Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

Proceedings of the National Academy of Sciences of the United States of America

Volumn 94, Issue 11, 1997, Pages 5804-5809

  Herzog, Roland W ^a   Hagstrom, J Nathan ^a   Kung, Szu Hoo ^a   Tai, Shing Jen ^a   Wilson, James M ^b   Fisher, Krishna J ^b   High, Katherine A ^a,b,c  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b UNIVERSITY OF PENNSYLVANIA   (United States)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; ENZYME RECONSTITUTION; FEMALE; GENE THERAPY; GENE TRANSFER; HEMOPHILIA B; MOUSE; NONHUMAN; PRIORITY JOURNAL; VIRUS RECOMBINANT;

ANIMALS; ANTIBODIES, HETEROPHILE; DEPENDOVIRUS; DNA PRIMERS; ENZYME-LINKED IMMUNOSORBENT ASSAY; FACTOR IX; FLUORESCENT ANTIBODY TECHNIQUE; GENE THERAPY; GENE TRANSFER TECHNIQUES; HOMEODOMAIN PROTEINS; HUMANS; IMMUNOLOGIC DEFICIENCY SYNDROMES; INJECTIONS, INTRAMUSCULAR; MICE; MICE, INBRED C57BL; MICE, MUTANT STRAINS; MUSCLE, SKELETAL; POLYMERASE CHAIN REACTION; PROTEIN BIOSYNTHESIS; PROTEINS;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 0030902985     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.94.11.5804     Document Type: Article

References (26)

1. Dai, Y., Schwartz, E. M., Gu, D., Zhang, W. W., Sarvetnick, N. & Verma, I. M. (1995) Proc. Natl. Acad. Sci. USA 92, 1401-1405.
2. Tripathy, S. K., Black, H. B., Goldwasser, E. & Leiden, J. M. (1996) Nat. Med. 2, 545-550.
3. Yang, Y., Haecker, S. E., Su, Q. & Wilson, J. M. (1996) Hum. Mol. Genet. 5, 1703-1712.
4. Tripathy, S. K., Svensson, E. C., Black, H. G., Goldwasser, E., Margalith, M., Hobart, P. M. & Leiden, J. M. (1996) Proc. Natl. Acad. Sci. USA 93, 10876-10880.
5. Carter, B. J. (1992) Curr. Opin. Biotech. 3, 533-539.
6. Skulimowski, A. W. & Samuiski, R. J. (1995) Methods Mol. Genet. 7, 7-12.
7. Fisher, K. J., Gao, G.-P., Weitzman, M. D., DeMatteo, R., Burda, J. F. & Wilson, J. F. (1996) J. Virol. 70, 520-532.
8. Samulski, R. J., Chang, L.-S. & Shenk, T. (1989) J. Virol. 63, 3822-3828.
9. Kaplitt, M. G., Leone, P., Samulski, R. J., Xiao, X., Pfaff, D. W., O'Malley, K. L. & During, M. J. (1994) Nat. Genet. 8, 148-154.
10. Fisher, K. J., Jooss, K., Alston, J., Yang, Y., Ehlen-Haecker, S., High, K., Pathak, R., Raper, S. E. & Wilson, J. M. (1997) Nat. Med. 3, 306-312.
11. Xiao, X., Li, J. & Samuiski, R. J. (1996) J. Virol. 70, 8098-8108.
12. Kessler, P. D., Podsakoff, G. M., Chen, X., McQuston, S. A., Colosi, P. C., Matelis, L. A., Kurtzman, G. J. & Byrne, B. J. (1996) Proc. Natl. Acad. Sci. USA 93, 14082-14087.
13. Kurachi, S., Hitomi, Y., Furukawa, M. & Kurachi, K. (1995) J. Biol. Chem. 270, 5276-5281.
14. Walter, J., You, Q., Hagstrom, N., Sands, M. & High, K. A. (1996) Proc. Natl. Acad. Sci. USA 93, 3056-3061.
15. Sambrook, J., Fritsch, E. F. & Maniatis, T. (1989) Molecular Cloning: A Laboratory Manual (Cold Spring Harbor Lab. Press, Plainview, NY), 2nd Ed., pp. 9-16-9-19.
16. Cheung, W.-F., Born, J. v. d., Kühn, K., Kjelléen, L., Hudson, B. G. & Stafford, D. W. (1996) Proc. Natl. Acad. Sci. USA 93, 11068-11073.
17. McLaughlin, S. K., Collins, P., Hermonat, P. L. & Muzyczka, N. (1988) J. Virol. 62, 1963-1973.
18. Kay, M. A., Rothenberg, S., Landen, C. N., Bellinger, D. A., Leland, F., Toman, C., Finegold, M., Thompson, A. R., Read, M. S., Brinkhous, K. M. & Woo, S. L. C. (1993) Science 262, 117-119.
19. Kay, M. A., Landen, C. N., Rothenberg, S. R., Taylor, L. A., Leland, F., Wiehle, S., Fang, B., Bellinger, D., Finegold, M., Thompson, A. R., Read, M., Brinkhous, K. M. & Woo, S. L. C. (1994) Proc. Natl. Acad. Sci. USA 91, 2353-2357.
20. Smith, T. A. G., Mehaffey, M. G., Kayda, D. B., Saunders, J. M., Yei, S., Trapnell, B. C., McClelland, A. & Kaleko, M. (1993) Nat. Genet. 5, 397-402.
21. Yao, S.-N., Smith, K. J. & Kurachi, K. (1994) Gene Ther. 1, 99-107.
22. Ferrari, F. K., Samuiski, T., Shenk, T. & Samuiski, R. J. (1996) J. Virol. 70, 3227-3234.
23. Cheung, W.-F., Hamaguchi, N., Smith, K. J. & Stafford, D. W. (1992) J. Biol. Chem. 267, 20529-20531.
24. Berns, K. I. (1990) Microbiol. Rev. 54, 316-329.
25. Tratschin, J. D., Miller, I. L., Smith, M. G. & Carter, B. J. (1985) Mol. Cell Biol. 5, 3251-3260.
26. Muzyczka, N. (1992) Curr. Top. Micmbiol. Immunol. 158, 97-129.