Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors

Nature Medicine

Volumn 5, Issue 1, 1999, Pages 64-70

  Snyder, Richard O ^a   Miao, Carol ^b   Meuse, Leonard ^c   Tubb, Julie ^b   Donahue, Brian A ^a   Lin, Hui Feng ^d   Stafford, Darrel W ^d   Patel, Salil ^a   Thompson, Arthur R ^b   Nichols, Timothy ^e   Read, Marjorie S ^e   Bellinger, Dwight A ^e   Brinkhous, Kenneth M ^e   Kay, Mark A ^c  

^a CELL GENESYS INC   (United States)

^b UNIVERSITY OF WASHINGTON   (United States)

^c STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d UNIVERSITY OF NORTH CAROLINA   (United States)

^e UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; BLOOD CLOTTING; DOG; GENE EXPRESSION REGULATION; GENE TARGETING; GENE THERAPY; HEMOPHILIA B; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN EXPRESSION;

ANIMALS; ANTIBODIES; BLEEDING TIME; CELL TRANSFORMATION, VIRAL; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DOGS; FACTOR IX; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; LIVER; MICE; MICE, INBRED C57BL; RECOMBINATION, GENETIC;

ADENOVIRIDAE; ANIMALIA; CANIS FAMILIARIS; MURINAE;

EID: 17444372024     PISSN: 10788956     EISSN: None     Source Type: Journal    
DOI: 10.1038/4751     Document Type: Article

References (31)

1. Lin, H.-F., Maeda, N., Smithies, O., Straight, D.L. & Stafford, D.W. A Coagulation Factor IX-Deficient Mouse Model for Human Hemophilia B. Blood 90, 3962-3966 (1997).
2. Bi, L. et al. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nature Gen. 10, (1995)
3. Wang, L. et al. A factor IX-deficient mouse model for hemophilia B gene therapy. Med. Sci. 94, 11563-11566 (1997).
4. Kundu, R.K. et al. Targeted Inactivation of the Coagulation Factor IX Gene Causes Hemophilia B. in Mice. Blood 92, 68-174 (1998).
5. Nichols, T.C. et al. The roles of von Willebrand factor and factor VIII in arterial thrombosis: studies in canine von Willebrand disease and hemophilia A. Blood 81, 2644-2651 (1993).
6. Giles, A.R., Tinlin, S. & Greenwood, R. A canine model of hemophilic (factor VIII:C deficiency) bleeding. Blood 60, 727-730 (1982).
7. Kingdon, H.S. & Hassell, T.M. Hemophilic dog model for evaluating therapeutic effectiveness of plasma protein fractions. Blood 58, 868-872 (1981).
8. Mauser, A.E., Whitlark, J. Whitney, K.M. & Lothrop, Jr., C.D. A deletion mutation causes hemophilia B in Lhasa Apso dogs. Blood 88, 3451-3455 (1996).
9. Brinkhous, K.M. et al. Pharmacokinetics and pharmacodynamics of a B-domain deleted recombinant factor VIII derivative, r-VIII SQ, in canine hemophilia. Thromb. Haemost. 69, 852 (1993).
10. Griggs, T.R., Webster, W.P., Cooper, H.A., Wagner, R.H. & Brinkhous, K.M. Von Willebrand Factor: Gene dosage relationships and transfusion response in bleeder swine - a new bioassay. Proc. Natl. Acad. Sci. USA 71, 2087-2090 (1974).
11. Neuenschwander, S. & Pliska, V. 1994. Factor VIII in blood plasma of haemophilic sheep: analysis of clotting time-plasma dilution curves. Haemostasis 24, 27-35 (1994).
12. Dai, Y. et al. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Med. Sci. 92, 1401-1405 (1995).
13. Herzog, R.W. et al. Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc. Natl. Acad. Sci. USA 94, 5804-5809 (1997).
14. Yao, S.N., Smith, K.J. & Kurachi, K. Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene Ther. 1, 99-107 (1994).
15. Yao, S.N., & Kurachi, K. Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. USA 89, 3357-3361 (1992).
16. Rosenberg, J.B. et al. Intracellular trafficking of factor VIII to von Willebrand factor granules. J. Clin. Invest. 101, 613-624 (1998).
17. Ewenstein, B.M. et al. Nephrotic syndrome as a complication of immune tolerance in hemophilia B. Stood 89, 1115-1116 (1997).
18. Warrier, I. et al. Factor IX inhibitors and anaphylaxis in hemophilia B. J. Pediatr. Hematol. Oncol. 19, 23-27 (1997).
19. Smith, T.A. et al. Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice. Nature Genet. 5, 397-402 (1993).
20. Kung, S.-H. et al. Human Factor IX Corrects the Bleeding Diathesis of Mice with Hemophilia B. Blood 91, 784-790.
21. Kay, M.A. et al. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. USA 91, 2353-2357 (1994).
22. Kay, M.A. et al. In vivo gene therapy of hemophilia B: sustained partial correction in factor IX-deficient dogs. Science 262, 117-119 (1993).
23. Snyder, R.O. et al. Persistent and therapeutic concnetrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nature Genet. 16, 270-275 (1997).
24. Miao, C.H. et al. The kinetics of rAAV integration in the liver. Nature Genet. 19, 13-15 (1998).
25. Nakai, H. et al. Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 91, 4600-4607 (1998).
26. Brinkhous, K.M., Landen, C.N. & Read, M.S. Evaluation of sensitivity of whole blood clotting time (WBCT), partial thromboplastin time (PTT), and F.IX one-stage bioassay tests with low plasma F.IX levels observed with transfusion or gene therapy in canine hemophilia B. Blood 82, suppl 1:592a (1993).
27. Samulski, R.J., Chang, L.-S. & Shenk, T. Helper-free stocks of recombinant adeno-associated viruses: Normal intergation does not require viral gene expression. J. Virol. 63, 3822-3828 (1989).
28. Dranoff, G. et al. Vaccination with irridated tumor cells engineered to secrete murine granulocyte-macrophage colony-stimulating factor stimulate potent, specific, and long-lasting anti-tumor immunity. Proc. Natl. Acad. Sci. USA 90, 3539-3543 (1993).
29. Zolotukhin, S., Potter, M., Hauswirth, W.W., Guy, J. & Muzyczka, N. A "humanized" green fluorescent protein cDNA adapted for high-level gene expression in mammalian cells. J. Virol. 70, 4646-4654 (1996).
30. Li, J., Samulski, R.J. & Xiao, X. Role for highly regulated rep gene expression in adeno-associated virus vector production. J. Virol. (in the press).
31. Kasper, C.K. & Pool, J.G. Measurement of milid factor VIII inhibitors in Bethesda units. Thromb. Diathesis Haemorrhagica 34, 875-876 (1975).