Adenovirus-mediated transfer of human factor IX gene in immunodeficient and normal mice: Evidence for prolonged stability and activity of the transgene in liver

Viral Immunology

Volumn 9, Issue 3, 1996, Pages 141-153

  Yao, Shou Nan ^a   Farjo, Ayad ^a   Roessler, Blake J ^a   Davidson, Beverly L ^a,b   Kurachi, Kotoku ^a  

^a UNIVERSITY OF MICHIGAN MEDICAL SCHOOL   (United States)

^b UNIVERSITY OF IOWA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTIBODY; BLOOD CLOTTING FACTOR 9; DNA; MESSENGER RNA;

ADENOVIRUS; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CELL DESTRUCTION; CELL SURVIVAL; CONTROLLED STUDY; CYTOTOXIC T LYMPHOCYTE; EXPRESSION VECTOR; GENE ACTIVITY; GENE EXPRESSION; GENE TARGETING; GENE THERAPY; GENE TRANSFER; GENETIC ENGINEERING; GENETIC STABILITY; HEMOPHILIA B; IMMUNE DEFICIENCY; IMMUNE RESPONSE; IMMUNOCOMPETENCE; LIVER; MALE; MOUSE; NONHUMAN; SOUTHERN BLOTTING; TRANSGENE; VIRUS GENOME; VIRUS RECOMBINANT;

ADENOVIRIDAE; ANIMALIA;

EID: 0029803090     PISSN: 08828245     EISSN: None     Source Type: Journal    
DOI: 10.1089/vim.1996.9.141     Document Type: Article

References (31)

