Advances in gene therapy using factor VIIa in hemophilia

Seminars in Hematology

Volumn 43, Issue SUPPL. 1, 2006, Pages

  Margaritis, Paris ^a   High, Katherine A ^a,b  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b HOWARD HUGHES MEDICAL INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

RECOMBINANT BLOOD CLOTTING FACTOR 7A;

BLEEDING; BLOOD CLOTTING; CONFERENCE PAPER; COST BENEFIT ANALYSIS; DOSE RESPONSE; DRUG EFFICACY; GENE THERAPY; GENE TRANSFER; HEMOPHILIA; HISTOLOGY; IN VIVO STUDY; MICROVASCULATURE; NONHUMAN; PRIORITY JOURNAL; PROTHROMBIN TIME; REPRODUCTION; SURVIVAL TIME; THROMBOPLASTIN TIME;

ANIMALS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FACTOR VII; GENE TARGETING; GENE THERAPY; GENE TRANSFER TECHNIQUES; GENETIC VECTORS; HEMOPHILIA B; HUMANS; LIVER; MICE; RECOMBINANT PROTEINS;

EID: 31444455364     PISSN: 00371963     EISSN: None     Source Type: Journal    
DOI: 10.1053/j.seminhematol.2005.11.015     Document Type: Conference Paper

References (23)

1. J. Addiego, C. Kasper, C. Abilgaard, M. Hilgartner, J. Lusher, B. Glader Frequency of inhibitor development in haemophiliacs treated with low-purity factor VIII Lancet 342 1993 462 464
2. G.L. Bray, E.D. Gomperts, S. Courter, R. Gruppo, E.M. Gordon, M. Manco-Johnson The Recombinate Study Group A multicenter study of recombinant factor VIII (recombinate) Safety, efficacy, and inhibitor risk in previously untreated patients with hemophilia A Blood 83 1994 2428 2435
3. L.W. Hoyer The incidence of factor VIII inhibitors in patients with severe hemophilia A Adv Exp Med Biol 386 1995 35 45
4. J.M. Lusher, S. Arkin, C.F. Abildgaard, R.S. Schwartz Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group N Engl J Med 328 1993 453 459
5. H.S. Strauss Acquired circulating anticoagulants in hemophilia A N Engl J Med 281 1969 866 873
6. L.J. Sjamsoedin, L. Heijnen, E.P. Mauser-Bunschoten, J.L. van Geijlswijk, H. van Houwelingen, P. van Asten The effect of activated prothrombin-complex concentrate (FEIBA) on joint and muscle bleeding in patients with hemophilia A and antibodies to factor VIII. A double-blind clinical trial N Engl J Med 305 1981 717 721
7. J.M. Lusher, P.M. Blatt, J.A. Penner, L.M. Aledort, P.H. Levine, G.C. White Autoplex versus proplex A controlled, double-blind study of effectiveness in acute hemarthroses in hemophiliacs with inhibitors to factor VIII Blood 62 1983 1135 1138
8. N.S. Key, L.M. Aledort, D. Beardsley, H.A. Cooper, G. Davignon, B.M. Ewenstein Home treatment of mild to moderate bleeding episodes using recombinant factor VIIa (Novoseven) in haemophiliacs with inhibitors Thromb Haemost 80 1998 912 918
9. A.D. Shapiro, G.S. Gilchrist, W.K. Hoots, H.A. Cooper, D.A. Gastineau Prospective, randomised trial of two doses of rFVIIa (NovoSeven) in haemophilia patients with inhibitors undergoing surgery Thromb Haemost 80 1998 773 778
10. J.M. Lusher Acute hemarthroses the benefits of early versus late treatment with recombinant activated factor VII Blood Coagul Fibrinolysis 11 suppl 1 2000 S45 S49
11. G. Kenet, A. Lubetsky, S. Gitel, J. Luboshitz, D. Varon, U. Martinowitz Treatment of bleeding episodes in patients with hemophilia and an inhibitor Comparison of two treatment protocols with recombinant activated factor VII Blood Coagul Fibrinolysis 11 suppl 1 2000 S35 S38
12. G. Kenet, A. Lubetsky, J. Luboshitz, U. Martinowitz A new approach to treatment of bleeding episodes in young hemophilia patients A single bolus megadose of recombinant activated factor VII (NovoSeven) J Thromb Haemost 1 2003 450 455
13. R. Parameswaran, A.D. Shapiro, J.C. Gill, C.M. Kessler Dose effect and efficacy of rFVIIa in the treatment of haemophilia patients with inhibitors Analysis from the Hemophilia and Thrombosis Research Society Registry Haemophilia 11 2005 100 106
14. J.D. Mount, R.W. Herzog, D.M. Tillson, S.A. Goodman, N. Robinson, M.L. McCleland Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy Blood 99 2002 2670 2676
15. N.R. Blacklow, M.D. Hoggan, M.S. Sereno, C.D. Brandt, H.W. Kim, R.H. Parrott A seroepidemiologic study of adenovirus-associated virus infection in infants and children Am J Epidemiol 94 1971 359 366
16. M.G. Kaplitt, P. Leone, R.J. Samulski, X. Xiao, D.W. Pfaff, K.L. O'Malley Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain Nat Genet 8 1994 148 154
17. P.D. Kessler, G.M. Podsakoff, X. Chen, S.A. McQuiston, P.C. Colosi, L.A. Matelis Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein Proc Natl Acad Sci U S A 93 1996 14082 14087
18. X. Xiao, J. Li, R.J. Samulski Efficient long-term gene transfer into muscle tissue of immunocompetent mice by adeno-associated virus vector J Virol 70 1996 8098 8108
19. A.M. Gallo-Penn, P.S. Shirley, J.L. Andrews, S. Tinlin, S. Webster, C. Cameron 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
20. T.R. Flotte, S.A. Afione, C. Conrad, S.A. McGrath, R. Solow, H. Oka Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector Proc Natl Acad Sci U S A 90 1993 10613 10617
21. T.R. Flotte, B.J. Carter Adeno-associated virus vectors for gene therapy Gene Ther 2 1995 357 362
22. R.O. Snyder, X. Xiao Vectors for gene therapy Curr Prot Genet 12.01-12.1.24 Suppl 10 1996
23. V.M. Rivera, X. Wang, S. Wardwell, N.L. Courage, A. Volchuk, T. Keenan Regulation of protein secretion through controlled aggregation in the endoplasmic reticulum Science 287 2000 826 830