Agency
National Institute of Health (NIH)
Institute
National Heart, Lung, and Blood Institute (NHLBI)
Type
Research Project (R01)
Project #
5R01HL053682-02
Application #
2231738
Study Section
Medical Biochemistry Study Section (MEDB)
Project Start
1995-09-01
Project End
2000-08-31
Budget Start
1996-09-01
Budget End
1997-08-31
Support Year
2
Fiscal Year
1996
Total Cost
Indirect Cost
Name
University of Washington
Department
Internal Medicine/Medicine
Type
Schools of Medicine
DUNS #
135646524
City
Seattle
State
WA
Country
United States
Zip Code
98195
Park, F; Ohashi, K; Kay, M A (2000) Therapeutic levels of human factor VIII and IX using HIV-1-based lentiviral vectors in mouse liver. Blood 96:1173-6
Miao, C H; Nakai, H; Thompson, A R et al. (2000) Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J Virol 74:3793-803
Nakai, H; Storm, T A; Kay, M A (2000) Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors. Nat Biotechnol 18:527-32
Miao, C H; Ohashi, K; Patijn, G A et al. (2000) Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther 1:522-32
Park, F; Ohashi, K; Chiu, W et al. (2000) Efficient lentiviral transduction of liver requires cell cycling in vivo. Nat Genet 24:49-52
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
Nakai, H; Storm, T A; Kay, M A (2000) Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol 74:9451-63
Snyder, R O; Miao, C; Meuse, L et al. (1999) Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med 5:64-70
Kay, M A; High, K (1999) Gene therapy for the hemophilias. Proc Natl Acad Sci U S A 96:9973-5
Nakai, H; Iwaki, Y; Kay, M A et al. (1999) Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J Virol 73:5438-47

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