In vivo expression of full-length human dystrophin from adenoviral vectors deleted of all viral genes

Human Gene Therapy

Volumn 7, Issue 15, 1996, Pages 1907-1914

  Haecker, Sarah Ehlen ^a,b   Stedman, Hansell H ^a   Balice Gordon, Rita J ^a   Smith, Daniel B J ^a   Greelish, James P ^a   Mitchell, Marilyn A ^a   Wells, Amber ^a   Sweeney, H Lee ^a   Wilson, James M ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b WISTAR INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

DYSTROPHIN; VIRUS VECTOR;

ADENOVIRUS; ANIMAL CELL; ARTICLE; CELL CULTURE; CONTROLLED STUDY; DUCHENNE MUSCULAR DYSTROPHY; GENE DELETION; GENE INSERTION; GENE THERAPY; GENE TRANSFER; GENETIC TRANSDUCTION; IMMUNOGENICITY; MOUSE; MUSCLE CELL; MYOTUBE; NONHUMAN; OPEN READING FRAME; PLASMID; PROTEIN EXPRESSION; RECOMBINANT GENE; SKELETAL MUSCLE; VIRUS GENE; VIRUS RECOMBINANT;

ADENOVIRIDAE; ANIMALIA;

EID: 0029962474     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.1996.7.15-1907     Document Type: Article

References (28)

