Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice

Molecular Therapy

Volumn 7, Issue 1, 2003, Pages 101-111

  Nakai, Hiroyuki ^a   Montini, Eugenio ^b   Fuess, Sally ^a   Storm, Theresa A ^a   Meuse, Leonard ^a   Finegold, Milton ^c   Grompe, Markus ^b   Kay, Mark A ^a  

^a STANFORD UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b OREGON HEALTH AND SCIENCE UNIVERSITY   (United States)

^c TEXAS CHILDREN S HOSPITAL   (United States)

Author keywords

Adeno associated virus; Factor IX; Gene therapy; Hereditary tyrosinemia; Integration; Inversted terminal repeat; Linear DNA; Mouse liver; Naked DNA

Indexed keywords

DNA; DOUBLE STRANDED DNA; INTEGRASE; NAKED DNA; PLASMID DNA; TRANSPOSASE;

ADENO ASSOCIATED VIRUS; ANIMAL TISSUE; ARTICLE; CHROMOSOME; CONTROLLED STUDY; DNA INTEGRATION; DNA SEQUENCE; DNA TRANSFECTION; DNA VECTOR; GENE EXPRESSION; GENE TRANSFER; GENETIC ENGINEERING; GENOME; HYDRODYNAMICS; IN VIVO STUDY; INVERTED TERMINAL REPEAT; LIVER CELL; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; TARGET CELL; TRANSGENE; TYROSINEMIA;

ADENO-ASSOCIATED VIRUS; ANIMALIA;

EID: 0037221408     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1525-0016(02)00023-0     Document Type: Article

References (32)

