Self-complementary adeno-associated virus 2 (AAV)-T cell protein tyrosine phosphatase vectors as helper viruses to Improve transduction efficiency of conventional single-stranded AAV vectors in vitro and in vivo

Molecular Therapy

Volumn 10, Issue 5, 2004, Pages 950-957

  Zhong, Li ^a,b   Chen, Linyuan ^a   Li, Yanjun ^a   Qing, Keyun ^a   Weigel Kelley, Kirsten A ^a,b   Chan, Rebecca J ^a   Yoder, Mervin C ^a   Srivastava, Arun ^a,b  

^a INDIANA UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF FLORIDA COLLEGE OF MEDICINE   (United States)

Author keywords

AAV vectors; Gene expression; Gene therapy; Hepatocytes; Second strand DNA synthesis

Indexed keywords

CELL PROTEIN; DOUBLE STRANDED DNA; PARVOVIRUS VECTOR; PROTEIN TYROSINE PHOSPHATASE; TYROSINE; VIRUS DNA;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL TISSUE; ARTICLE; CONTROLLED STUDY; DNA SYNTHESIS INHIBITION; GENE EXPRESSION; GENETIC TRANSDUCTION; HELPER VIRUS; HEMATOPOIETIC CELL; HUMAN; HUMAN CELL; IN VITRO STUDY; IN VIVO STUDY; LIVER CELL; MALE; MOUSE; MOUSE STRAIN; NONHUMAN; PROTEIN DEPHOSPHORYLATION; PROTEIN PHOSPHORYLATION; T LYMPHOCYTE; TRANSGENE; TRANSGENIC MOUSE; VIRAL GENE THERAPY; VIRUS RECOMBINANT;

ANIMALS; CELL LINE, TUMOR; DEPENDOVIRUS; DNA, VIRAL; GENE THERAPY; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HELPER VIRUSES; HEPATOCYTES; HUMANS; MICE; MICE, INBRED C57BL; PROTEIN-TYROSINE-PHOSPHATASE; TRANSDUCTION, GENETIC;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 2; MURINAE; MUS MUSCULUS;

EID: 7044241347     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2004.07.018     Document Type: Article

References (37)