1. ACSADI, G., DICKSON, G., LOVE, D.R., JANI, A., WALSH, F.S., GURUSINGHE, A., WOLFF, J.A., and DAVIES, K.E. (1991). Human dystrophin expression in mdx mice after intramuscular injection of DNA constructs. Nature 352, 815-818.
2. ACSADI, G., LOCHMULLER, H., JANI, A., HUARD, J., MASSIE, B., PRESCOTT, S., SIMONEAU, M., PETROF, B.J., and KARPATI, G. (1996). Dystrophin expression in muscle of mdx mice after adenovirus-mediated in vivo gene transfer. Hum. Gene Ther. 7, 129-140.
3. BARR, E., and LEIDEN, J.M. (1991). Systemic delivery of recombinant proteins by genetically modified myoblasts. Science 254, 1507-1509.
4. DAI, Y., SCHWARZ, E.M., GU, D., ZHANG, W., SARVETNICK, N., and VERMA, I. (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. USA 92, 1401-1405.
5. DAVIS, A., and WILSON, J.M. (1996). Adenoviral vectors. In Current Protocols in Human Genetics. N. Dracopoli, J. Haines, B. Korf, et. al., eds. (John Wiley & Sons, Inc) (in press).
6. DECONINCK, N., RAGOT, T., MARECHAL, G., PERRICAUDET, M., and GILLIS, J.M. (1996). Functional protection of dystrophic mouse (mdx) muscles after adenovirus-mediated transfer of a dystrophin minigene. Proc. Natl. Acad. Sci. USA 93, 3570-3574.
7. DHAWAN, J., PAN, L.C., PAVLATH, G.K., TRAVIS, M.A., LANCTOT, A.M., and BLAU, H.M. (1991). Systemic delivery of human growth hormone by injection of genetically engineered myoblasts. Science 254, 1509-1512.
8. DICKSON, G., LOVE, D.R., DAVIES, K.E., WELLS, K.E., PIPER, T.A., and WALSH, F.S. (1991). Human dystrophin gene transfer: production and expression of a functional recombinant DNA-based gene. Hum. Genet. 88, 53-58.
9. FABBZIZIO, E., LEGER, J., ANOAL, M., LEGER, J.J., and MORNET, D. (1993). Monoclonal antibodies targeted against the C-terminal domain of dystrophin or utrophin. FEBS Lett. 322, 101-141.
10. FISHER, K.J., BURDA, J., CHEN, S.-J., and WILSON, J.M. (1996). Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 217, 11-22.
11. GOSPE, S.M., LAZARO, R.P., LAVA, N.S., GROOTSCHOLTEN, P.M., SCOTT, M.O., and FISCHBECK, K.H. (1989). Familial X-linked myalgia and cramps: A nonprogressive myopathy associated with a deletion in the dystrophin. Gene Neurol. 39, 1277-1280.
12. HUARD, J., LOCHMULLER, H., ACSADI, G., JANI, A., MASSIE, B., and KARPATI, G. (1995). The route of adminstration is a major determinant of the transduction efficiency of rat tissues by adenoviral recombinants. Gene Ther. 2, 107-115.
13. KOCHANEK, S., CLEMENS, P.R., MITANI, K., CHEN, H.-H., CHAN, S., and CASKEY, C.T. (1996). A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DNA independently expressing both full-length dystrophin and β-galactosidase. Proc. Natl. Acad. Sci. USA 93, 5731-5736.
14. KUMAR-SINGH, R., and CHAMBERLAIN, J.S. (1996). Encapsidated adenovirus mini-chromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Hum. Mol. Genet. 5, 913-921.
15. LIN, Z., ESHLEMAN, J., GRUND, C., FISCHMAN, D.A., MASAKI, T., FRANKE, W.W., and HOLTZER, H.H. (1989). Differential response of myofibrillar and cytoskeletal proteins in cells treated with phorbol myristate acetate. J. Cell Biol. 108, 1079-1091.
16. MORGAN, J.E. (1994). Cell and gene therapy in Duchenne muscular dystrophy. Hum. Gene Ther. 5, 165-173.
17. MULLIGAN, R.C. (1993). The basic science of gene therapy. Science 260, 926-932.
18. PARDOLL, D.M., and BECKERLEG, A.M. (1995). Exposing the immunology of naked DNA vaccines. Immunity 3, 165-169.
19. PARTRIDGE, T.A., MORGAN, J.E., COULTON, G.R., HOFFMAN, E.P., and KUNKEL, L.M. (1989). Conversion of mdx myofibres from dystrophin-negative to -positive by injection of normal myoblasts. Nature 337, 176-179.
20. QUANTIN, B., PERRICAUDET, L.D., TAJBAKHSH, S., and MANDEL, J.-L. (1992). Adenovirus as an expression vector in muscle cells in vivo. Proc. Natl. Acad. Sci. USA 89, 2581-2584.
21. RAGOT, T., VINCENT, N., CHAFEY, P., VIGNE, E., GILGENKRANTZ, H., COUTON, D., CARTAUD, J., BRIAND, P., KAPLAN, J.C., PERRICAUDET, M., and KAHN, A. (1993). Efficient adenovirus-mediated transfer of a human minidystrophin gene to skeletal muscle of mdx mice. Nature 361, 647-650.
22. SCHROEDL, N.A., FUNDAGE, V.L., BACON, C.R., SMITH, S.M., and HARTZELL, C.R. (1988). Hemin increases arobic capacity of cultured regenerating skeletal myotubes. Am. J. Physiol. 255, C519-525.
23. STRATFORD-PERRICAUDET, L.D., MAKEH, I., PERRICAUDET, M., and BRIAND, P. (1992). Widespread, long-term gene transfer to mouse skeletal muscles and heart. J. Clin. Invest. 90, 626-630.
24. TRIPATHY, S.K., BLACK, H.B., GOLDWASSER, E., and LEIDEN, J.M. (1996). Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nat. Med. 2, 545-550.
25. VINCENT, N., RAGOT, T., GILGENKRANTZ, H., COUTON, D., CHAFEY, P., GREGOIRE, A., BRIAND, P., KAPLAN, J.-C., KAHN, A., and PERRICAUDET, M. (1993). Long-term correction of mouse dystrophic degeneration by adenovirus-mediated transfer of a minidystrophin gene. Nature Genet. 5, 130-134.
26. WOLFF, J.A., MALONE, R.W., WILLIAMS, P., CHONG, W., ACSADI, G., JAM, A., and FELGNER, P.L. (1990). Direct gene transfer into mouse muscle in vivo. Science 247, 1465-1468.
27. YANG, Y., ERTL, H.C.J., and WILSON, J.M. (1994). MHC class I-restricted cytotoxic T lymphocytes to viral antigens destroy hepatocytes in mice infected with E1-deleted recombinant adenoviruses. Immunity 1, 433-442.
28. YANG, Y., LI, Q., ERTL, H.C.J., and WILSON, J.M. (1995). Cellular and humoral immune responses to viral antigens create barriers to lung-directed gene therapy with recombinant adenoviruses. J. Virol. 69, 2004-2015.