Genomic DNA transfer with a high-capacity adenovirus vector results in improved in vivo gene expression and decreased toxicity

Nature Genetics

Volumn 18, Issue 2, 1998, Pages 180-183

  Schiedner, Gudrun ^a,d   Morral, Núria ^a,b   Parks, Robin J ^c   Wu, Ying ^a   Koopmans, Suzanne C ^a   Langston, Claire ^a   Graham, Frank L ^c   Beaudet, Arthur L ^a,b   Kochanek, Stefan ^a,d  

^a BAYLOR COLLEGE OF MEDICINE   (United States)

^b HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^c MCMASTER UNIVERSITY   (Canada)

^d UNIVERSITY OF COLOGNE   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

DNA;

ARTICLE; CLONING VECTOR; DNA TRANSFER; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; GENOME; HUMAN; PLASMID; PRIORITY JOURNAL; TRANSCRIPTION REGULATION;

ADENOVIRIDAE; ALPHA 1-ANTITRYPSIN; ANIMALS; DNA-BINDING PROTEINS; GENE EXPRESSION REGULATION; GENE THERAPY; GENES, RAG-1; GENETIC VECTORS; HOMEODOMAIN PROTEINS; HUMANS; LIVER; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; PROMOTER REGIONS (GENETICS); RECOMBINANT PROTEINS; TRANSCRIPTION, GENETIC; TRANSFECTION;

EID: 0031916435     PISSN: 10614036     EISSN: None     Source Type: Journal    
DOI: 10.1038/ng0298-180     Document Type: Article

References (30)

