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Cited by 367

The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.

Rapid and highly efficient transduction by double-stranded adeno-associated virus vectors in vitro and in vivo

Wang, Z, Ma, H-I, Li, J, Sun, L, Zhang, J, Xiao, X·2003·Gene Therapy
DOI 10.1038/sj.gt.3302133
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Abstract

Adeno-associated virus (AAV) is a promising gene vector based on a single-stranded (ss) DNA virus. Its transgene expression requires the conversion of ssDNA to double-stranded (ds) genome, a slow process responsible for the delayed transduction and occasional inefficiency. By mutating the inverted terminal repeat, we have made novel AAV vectors that predominantly package the self-complementary dsDNA genome. The dsAAV consistently demonstrated superior and accelerated transduction in vitro and in vivo. Dramatic increases in transgene expression were observed in most of the cell lines examined, including B16 melanoma and 3LL lung cancer that are difficult to be transduced by the conventional ssAAV vectors. Similar increases were also observed in vivo in a variety of tissues including muscle and liver. The dsAAV transduced a vast majority of the hepatocytes for more than 6 months, while the ssAAV transduced only a small fraction. In addition to circumventing the requirement for DNA synthesis, the dsAAV exhibited higher in vivo DNA stability and more effective circularization than the ssAAV, suggesting potential molecular mechanisms for the faster, stronger and prolonged transgene expression.

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Crossref · 10.1038/sj.gt.3302133

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Research works printed but not linked: 25

  1. Samulski RJ, Sally M, Muzyczka N . Adeno-associated viral vectors. In: Friedmann T (ed). Development of Human Gene Therapy. Cold Spring Harbor Laboratory Press: Cold Spring Harbor, 1999, pp 131–172.
  2. Fisher KJ et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis. J Virol 1996; 70: 520–532. · DOI
  3. Nakai H, Storm TA, Kay MA . Recruitment of single-stranded recombinant adeno-associated virus vector genomes and intermolecular recombination are responsible for stable transduction of liver in vivo. J Virol 2000; 74: 9451–9463. · DOI · Paper page
  4. Lewin AS et al. Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa [published erratum appears in Nat Med 1998 Sep;4(9):1081]. Nat Med 1998; 4: 967–971. · DOI
  5. Ma HI et al. Suppression of intracranial human glioma growth after intramuscular administration of an adeno-associated viral vector expressing angiostatin. Cancer Res 2002; 62: 756–763.
  6. Shimazaki K et al. Adeno-associated virus vector-mediated bcl-2 gene transfer into post-ischemic gerbil brain in vivo: prospects for gene therapy of ischemia-induced neuronal death. Gene Therapy 2000; 7: 1244–1249. · DOI
  7. Miao CH et al. Nonrandom transduction of recombinant adeno-associated virus vectors in mouse hepatocytes in vivo: cell cycling does not influence hepatocyte transduction. J Virol 2000; 74: 3793–3803. · DOI
  8. Song S et al. Stable therapeutic serum levels of human alpha-1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors. Gene Therapy 2001; 8: 1299–1306. · DOI
  9. Mount JD et al. Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy. Blood 2002; 99: 2670–2676. · DOI · Paper page
  10. Chao H, Mao L, Bruce AT, Walsh CE . Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood 2000; 95: 1594–1599. · DOI
  11. Xu L et al. CMV-beta-actin promoter directs higher expression from an adeno-associated viral vector in the liver than the cytomegalovirus or elongation factor 1 alpha promoter and results in therapeutic levels of human factor X in mice. Hum Gene Ther 2001; 12: 563–573. · DOI
  12. Qing K et al. Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression. Proc Natl Acad Sci USA 1997; 94: 10879–10884. · DOI
  13. King JA, Dubielzig R, Grimm D, Kleinschmidt JA . DNA helicase-mediated packaging of adeno-associated virus type 2 genomes into preformed capsids. EMBO J 2001; 20: 3282–3291. · DOI · Paper page
  14. Clark KR, Liu X, McGrath JP, Johnson PR . Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther 1999; 10: 1031–1039. · DOI
  15. Girod A et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med 1999; 5: 1052–1056. · DOI · Paper page
  16. Nakai H et al. A limited number of transducible hepatocytes restricts a wide-range linear vector dose response in recombinant adeno-associated virus-mediated liver transduction. J Virol 2002; 76: 11343–11349. · DOI
  17. Hansen J et al. Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts. J Virol 2000; 74: 992–996. · DOI
  18. Duan D et al. Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue [published erratum appears in J Virol 1999 Jan;73(1):861]. J Virol 1998; 72: 8568–8577. · DOI · Paper page
  19. Wang JC, Lynch AS . Transcription and DNA supercoiling. Curr Opin Genet Dev 1993; 3: 764–768. · DOI
  20. Song S, Laipis PJ, Berns KI, Flotte TR . Effect of DNA-dependent protein kinase on the molecular fate of the rAAV2 genome in skeletal muscle. Proc Natl Acad Sci USA 2001; 98: 4084–4088. · DOI
  21. Nakai H et al. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 2001; 75: 6969–6976. · DOI · Paper page
  22. Xiao X et al. Adeno-associated virus (AAV) vector antisense gene transfer in vivo decreases GABA(A) alpha1 containing receptors and increases inferior collicular seizure sensitivity. Brain Res 1997; 756: 76–83. · DOI
  23. McCaffrey AP et al. RNA interference in adult mice. Nature 2002; 418: 38–39. · DOI
  24. Xiao X, Li J, Samulski RJ . Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224–2232. · DOI · Paper page
  25. Snyder R, Xiao X, Samulski RJ . Production of recombinant adeno-asociated viral vectors. In: Smith D (ed). Current Protocols in Human Genetics. John Wiley & Sons Ltd: New York, 1996: 12.1.1–12.2.23.

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Metadata

Authors
Wang, Z., Ma, H.-I., Li, J., Sun, L., Zhang, J., & Xiao, X.
Venue
Gene Therapy
Publisher
Springer Science and Business Media LLC
Published
2003-11-17
Volume
10
Issue
26
Pages
2105-2111
ISSN
  • 0969-7128
  • 1476-5462
DOI
10.1038/sj.gt.3302133
Cited by
367
References
6
Language
en
Licence

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