Experimental Hematology
Volume 36, Issue 7 , Pages 823-831 , July 2008

Tightly regulated gene expression in human hematopoietic stem cells after transduction with helper-dependent Ad5/35 vectors

  • Hongje Wang

      Affiliations

    • Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Wash., USA
  • ,
  • Hua Cao

      Affiliations

    • Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Wash., USA
  • ,
  • Martin Wohlfahrt

      Affiliations

    • Fred Hutchinson Cancer Research Center, Seattle, Wash., USA
  • ,
  • Hans-Peter Kiem

      Affiliations

    • Fred Hutchinson Cancer Research Center, Seattle, Wash., USA
  • ,
  • André Lieber

      Affiliations

    • Division of Medical Genetics, Department of Medicine, University of Washington, Seattle, Wash., USA
    • Department of Pathology, University of Washington, Seattle, Wash., USA
    • Corresponding Author InformationOffprint requests to: André Lieber, M.D., Ph.D., Division of Medical Genetics, University of Washington, Box 357720, Seattle, WA 98195

Received 26 September 2007 ,Revised 7 December 2007 ,Accepted 18 January 2008.

References 

  1. Wang H, Lieber A. A helper-dependent capsid-modified adenovirus vector expressing adeno-associated virus rep78 mediates site-specific integration of a 27-kilobase transgene cassette. J Virol. 2006;80:11699–11709
  2. Miller JC, Holmes MC, Wang J, et al. An improved zinc-finger nuclease architecture for highly specific genome editing. Nat Biotechnol. 2007;25:778–785
  3. Yanez-Munoz RJ, Balaggan KS, MacNeil A, et al. Effective gene therapy with nonintegrating lentiviral vectors. Nat Med. 2006;12:348–353
  4. Shayakhmetov DM, Papayannopoulou T, Stamatoyannopoulos G, Lieber A. Efficient gene transfer into human CD34(+) cells by a retargeted adenovirus vector. J Virol. 2000;74:2567–2583
  5. Gaggar A, Shayakhmetov D, Lieber A. CD46 is a cellular receptor for group B adenoviruses. Nat Med. 2003;9:1408–1412
  6. Yotnda P, Onishi H, Heslop HE, et al. Efficient infection of primitive hematopoietic stem cells by modified adenovirus. Gene Ther. 2001;8:930–937
  7. Nilsson M, Karlsson S, Fan X. Functionally distinct subpopulations of cord blood CD34+ cells are transduced by adenoviral vectors with serotype 5 or 35 tropism. Mol Ther. 2004;9:377–388
  8. Lieber A, He CY, Kirillova I, Kay MA. 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. 1996;70:8944–8960
  9. Kochanek S, Schiedner G, Volpers C. High-capacity ‘gutless’ adenoviral vectors. Curr Opin Mol Ther. 2001;3:454–463
  10. Wang H, Shayakhmetov DM, Leege T, et al. A capsid-modified helper-dependent adenovirus vector containing the beta-globin locus control region displays a nonrandom integration pattern and allows stable, erythroid-specific gene expression. J Virol. 2005;79:10999–11013
  11. Balamotis MA, Huang K, Mitani K. Efficient delivery and stable gene expression in a hematopoietic cell line using a chimeric serotype 35 fiber pseudotyped helper-dependent adenoviral vector. Virology. 2004;324:229–237
  12. Hearing P, Shenk T. The adenovirus type 5 E1A transcriptional control region contains a duplicated enhancer element. Cell. 33:695–703.
  13. Grable M, Hearing P. cis and trans requirements for the selective packaging of adenovirus type 5 DNA. J Virol. 1992;66:723–731
  14. Shi Q, Wang Y, Worton R. Modulation of the specificity and activity of a cellular promoter in an adenoviral vector. Hum Gene Ther. 1997;8:403–410
  15. Friedman JM, Babiss LE, Clayton DF, Darnell JE. Cellular promoters incorporated into the adenovirus genome: cell specificity of albumin and immunoglobulin expression. Mol Cell Biol. 1986;6:3791–3797
  16. Xu ZL, Mizuguchi H, Mayumi T, Hayakawa T. Regulated gene expression from adenovirus vectors: a systematic comparison of various inducible systems. Gene. 2003;309:145–151
  17. Salucci V, Scarito A, Aurisicchio L, et al. Tight control of gene expression by a helper-dependent adenovirus vector carrying the rtTA2(s)-M2 tetracycline transactivator and repressor system. Gene Ther. 2002;9:1415–1421
  18. Steinwaerder DS, Lieber A. Insulation from viral transcriptional regulatory elements improves inducible transgene expression from adenovirus vectors in vitro and in vivo. Gene Ther. 2000;7:556–567
  19. Ye X, Liang M, Meng X, et al. Insulation from viral transcriptional regulatory elements enables improvement to hepatoma-specific gene expression from adenovirus vectors. Biochem Biophys Res Commun. 2003;307:759–764
  20. Majem M, Cascallo M, Bayo-Puxan N, Mesia R, Germa JR, Alemany R. Control of E1A under an E2F-1 promoter insulated with the myotonic dystrophy locus insulator reduces the toxicity of oncolytic adenovirus Ad-Delta24RGD. Cancer Gene Ther. 2006;13:696–705
  21. Hallenbeck PL, Chang YN, Hay C, et al. A novel tumor-specific replication-restricted adenoviral vector for gene therapy of hepatocellular carcinoma. Hum Gene Ther. 1999;10:1721–1733
  22. Szulc J, Wiznerowicz M, Sauvain MO, Trono D, Aebischer P. A versatile tool for conditional gene expression and knockdown. Nat Methods. 2006;3:109–116
  23. Gossen M, Freundlieb S, Bender G, Muller G, Hillen W, Bujard H. Transcriptional activation by tetracyclines in mammalian cells. Science. 1995;268:1766–1769
  24. Hartigan-O'Connor D, Barjot C, Salvatori G, Chamberlain JS. Generation and growth of gutted adenoviral vectors. Methods Enzymol. 2002;346:224–246
  25. Barjot C, Hartigan-O'Connor D, Salvatori G, Scott JM, Chamberlain JS. Gutted adenoviral vector growth using E1/E2b/E3-deleted helper viruses. J Gene Med. 2002;4:480–489
  26. Avanzi GC, Lista P, Giovinazzo B, et al. Selective growth response to IL-3 of a human leukaemic cell line with megakaryoblastic features. Br J Haematol. 1988;69:359–366
  27. Sandig V, Youil R, Bett AJ, et al. Optimization of the helper-dependent adenovirus system for production and potency in vivo. Proc Natl Acad Sci U S A. 2000;97:1002–1007
  28. Moosmann P, Georgiev O, Thiesen HJ, Hagmann M, Schaffner W. Silencing of RNA polymerases II and III-dependent transcription by the KRAB protein domain of KOX1, a Kruppel-type zinc finger factor. Biol Chem. 1997;378:669–677
  29. Tate VE, Philipson L. Parental adenovirus DNA accumulates in nucleosome-like structures in infected cells. Nucleic Acids Res. 1979;6:2769–2785
  30. Mirza MA, Weber J. Structure of adenovirus chromatin. Biochim Biophys Acta. 1982;696:76–86
  31. Carlson CA, Shayakhmetov DM, Lieber A. An adenoviral expression system for AAV Rep78 using homologous recombination. Mol Ther. 2002;6:91–98

PII: S0301-472X(08)00050-7

doi: 10.1016/j.exphem.2008.01.014

Experimental Hematology
Volume 36, Issue 7 , Pages 823-831 , July 2008