1. Stratford-Perricaudet, L.D., Levrero, M., Chasse, J.-F., Perricaudet, M. & Briand, P. Evaluation of the transfer and expression in mice of an enzyme-encoding gene using a human adenovirus vector. Hum. Gene Ther. 1, 241-256 (1990).
2. Yang, Y. et al. Cellular immunity to viral antigens limits E1-deleted adenoviruses for gene therapy. Proc. Natl. Acad. Sci. USA 91, 4407-4411 (1994).
3. Armentano, D. et al. Characterization of an adenovirus gene transfer vector containing an E4 deletion. Hum. Gene Ther. 6, 1343-1353 (1995).
4. Krougliak, V. & Graham, F.L. Development of cell lines capable of complementing E1, E4, and protein IX defective adenovirus type 5 mutants. Hum. Gene Tner. 6, 1575-1586 (1995).
5. Wang, Q., Jia, X.-C. & Finer, M.H. A packaging cell line for propagation of recombinant adenovirus vectors containing two lethal gene-region deletions. Gene Ther. 2, 775-783 (1995).
6. Zhou, H., O'Neal, W., Morral, N. & Beaudet, A.L. Development of a complementing cell line and a system for construction of adenoviral vectors deleted for E1 and E2a. J. Virol. 70, 7030-7038 (1996).
7. Guo, Z.S., Wang, L.-H., Eisensmith, R.C. & Woo, S.L.C. Evaluation of promoter strength for hepatic gene expression in vivo following adenovirus-mediated gene transfer. Gene Ther. 3, 802-810 (1996).
8. Mitani, K., Graham, F.L., Caskey, C.T. & Kochanek, S. Rescue, propagation, and partial purification of a helper virus-dependent adenovirus vector. Proc. Natl. Acad. Sci. USA 92, 3854-3858 (1995).
9. Kochanek, S. et al. A new adenoviral vector: Replacement of all viral coding sequences with 28 kb of DMA independently expressing both full-length dystrophin and β-galactosidase Proc. Natl. Acad. Sci. U.S.A. 93, 5731-5736 (1996).
10. Kumar-Singh, R. & Chamberlain, J.S. Encapsidated adenovirus minichromosomes allow delivery and expression of a 14 kb dystrophin cDNA to muscle cells. Hum. Mol. Genet. 5, 913-921 (1996).
11. Fisher, K.J., Choi, H., Burda, J., Chen, S.-J. & Wilson, J.M. Recombinant adenovirus deleted of all viral genes for gene therapy of cystic fibrosis. Virology 217, 11-22 (1996).
12. Clemens, P.R. et al. In vivo muscle gene transfer of full-length dystrophin with an adenoviral vector that lacks all viral genes. Gene Ther. 3, 965-972 (1996).
13. Chen, H.-H. et al. Persistence in muscle of an adenoviral vector that lacks all viral genes. Proc. Natl. Acad. Sci. USA 94, 1645-1650 (1997).
14. Hardy, S., Kitamura, M., Harris-Stansil, T., Dai, Y. & Phipps, M.L. Construction of adenovirus vectors through Cre-lox recombination J. Virol. 71, 1842-1849 (1997).
15. Parks, R.J. et al. A helper-dependent adenovirus vector system: Removal of helper virus by Cre-mediated excision of the viral packaging signal. Proc. Natl. Acad. Sci. USA 93, 13565-13570 (1996).
16. Dycaico, M.J. et al. Neonatal hepatitis induced by alpha 1-antitrypsin: A transgenic mouse model. Science 242, 1409-1412 (1988).
17. Edwards, A. et al. Automated DNA sequencing of the human HPRT locus. Genomics 6, 593-608 (1990).
18. Parks, R.J. & Graham, F.L. A helper-dependent system for adenovirus vector production helps define a lower limit for efficient DNA packaging. J. Virol. 71, 3293-3298 (1997).
19. 1-antitrypstn gene is transcribed from two different promoters in macrophages and hepatocytes. EMBO J. 6, 2767-2771 (1987).
20. 1 antitrypsin gene. J. Clin. Invest. 89, 1214-1222 (1992).
21. Morral, N., O'Neal, W., Zhou, H., Langsten, C. & Beaudet, A. Immune responses to reporter proteins and high viral dose limit duration of expression with adenoviral vectors: Comparison of E2a wild type and E2a deleted vectors. Hum. Gene Ther. 8, 1275-1286 (1997).
22. Tripathy, S.K., Black, H.B., Goldwasser, E. & Leiden, J.M. Immune responses to transgene-encoded proteins limit the stability of gene expression after injection of replication-defective adenovirus vectors. Nature Med. 2, 545-550 (1996).
23. Yang, Y., Haecker, S.E., Su, Q. & Wilson, J.M. Immunology of gene therapy with adenoviral vectors in mouse skeletal muscle. Hum. Mol. Genet. 5, 1703-1712 (1996).
24. Kozarsky, K.F., Jooss, K., Donahee, M., Strauss, J.F. 3rd & Wilson, J.M. Effective treatment of familial hypercholesterolaemia in the mouse model using adenovirus-mediated transfer of the VLDL receptor gene. Nature Genet. 13, 54-62 (1996).
25. Kay, M.A. et al. Hepatic gene therapy: persistent expression of human alpha-1antitrypsin in mice after direct gene delivery in vivo. Hum. Gene Ther. 3, 641-647 (1992).
26. 1-antitrypsin after adenoviral-mediated gene transfer into mouse hepatocytes. Hepatology 21, 815-819 (1995).
27. Mombaerts, P. et al. RAG-1-deficient mice have no mature B and T lymphocytes. Cell 68, 869-877 (1992).
28. Sykes, R.C., Lin, D., Hwang, S.J., Framson, P.E. & Chinault, A.C. Yeast ARS function and nuclear matrix association coincide in a short sequence from the human HPRT locus. Mol. Gen. Genet. 212, 301-309 (1988).
29. Gerdes, J., Schwab, U., Lemke, H. & Stein, H. Production of a mouse monoclonal antibody reactive with a human nuclear antigen associated with cell proliferation. Int. J. Cancer 31, 13-20 (1983).
30. Lieber, A., He, C.-Y, Kirillova, I. & Kay, M.A. Recombinant adenoviruses with large deletions generated by cre-mediated excision exhibit different biological properties compared with first-generation vectors in vitro and in vivo. J. Virol. 70, 8944-8960 (1996).