Efficient transgene reconstitution with hybrid dual AAV vectors carrying the minimized bridging sequences

Human Gene Therapy

Volumn 22, Issue 1, 2011, Pages 77-83

  Ghosh, Arkasubhra ^a,b   Yue, Yongping ^a   Duan, Dongsheng ^a  

^a University of Missouri   (United States)

^b INSTITUTE OF MOLECULAR AND CELL BIOLOGY   (Singapore)

Author keywords

[No Author keywords available]

Indexed keywords

ALKALINE PHOSPHATASE PLACENTA ISOENZYME; BETA GALACTOSIDASE; DNA FRAGMENT; PARVOVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ARTICLE; CONTROLLED STUDY; DNA SEQUENCE; GENE CASSETTE; GENE EXPRESSION; GENE SEQUENCE; GENE TARGETING; GENETIC ENGINEERING; HOMOLOGOUS RECOMBINATION; MALE; MOUSE; NONHUMAN; TRANSGENE; VIRUS GENOME; VIRUS HYBRID; VIRUS RECOMBINANT;

ANIMALS; BASE SEQUENCE; BETA-GALACTOSIDASE; CELL LINE; DEPENDOVIRUS; GENETIC VECTORS; HUMANS; LAC OPERON; MALE; MICE; MICE, INBRED C57BL; RECOMBINATION, GENETIC; TRANSGENES;

ADENO-ASSOCIATED VIRUS; MURINAE;

EID: 79951904478     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/hum.2010.122     Document Type: Article

References (17)

1. Allalunis-Turner, M.J., Barron, G.M., Day, R.S. 3rd, Dobler, K.D., and Mirzayans, R. (1993). Isolation of two cell lines from a human malignant glioma specimen differing in sensitivity to radiation and chemotherapeutic drugs. Radiat. Res. 134, 349-354. (Pubitemid 23209259)
2. Allen, J.M., Halbert, C.L., and Miller, A.D. (2000). Improved adeno-associated virus vector production with transfection of a single helper adenovirus gene, E4orf6. Mol. Ther. 1, 88-95.
3. Bostick, B., Ghosh, A., Yue, Y., Long, C., and Duan, D. (2007). Systemic AAV-9 transduction in mice is influenced by animal age but not by the route of administration. Gene Ther. 14, 1605-1609. (Pubitemid 350056232)
4. Dong, B., Nakai, H., and Xiao, W. (2010). Characterization of genome integrity for oversized recombinant AAV vector. Mol. Ther. 18, 87-92.
5. Dong, J.Y., Fan, P.D., and Frizzell, R.A. (1996). Quantitative analysis of the packaging capacity of recombinant adeno-associated virus. Hum. Gene Ther. 7, 2101-2112. (Pubitemid 26400735)
6. Duan, D., Yue, Y., Yan, Z., Yang, J., and Engelhardt, J.F. (2000). Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus. J. Clin. Invest. 105, 1573-1587. (Pubitemid 30390308)
7. Ghosh, A., and Duan, D. (2007). Expending adeno-associated viral vector capacity: a tale of two vectors. Biotechnol. Genet. Eng. Rev. 24, 165-177. (Pubitemid 350032948)
8. Ghosh, A., Yue, Y., and Duan, D. (2006). Viral serotype and the transgene sequence influence overlapping adeno-associated viral (AAV) vector-mediated gene transfer in skeletal muscle. J. Gene Med. 8, 298-305.
9. Ghosh, A., Yue, Y., Long, C., Bostick, B., and Duan, D. (2007). Efficient whole-body transduction with trans-splicing adeno-associated viral vectors. Mol. Ther. 15, 750-755. (Pubitemid 46431995)
10. Ghosh, A., Yue, Y., Lai, Y., and Duan, D. (2008). A hybrid vector system expands adeno-associated viral vector packaging capacity in a transgene independent manner. Mol. Ther. 16, 124-130.
11. Grieger, J.C., and Samulski, R.J. (2005). Packaging capacity of adeno-associated virus serotypes: impact of larger genomes on infectivity and postentry steps. J. Virol. 79, 9933-9944. (Pubitemid 41022335)
12. Halbert, C.L., Allen, J.M., and Miller, A.D. (2002). Efficient mouse airway transduction following recombination between AAV vectors carrying parts of a larger gene. Nat. Biotechnol. 20, 697-701. (Pubitemid 34734136)
13. Lai, Y., Yue, Y., Liu, M., Ghosh, A., Engelhardt, J.F., Chamberlain, J.S., and Duan, D. (2005). Efficient in vivo gene expression by trans-splicing adeno-associated viral vectors. Nat. Bio-technol. 23, 1435-1439. (Pubitemid 41679397)
14. Lai, Y., Yue, Y., Bostick, B., and Duan, D. (2010a). Delivering large therapeutic genes for muscle gene therapy. In Muscle gene therapy. D. Duan, ed. (Springer, New York, NY) pp. 205-218.
15. Lai, Y., Yue, Y., and Duan, D. (2010b). Evidence for the failure of adeno-associated virus serotype 5 to package a viral genome>or 8.2 kb. Mol. Ther. 18, 75-79.
16. Wu, Z., Yang, H., and Colosi, P. (2010). Effect of genome size on AAV vector packaging. Mol. Ther. 18, 80-86.
17. Yue, Y., Ghosh, A., Long, C., Bostick, B., Smith, B.F., Kornegay, J.N., and Duan, D. (2008). A single intravenous injection of adeno-associated virus serotype-9 leads to whole body skeletal muscle transduction in dogs. Mol. Ther. 16, 1944-1952.