Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Project (R01)
Project #
5R01HL053668-03
Application #
2231712
Study Section
Special Emphasis Panel (ZHL1-CSR-K (S1))
Project Start
1994-09-30
Project End
1999-08-31
Budget Start
1996-09-01
Budget End
1997-08-31
Support Year
3
Fiscal Year
1996
Total Cost
Indirect Cost
Name
Children's Hospital of Philadelphia
Department
Type
DUNS #
073757627
City
Philadelphia
State
PA
Country
United States
Zip Code
19104
Arruda, V R; Hagstrom, J N; Deitch, J et al. (2001) Posttranslational modifications of recombinant myotube-synthesized human factor IX. Blood 97:130-8
Hagstrom, J N; Couto, L B; Scallan, C et al. (2000) Improved muscle-derived expression of human coagulation factor IX from a skeletal actin/CMV hybrid enhancer/promoter. Blood 95:2536-42
Kay, M A; Manno, C S; Ragni, M V et al. (2000) Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector. Nat Genet 24:257-61
Herzog, R W; High, K A (1999) Adeno-associated virus-mediated gene transfer of factor IX for treatment of hemophilia B by gene therapy. Thromb Haemost 82:540-6
Kay, M A; High, K (1999) Gene therapy for the hemophilias. Proc Natl Acad Sci U S A 96:9973-5
Gu, W; Brooks, M; Catalfamo, J et al. (1999) Two distinct mutations cause severe hemophilia B in two unrelated canine pedigrees. Thromb Haemost 82:1270-5
Herzog, R W; Yang, E Y; Couto, L B et al. (1999) Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector. Nat Med 5:56-63
Kung, S H; Hagstrom, J N; Cass, D et al. (1998) Human factor IX corrects the bleeding diathesis of mice with hemophilia B. Blood 91:784-90
Nakai, H; Herzog, R W; Hagstrom, J N et al. (1998) Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 91:4600-7
Herzog, R W; Hagstrom, J N; Kung, S H et al. (1997) Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus. Proc Natl Acad Sci U S A 94:5804-9

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