, Sainte Marie M, Mazurier C, Wiesel ML. A new recombinant procoagulant protein derived from the cDNA encoding human factor VIII. Protein Eng 1988; 2: 301-6.
19. Sarver N, Ricca GA, Link J, Nathan MH, Newman J, Drohan WN. Stable expression of recombinant factor VIII molecules using a bovine papillomavirus vector. DNA 1987; 6: 553-64.
20. 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 1993; 81: 2925-35.
21. Dorner AJ, Bole DG, Kaufman RJ. The relationship of N-linked glycosylation and heavy chain-binding protein association with the secretion of glycoproteins. J Cell Biol 1987; 105: 2665-74.
22. 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 1992; 11: 1563-71.
23. Mittereder N, March KL, Trapnell BC. Evaluation of the concentration and bioactivity of adenovirus vectors for gene therapy. J Virol 1996; 70: 7498-509.
24. Tolstoshev P, Mittereder N, Yei S, Trapnell BC. Adenovirus vectors for gene therapy. In: Cancer chemotherapy: Challenges for the future. Excerpta Medica, Ltd. Tokyo 1994; pp. 23-31.
25. Knowles BB, Howe CC, Aden DP. Human hepatocellular carcinoma cell lines secrete the major plasma proteins and hepatitis B surface antigen. Science 1980; 209: 497-9.
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. Nature Genetics 1995; 10: 119-21.
27. 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 1993; 262: 117-9.
28. Berntorp E. Second generation, B-domain deleted recombinant factor VIII. Thromb Haemost 1997; 78: 256-60.
29. Fijnvandraat K, Berntorp E, ten Cate JW, Johnsson H, Peters M, Savidge G, Tengborn L, Spira J, Stahl C. Recombinant, B-domain deleted factor VIII (r-VIII SQ): pharmacokinetics and initial safety aspects in hemophilia A patients. Thromb Haemost 1997; 77: 298-302.
30. Rosenberg JB, Foster PA, Kaufman RJ, Vokac EA, Moussalli M, Kroner PA, Montgomery RR. Intracellular trafficking of factor VIII to von Willebrand factor storage granules. J Clin Invest 1998; 101: 613-24.
31. Swaroop M, Moussalli M, Pipe SW, Kaufman RJ. Mutagenesis of a potential immunoglobulin-binding protein-binding site enhances secretion of coagulation factor VIII. J Biol Chem 1997; 272: 24121-4.
32. Stel HV, van der Kwast TH, Veerman EC. Detection of factor VIII/coagulant antigen in human liver tissue. Nature 1983; 303: 530-2.
33. Stel HV, Oudejan CB, Veerman EC, Meijer CJ. Immunohistological localization of factor VIII in placental endothelial cells. Br J Haematol. 1986; 63: 565-9.
34. Schiedner G, Morral N, Parks RJ, Wu Y, Koopmans SC, Langston C, Graham FL, Beaudet AL, Kochanek S. Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity. Nat Genet 1998; 18: 180-3.
35. Morsy MA, Gu M, Motzel S, Zhau J, Lin J, Su Q, Allen H, Franlin L, Parks RJ, Graham FL, Kochanek S, Bett AJ, Caskey CT. An adenoviral vector deleted for all viral coding sequences results in enhanced safety and extended expression of a leptin transgene. Proc Natl Acad Sci USA 1998; 95: 7866-71.
36. Hoyer LW, Hemophilia A. N Engl J Med 1994; 330: 38-47.