Long-term expression of canine FVIIa in hemophilic dogs

Thrombosis Research

Volumn 125, Issue SUPPL. 1, 2010, Pages

  Margaritis, Paris ^a  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

animal model; canine; Factor VIIa; gene transfer; hemophilia

Indexed keywords

BLOOD CLOTTING FACTOR 7A; COMPLEMENTARY DNA; PARVOVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 7A;

BIOLOGICAL ACTIVITY; DOG; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; GENE THERAPY; GENE TRANSFER; HEMOPHILIA; HEMOPHILIA A; HEMOPHILIA B; HEMOSTASIS; IN VITRO STUDY; LIVER; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTHROMBIN TIME; REVIEW; TRANSGENE;

ANIMALS; BLOOD COAGULATION; DISEASE MODELS, ANIMAL; DNA, COMPLEMENTARY; DOGS; FACTOR VIIA; GENE TRANSFER TECHNIQUES; HEMOPHILIA A; HEMOSTASIS; MICE; PHENOTYPE; PROMOTER REGIONS, GENETIC; RECOMBINANT PROTEINS; TRANSGENES;

EID: 77949269052     PISSN: 00493848     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.thromres.2010.01.040     Document Type: Review

References (27)

1. Leissinger C.A. Inhibitor development in patients with hemophilia: an overview. Semin Hematol 43 2 Suppl 4 (2006) S1-S2
2. Margaritis P., Arruda V.R., Aljamali M., Camire R.M., Schlachterman A., and High K.A. Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII. J Clin Invest 7 (2004) 1025-1031
3. Janson T.L., Stormorken H., and Prydz H. Species specificity of tissue thromboplastin. Haemostasis 14 (1984) 440-444
4. Kadish J.L., Wenc K.M., and Dvorak H.F. Tissue factor activity of normal and neoplastic cells: quantitation and species specificity. J Natl Cancer Inst 70 (1983) 551-557
5. Petersen L.C., Norby P.L., Branner S., Sorensen B.B., Elm T., Stennicke H.R., et al. Characterization of recombinant murine factor VIIa and recombinant murine tissue factor: a human-murine species compatibility study. Thromb Res 116 (2005) 75-85
6. Aljamali M.N., Margaritis P., Schlachterman A., Tai S.J., Roy E., Bunte R., et al. Long-term expression of murine activated factor VII is safe, but elevated levels cause premature mortality. J Clin Invest 118 (2008) 1825-1834
7. Russell K.E., Olsen E.H., Raymer R.A., Merricks E.P., Bellinger D.A., Read M.S., et al. Reduced bleeding events with subcutaneous administration of recombinant human factor IX in immune-tolerant hemophilia B dogs. Blood 102 (2003) 4393-4398
8. Brinkhous K., Sandberg H., Widlund L., Read M., Nichols T., Sigman J., et al. Preclinical pharmacology of albumin-free B-domain deleted recombinant factor VIII. Semin Thromb Hemost 28 (2002) 269-272
9. Brinkhous K.M., Hedner U., Garris J.B., Diness V., and Read M.S. Effect of recombinant factor VIIa on the hemostatic defect in dogs with hemophilia A, hemophilia B, and von Willebrand disease. Proc Natl Acad Sci U S A 86 (1989) 1382-1386
10. Brinkhous K.M., Sandberg H., Garris J.B., Mattsson C., Palm M., Griggs T., et al. Purified human factor VIII procoagulant protein: comparative hemostatic response after infusions into hemophilic and von Willebrand disease dogs. Proc Natl Acad Sci U S A 82 (1985) 8752-8756
11. Brinkhous K.M., Shanbrom E., Roberts H.R., Webster W.P., Fekete L., and Wagner R.H. A new high-potency glycine-precipitated antihemophilic factor (AHF) concentrate. Treatment of classical hemophilia and hemophilia with inhibitors. JAMA 205 (1968) 613-617
12. Brinkhous K.M., Sigman J.L., Read M.S., Stewart P.F., McCarthy K.P., Timony G.A., et al. Recombinant human factor IX: replacement therapy, prophylaxis, and pharmacokinetics in canine hemophilia B. Blood 88 (1996) 2603-2610
13. Jiang H., Lillicrap D., Patarroyo-White S., Liu T., Qian X., Scallan C.D., et al. Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs. Blood 108 (2006) 107-115
14. Mount J.D., Herzog R.W., Tillson D.M., Goodman S.A., Robinson N., McCleland M.L., et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 99 (2002) 2670-2676
15. Sarkar R., Mucci M., Addya S., Tetreault R., Bellinger D.A., Nichols T.C., et al. Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice. Hum Gene Ther 17 (2006) 427-439
16. Wang L., Calcedo R., Nichols T.C., Bellinger D.A., Dillow A., Verma I.M., et al. Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy. Blood 105 (2005) 3079-3086
17. Callan M.B., Aljamali M.N., Margaritis P., Griot-Wenk M.E., Pollak E.S., Werner P., et al. A novel missense mutation responsible for factor VII deficiency in research Beagle colonies. J Thromb Haemost 4 (2006) 2616-2622
18. Margaritis P., Roy E., Aljamali M.N., Downey H.D., Giger U., Zhou S., et al. Successful treatment of canine hemophilia by continuous expression of canine FVIIa. Blood 113 (2009) 3682-3689
19. Nathwani A.C., Gray J.T., Ng C.Y., Zhou J., Spence Y., Waddington S.N., et al. Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver. Blood 107 (2006) 2653-2661
20. Wu Z., Sun J., Zhang T., Yin C., Yin F., Van Dyke T., et al. Optimization of self-complementary AAV vectors for liver-directed expression results in sustained correction of hemophilia B at low vector dose. Mol Ther 16 (2008) 280-289
21. Persson E., Bak H., and Olsen O.H. Substitution of valine for leucine 305 in factor VIIa increases the intrinsic enzymatic activity. J Biol Chem 276 (2001) 29195-29199
22. Persson E., Bak H., Ostergaard A., and Olsen O.H. Augmented intrinsic activity of Factor VIIa by replacement of residues 305, 314, 337 and 374: evidence of two unique mutational mechanisms of activity enhancement. Biochem J 379 (2004) 497-503
23. Harvey S.B., Stone M.D., Martinez M.B., and Nelsestuen G.L. Mutagenesis of the gamma-carboxyglutamic acid domain of human factor VII to generate maximum enhancement of the membrane contact site. J Biol Chem 278 (2003) 8363-8369
24. Nelsestuen G.L., Stone M., Martinez M.B., Harvey S.B., Foster D., and Kisiel W. Elevated function of blood clotting factor VIIa mutants that have enhanced affinity for membranes. Behavior in a diffusion-limited reaction. J Biol Chem 276 (2001) 39825-39831
25. Lauritzen B., Tranholm M., and Ezban M. rFVIIa and a new enhanced rFVIIa-analogue, NN1731, reduce bleeding in clopidogrel-treated and in thrombocytopenic rats. J Thromb Haemost 7 (2009) 651-657
26. Tranholm M., Kristensen K., Kristensen A.T., Pyke C., Rojkjaer R., and Persson E. Improved hemostasis with superactive analogs of factor VIIa in a mouse model of hemophilia A. Blood 102 (2003) 3615-3620
27. Moss J., Scharling B., Ezban M., and Sorensen T.M. Evaluation of the safety and pharmacokinetics of a fast-acting recombinant FVIIa analogue, NN1731, in healthy male subjects. J Thromb Haemost 7 (2009) 299-305