1. Miao, C. H., Thompson, A. R., Loeb, K., and Ye, X. (2001). Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol. Ther. 3: 947-957.
2. Wolff, J. A., Ludtke, J. J., Acsadi, G., Williams, P., and Jani, A. (1992). Long-term persistence of plasmid DNA and foreign gene expression in mouse muscle. Hum. Mol. Genet. 1: 363-369.
3. Carter, P. J., and Samulski, R. J. (2000). Adeno-associated viral vectors as gene delivery vehicles. Int. J. Mol. Med. 6: 17-27.
4. Yang, C. C., et al. (1997). Cellular recombination pathways and viral terminal repeat hairpin structures are sufficient for adeno-associated virus integration in vivo and in vitro. J. Virol. 71: 9231-9247.
5. Grompe, M., et al. (1995). Pharmacological correction of neonatal lethal hepatic dysfunction in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 10: 453-460.
6. Overturf, K., et al. (1996). Hepatocytes corrected by gene therapy are selected in vivo in a murine model of hereditary tyrosinaemia type I. Nat. Genet. 12: 266-273.
7. Grompe, M., St-Louis, M., Demers, S. I., al-Dhalimy, M., Leclerc, B., and Tanguay, R. M. (1994). A single mutation of the fumarylacetoacetate hydrolase gene in French Canadians with hereditary tyrosinemia type I. N. Engl. J. Med. 331: 353-357.
8. Grompe, M., et al. (1993). Loss of fumarylacetoacetate hydrolase is responsible for the neonatal hepatic dysfunction phenotype of lethal albino mice. Genes Dev. 7: 2298-2307.
9. Overturf, K., al-Dhalimy, M., Ou, C. N., Finegold, M., and Grompe, M. (1997). Serial transplantation reveals the stem-cell-like regenerative potential of adult mouse hepatocyte. Am. J. Pathol. 151: 1273-1280.
10. Chen, S. J., Tazelaar, J., Moscioni, A. D., and Wilson, J. M. (2000). In vivo selection of hepatocytes transduced with adeno-associated viral vectors. Mol. Ther. 1: 414-422.
11. Nakai, H., Storm, T. A., and 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: 9451-9463.
12. Chen, Z. Y., Yant, S., He, C. Y., Meuse, L., Shen, S., and Kay, M. A. (2001). Linear DNAs concatemerize in vivo and result in sustained transgene expression in mouse liver. Mol. Ther. 3: 403-410.
13. Nakai, H., Iwaki, Y., Kay, M. A., and Couto, L. B. (1999). Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J. Virol. 73: 5438-5447.
14. Kay, M. A., et al. (1992). Hepatic gene therapy: Persistent expression of human α1-antitrypsin in mice after direct gene delivery in vivo. Hum. Gene Ther. 3: 641-647.
15. Nakai, H., Yant, S. R., Storm, T. A., Fuess, S., Meuse, L., and Kay, M. A. (2001). Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J. Virol. 75: 6969-6976.
16. Diwan, B. A., Henneman, J. R., Rice, J. M., and Nims, R. W. (1996). Enhancement of thyroid and hepatocarcinogenesis by 1,4-bis[2-(3,5-dichloropyridyloxy)]benzene in rats at doses that cause maximal induction of CYP2B. Carcinogenesis 17: 37-43.
17. Ohashi, K., Park, F., and Kay, M. A. (2002). The role of hepatocyte direct hyperplasia on lentiviral-mediated liver transduction in vivo. Hum. Gene Ther. 13: 653-663.
18. Fisher, K. J., Gao, G. P., Weitzman, M. D., DeMatteo, R., Burda, J. F., and Wilson, J. M. (1996). Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J. Virol. 70: 520-532.
19. Ferrari, F. K., Samulski, T., Shenk, T., and Samulski, R. J. (1996). Second-strand synthesis is a rate-limiting step for efficient transduction by recombinant adeno-associated virus vectors. J. Virol. 70: 3227-3234.
20. Garrick, D., Fiering, S., Martin, D. I., and Whitelaw, E. (1998). Repeat-induced gene silencing in mammals. Nat. Genet. 18: 56-59.
21. Miao, C. H., et al. (1998). The kinetics of rAAV integration in the liver. Nat. Genet. 19: 13-15.
22. Miao, C. H., 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: 3793-3803.
23. Miller, D. G., Rutledge, E. A., and Russell, D. W. (2002). Chromosomal effects of adeno-associated virus vector integration. Nat. Genet. 30: 147-148.
24. Duan, D., Yan, Z., Yue, Y., and Engelhardt, J.F. (1999). Structural analysis of adeno-associated virus transduction circular intermediates. Virology 261: 8-14.
25. Xiao, X., Xiao, W., Li, J., and Samulski, R. J. (1997). A novel 165-base-pair terminal repeat sequence is the sole cis requirement for the adeno-associated virus life cycle. J. Virol. 71: 941-948.
26. Burton, M., Nakai, H., Colosi, P., Cunningham, J., Mitchell, R., and Couto, L. (1999). Coexpression of factor Vill heavy and light chain adeno-associated virol vectors produces biologically active protein. Proc. Natl. Acad Sci. USA 96: 12725-12730.
27. Zhang, G., Budker, V., and Wolff, J. A. (1999). High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum. Gene Ther. 10: 1735-1737.
28. Liu, F., Song, Y., and Liu, D. (1999). Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 6: 1258-1266.
29. Nakai, H., et al. (1998). Adeno-associated viral vector-mediated gene transfer of human blood coagulation factor IX into mouse liver. Blood 91: 4600-4607.
30. Yant, S. R., Meuse, L., Chiu, W., Ivics, Z., Izsvak, Z., and Kay, M. A. (2000). Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat. Genet. 25: 35-41.
31. Miller, M. W., et al. (1993). Cloning of the mouse agouti gene predicts a secreted protein ubiquitously expressed in mice carrying the lethal yellow mutation. Genes Dev. 7: 454-467.
32. Nakai, H., Fuess, S., Storm, T. A., Meuse, L. A., and Kay, M. A. (2003). Free DNA ends are essential for concatemerization of synthetic double-stranded adeno-associated virus vector genomes transfected into mouse hepatocytes in vivo. Mol. Ther. 7: .