1. K.I. Berns C. Giraud Biology of adeno-associated virus Curr. Top. Microbiol. Immunol. 218 1996 1-23
2. N. Muzyczka Use of adeno-associated virus as a general transduction vector for mammalian cells Curr. Top. Microbiol. Immunol. 158 1992 97-129
3. K.J. Fisher et al. Transduction with recombinant adeno-associated virus for gene therapy is limited by leading-strand synthesis J. Virol. 70 1996 520-532
4. F.K. Ferrari T. Samulski T. Shenk R.J. Samulski Second-strand synthesis is a rate limiting step for efficient transduction by recombinant adeno-associated virus vectors J. Virol. 70 1996 3227-3234
5. C. Mah et al. Adeno-associated virus 2-mediated gene transfer: Role of epidermal growth factor receptor protein tyrosine kinase in transgene expression J. Virol. 72 1998 9835-9841
6. K.Y. Qing et al. Adeno-associated virus type 2-mediated gene transfer: correlation of tyrosine phosphorylation of the cellular single-stranded D sequence-binding protein with transgene expression in human cells in vitro and murine tissues in vivo J. Virol. 72 1998 1593-1599
7. K.Y. Qing et al. Adeno-associated virus type 2-mediated gene transfer: role of cellular FKBP52 protein in transgene expression J. Virol. 75 2001 8968-8976
8. K.Y. Qing et al. Adeno-associated virus type 2-mediated gene transfer: role of cellular T-cell protein tyrosine phosphatase in transgene expression in established cell lines in vitro and transgenic mice in vivo J. Virol. 77 2003 2741-2746
9. K.Y. Qing et al. Role of tyrosine phosphorylation of a cellular protein in adeno-associated virus 2-mediated transgene expression Proc. Natl. Acad. Sci. USA 94 1997 10879-10884
10. X.-S. Wang K.Y. Qing S. Ponnazhagan A. Srivastava Adeno-associated virus type 2 DNA replication in vivo: Mutation analyses of the D sequence in viral inverted terminal repeats J. Virol. 71 1997 3077-3082
11. S. Ponnazhagan et al. Adeno-associated virus 2-mediated gene transfer in vivo: Organ-tropism and expression of transduced sequences in mice Gene 190 1997 203-210
12. M.J. Czar R.H. Lyons M.J. Welsh J.-M. Renoir W.B. Pratt Evidence that the FK506-binding protein 56 is required for trafficking of the glucocorticoid receptor from the cytoplasm to the nucleus Mol. Endocrinol. 9 1995 1549-1560
13. Y. Miyata et al. Phosphorylation of the immunosuppressant FK506-binding protein FKBP52 by casein kinase II: Regulation of HSP90-binding activity of FKBP52 Proc. Natl. Acad. Sci. USA 94 1997 14500-14505
14. J.A. Lorenzen C.Y. Dadabay E.H. Fischer COOH-terminal sequence motifs target the T cell protein tyrosine phosphatase to the ER and nucleus J. Biol. Chem. 131 1995 631-643
15. K.E. You-Ten et al. Impaired bone marrow microenvironment and immune function in T cell protein tyrosine phosphatase-deficient mice J. Exp. Med. 186 1997 683-693
16. L. Zhong et al. Improved transduction of primary murine hepatocytes by recombinant adeno-associated virus 2 vectors in vivo Gene Ther. 11 2004 1165-1169
17. D.M. McCarty P.E. Monahan R.J. Samulski Self-complementary recombinant adeno-associated virus (scAAV) vectors promote efficient transduction independently of DNA synthesis Gene Ther. 8 2001 1248-1254
18. J. Hansen K.Y. Qing H.-J. Kwon C. Mah A. Srivastava Impaired intracellular trafficking of adeno-associated virus type 2 vectors limits efficient transduction of murine fibroblasts J. Virol. 74 2000 992-997
19. R.O. Snyder et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors Nat. Genet. 16 1997 270-276
20. H. Nakai T.A. Storm M.A. Kay Increasing the size of rAAV-mediated expression cassettes in vivo by intermolecular joining of two complementary vectors Nat. Biotechnol. 18 2000 527-532
21. S.F. Chen J. Tazelaar J.M. Wilson Selective repopulation of normal mouse liver by hepatocytes transduced in vivo with recombinant adeno-associated virus Hum. Gene Ther. 12 2001 45-50
22. S. Song et al. Stable therapeutic serum levels of human 1 antitrypsin (AAT) after portal vein injection of recombinant adeno-associated virus (rAAV) vectors Gene Ther. 8 2001 1299-1306
23. J. Hansen K.Y. Qing A. Srivastava Infection of purified nuclei by adeno-associated virus Mol. Ther. 4 2001 289-296
24. K.Y. Qing C. Mah J. Hansen S.Z. Zhou V.J. Dwarki A. Srivastava Human fibroblast growth factor receptor 1 is a co-receptor for infection by adeno-associated virus 2 Nat. Med. 5 1999 71-77
25. C. Summerford J.S. Bartlett R.J. Samulski αVβ5 integrin: A co-receptor for adeno-associated virus 2 infection Nat. Med. 5 1999 78-82
26. C. Summerford R.J. Samulski Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions J. Virol. 72 1998 1438-1445
27. J.S. Bartlett R. Wilcher R.J. Samulski Infectious entry pathway of adeno-associated virus and adeno-associated virus vectors J. Virol. 74 2000 2777-2785
28. D. Duan Q. Li A.W. Kao Y. Yue J.E. Pessin J.F. Engelhardt Dynamin is required for recombinant adeno-associated virus type 2 infection J. Virol. 73 1999 10371-10376
29. D. Duan Y. Yue Z. Yan J. Yang J.F. Engelhardt Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus J. Clin. Invest. 105 2000 1573-1587
30. J. Hansen K.Y. Qing A. Srivastava Adeno-associated virus type 2-mediated gene transfer: Altered endocytic processing enhances transduction efficiency in murine fibroblasts J. Virol. 75 2001 4080-4090
31. S. Sanlioglu P.K. Benson J. Yang E.M. Atkinson T. Reynolds J.F. Engelhardt Endocytosis and nuclear trafficking of adeno-associated virus type 2 are controlled by rac 1 and phosphatidylinositol-3 kinase activation J. Virol. 74 2000 9184-9196
32. G. Seisenberger M.U. Ried T. Endress H. Buning M. Hallek C. Brauchle Real-time single-molecule imaging of the infection pathway of an adeno-associated virus Science 294 2001 1929-1932
33. W. Xiao K.H. Warrington Jr. P. Hearing J. Hughes N. Muzyczka Adenovirus-facilitated nuclear translocation of adeno-associated virus type 2 J. Virol. 76 2002 11505-11517
34. C.E. Thomas T.A. Storm Z. Huang M.A. Kay Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors J. Virol. 78 2004 3110-3122
35. X.-S. Wang S. Ponnazhagan A. Srivastava Rescue and replication signals of the adeno-associated virus 2 genome J. Mol. Biol. 250 1995 573-580
36. A. Auricchio M. Hildinger E. O'Connor G.P. Gao J.M. Wilson Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column Hum. Gene. Ther. 12 2001 71-76
37. D.M. Kube A. Srivastava Quantitative DNA slot blot analysis: Inhibition of DNA binding to membranes by magnesium ions Nucleic Acids Res. 25 1997 3375-3376