1. Ausubel, F.M., R. Brent, R.E. Kingston, D.D. Moore, J.G. Seidman, J.A. Smith, and K. Struhl. 1991. Current Protocols in Molecular Biology, Vol. 1, pp. 2.2.1-2.2.3. Greene Publishing Associates, & John Wiley, Brooklyn, NY.
2. Akli, S., C. Caillaud, E. Vigne, L.D. Stratford-Perricaudet, L. Poenaru, M. Perricaudet, A. Kahn, and M.R. Peschanski. 1993. Transfer of a foreign gene into the brain using adenoviral vectors. Nat. Genet. 3:224-228.
3. Ali, M., N.R. Lemoine, and C.J.A. Ring. 1994. The use of DNA viruses as vectors for gene therapy. Gene Ther. 1:367-384.
4. Bajocchi, G., S.H. Feldman, R.G. Crystal, and A. Mastrangeli. 1993. Direct in vivo gene transfer to ependymal cells in the central nervous system using recombinant adenovirs vectors. Nat. Genet. 3:229-234.
5. Berkner, K.L. 1988. Development of adenoviral vectors for the expression of heterologous genes. BioTechniques 6:616-629.
6. Bout, A., J.-L. Imler, H. Schultz, M. Perricaudet, C. Zurcher, P. Herbrink, D. Valerio, and A. Pavirani. 1994. In vivo adenovirus-mediated transfer of human CFTR cDNA to Rhesus monkey airway epithelium: Efficacy, toxicity, and safety. Gene Ther. 1:385-394.
7. Carlson, G.A., S.T. Marshall, and A.T. Truesdale. 1985 Adaptive immune defects and delayed rejection of allogeneic tumor cells in beige mice. Cell. Immunol. 87:348-356.
8. Dai, Y., E.M. Schwarz, D. Gu, W.-W. Zhang, N. Sarvetnick, and I.M. Verma. 1995. Cellular and humoral immune responses to adenoviral vectors containing factor IX gene: Tolerization of factor IX and vector antigens allows for long-term expression. Proc. Natl. Acad. Sci. U.S.A. 92, 1401-1405.
9. Davidson, B.L., E.D. Allen, K.F. Kozarsky, J.M. Wilson, and B.J. Roessler. 1993. A model system for in vivo gene transfer into the central nervous system using an adenoviral vector. Nat. Genet. 3:219-223.
10. Defosse, D.L., P.H. Daray, and R.C. Johnson. 1992. The NIH-3 immunodeficient mouse is a model for Lyme borreliosis myositis and carditis. Am. J. Pathol. 141:3-10.
11. Engelhardt, J.F., X. Ye, B. Doranz, and J.M. Wilson. 1994. Ablation of E2A in recombinant adenoviruses improves transgene persistence and decreases inflammatory responses in mouse liver. Proc. Natl. Acad. Sci. U.S.A. 91:6196-6200.
12. French, B.A., W. Mazur, R.S. Geske, and R. Bolli. 1994. Direct in vivo gene transfer into porcine myocardium using replication-deficient adenoviral vectors. Circulation 90:2414-2424.
13. Graham, F.L., and Eb A.J. van der. 1973. A new technique for the assay of infectivity of human adenovirus 5 DNA. Virology 52:456-467.
14. Jaffe, H.A., C. Danel, G. Longenecker, M. Metzger, Y. Setoguchi, M.A. Rosenfeld, T.W. Gant, S.S. Thorgeirsson, L.D. Stratford-Perricaudet, M. Perricaudet, A. Pavirani, J.-P. Lecocq, and R.G. Crystal. 1992. Adenovirus-mediated in vivo gene transfer and expression in normal rat liver. Nat. Genet. 1:372-378.
15. Kay, M.A., C.N. Landen, S.R. Rothenberg, L.A. Taylor, F. Leland, S. Wiehle, B. Fang, D. Bellinger, M. Finegold, A.R. Thompson, M. Read, K.M. Brinkhous, and S.L.C. Woo. 1994. In vivo hepatic gene therapy: complete albeit transient correction of factor IX deficiency in hemophilia B dogs. Proc. Natl. Acad. Sci. U.S.A. 91:2353-2357.
16. Le Gal La Salle, G., J.J. Robert, S. Berrard, V. Ridoux, L.D. Stratford-Perricaudet, M. Perricaudet, and J. Mallet. 1993. An adenovirus vector for gene transfer into neurons and glia in the brain. Science 259:988-990.
17. Lieber, A., M.J. Vrancken Peeters, L. Meuse, N. Fausto, J. Perkins, and M.A. Kay. 1995. Adenovirus-mediated urokinase gene transfer induces liver regeneration and allows for efficient retrovirus transduction of hepatocytes in vivo. Proc. Natl. Acad. Sci. U.S.A. 92:6210-6214.
18. Roessler, B.J., E.D. Allen, J.M. Wilson, J.W. Hartman, and B.L. Davidson. 1993. Adenoviral-mediated gene transfer to rabbit synovium in vivo. J. Clin. Invest. 92:1085-1092.
19. Roessler, B.J., J.W. Hartman, D.K. Vallance, J.M. Latta, S.L. Janich, and B.L. Davidson. 1995. Inhibition of interleukin-1-induced effects in synoviocytes transduced with the human IL-1 receptor antagonist cDNA using an adenoviral vector. Hum. Gen. Ther. 6:307-316.
20. Rosenfeld, M.A., W. Siegfried, K. Yoshimura, K. Yoneyama, M. Fukayama, L.E. Stier, P.K. Paakko, P. Gilardi, L.D. Stratford-Perricaudet, and M. Perricaudet. 1991. Adenovirus-mediated transfer of a recombinant alpha 1-antitrypsin gene to the lung epithelium in vivo. Science 252:431-434.
21. Rosenfeld, M.A., K. Yoshimura, B.C. Trapnell, K. Yoneyama, E.R. Rosenthal, W. Dalemans, M. Fukayama, J. Bargon, L.E. Stier, L. Stratford-Perricaudet, M. Perricaudet, W.B. Guggino, A. Pavirani, J.-P. Lecocq, and R.G. Cyrstal. 1992. In vivo transfer of the human cystic fibrosis transmembrane conductance regulator gene to the airway epithelium. Cell 68:143-155.
22. Smith, T.A., M.G. Mehaffey, D.B. Kayda, J.M. Saunders, S. Yei, B.C. Trapnell, A. McClelland, and M. Kaleko. 1993. Adenovirus mediated expression of therapeutic plasma levels of human factor IX in mice. Nat. Genet. 5:397-402.
23. Vincent, N., T. Ragot, H. Gilgenkrantz, D. Couton, P. Chafey, A. Gregoire, P. Briand, J.C. Kaplan, A. Kahn, and M. Perricaudet. 1993. Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nat. Genet. 5:130-134.
24. Wortis, H.H. 1992. X-linked immune deficiency (xid). ILAR News 34:12-14.
25. Yang, Y., F.A. Nunes, K. Berencsi, E.E. Furth, E. Gonczol, and J.M. Wilson. 1994. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. U.S.A. 91:4407-4411.
26. Yang, Y., Q. Li, H.C.J. Ertl, and J.M. Wilson. 1995. Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J. Virol. 69:2004-2015.
27. Yao, S.-N., and K. Kurachi. 1992. Expression of human factor IX in mice after injection of genetically modified myoblasts. Proc. Natl. Acad. Sci. U.S.A. 89:3357-3361.
28. Yao, S.-N., J.M. Wilson, E.G. Nabel, S. Kurachi, H.L. Hachiya, and K. Kurachi. 1991. Expression of human factor IX in rat capillary endothelial cells: Toward somatic gene therapy for hemophilia B. Proc. Natl. Acad. Sci. U.S.A. 88:8101-8105.
29. Yao, S.-N., K.J. Smith, and K. Kurachi. 1994. Primary myoblast-mediated gene transfer: persistent expression of human factor IX in mice. Gene. Ther. 1:99-107.
30. Zabner, J., L.A. Couture, R.J. Gregory, S.M. Graham, A.E. Smith, and M.J. Welsh. 1993. Adenovirus-mediated gene transfer transiently corrects the chloride transport defect in nasal epithelia of patients with cystic fibrosis. Cell 75:207-216.
31. Rao, L., M. Debbas, P. Sabbatini, D. Hockenbery, S. Kornmeyer, and E. White. 1992. The adenovirus E1A proteins induce apoptosis, which is inhibited by the E1B 19-Da and Bcl-2 proteins. Proc. Natl. Acad. Sci. U.S.A. 89:7742-7746.