Skip to main content
SpringerLink
Log in
Book cover

Adeno-Associated Virus (AAV) Vectors in Gene Therapy pp 61–73Cite as

Adeno-associated Virus Vectors for Gene Therapy of the Hematopoietic System

  • Chapter

Part of the book series: Current Topics in Microbiology and Immunology ((CT MICROBIOLOGY,volume 218))

Abstract

Over the past 30 years, bone marrow transplantation has come to be accepted as standard curative therapy for many pathophysiologic conditions (Forman et al. 1994) including malignancies (Brenner et al. 1993) and metabolic diseases (Blaese 1993). Thus, the reconstitution of the hematopoietic system with normal allogeneic cells capable of providing long term engraftment with disease-free, physiologically functional cells has been used to treat various inherited diseases including severe combined immunodeficiency disease (SCID), lysosomal storage disorders and various hemoglobinopathies. However, difficulties in acquisition of histocompatible marrow cells for transplantation and onset of graft vs host disease has led to the continued search for better therapeutic strategies. Meanwhile, the delineation of the genetic bases of many inherited diseases and the subsequent isolation of appropriate wild-type genes promoted the feasibility of disease correction at the DNA level. To this end, much effort has focussed upon the development of both the identification of genetic strategies as well as efficient methods of delivery of therapeutic genes to appropriate target cells.

This is a preview of subscription content, log in via an institution.

Chapter
USD   29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD   84.99
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD   109.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Learn about institutional subscriptions

Preview

Unable to display preview. Download preview PDF.

Unable to display preview. Download preview PDF.

References

  • Alexander IE, Russell DW, Miller AD (1994) DNA-damaging agents greatly increase the transduction of nondividing cells by adeno-associated virus vectors. J Virol 68: 8282–8287

    PubMed  CAS  Google Scholar 

  • Baum CM, Weissman IL, Tsukamoto AS, Buckle AM, Peault B (1992) Isolation of a candidate human hematopoietic stem-cell population. Proc Natl Acad Sci USA 89: 2804–2808

    Article  PubMed  CAS  Google Scholar 

  • Berns K, Pinkerton T, Thomas GF, Hoggan MN (1975) Detection of adeno-associated virus (AAV)-specific nucleotide sequences in DNA isolated from latently infected Detroit 6 cells. Virology 68: 556–560

    Article  PubMed  CAS  Google Scholar 

  • Berns KI, Bohenzky RA (1987) Adeno-associated viruses: an update. Adv Virus Res 32: 243–306

    Article  PubMed  CAS  Google Scholar 

  • Bertrand E, Carbonelle E, Lee NS, Zhou C, Kohn D, Good P, Engelke D, Chatterjee S, Rossi J (1996) Development of viral vectors for expression of ribozyme and antisense RNAs in mammalian cells. Nucleic Acids Res (submitted)

    Google Scholar 

  • Beutler E (1993) Gaucher disease as a paradigm of current issues regarding single gene mutations of humans. Proc Natl Acad Sci USA 90: 5384–5390

    Article  PubMed  CAS  Google Scholar 

  • Blaese RM (1993) Development of gene therapy for immunodeficiency: adenosine deaminase deficiency. Pediatr Res 33: S49

    Article  PubMed  CAS  Google Scholar 

  • Brar D, Wong KK Jr, Permana P, Chatterjee S (1994) Promoter interactions in adeno-associated virus vectors encoding multiple gene cassettes: potential use in anti-oncogene vector design. Cancer Gene Ther 1: 321

    Google Scholar 

  • Brenner MK, Rill DR, Holladay MS, Heslop HE, Moen RC, Buschle M, Krance RA, Santana VM, Anderson WF, Ihle JN (1993) Gene marking to determine whether autologous marrow infusion restores long-term haemopoiesis in cancer patients. Lancet 342: 1134–1137

    Article  PubMed  CAS  Google Scholar 

  • Buller RML, Janik J, Sebring ED, Rose JA (1981) Herpes simplex virus types 1 and 2 completely help adenovirus-associated virus replication. J Virol 40: 241–247

    PubMed  CAS  Google Scholar 

  • Carter RB, Abrams-Ogg AC, Dick JE, Kruth SA, Valli VE, Kamel-Reid S, Dube ID (1992) Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors. Blood 79: 356

    PubMed  CAS  Google Scholar 

  • Challita PM, Kohn DB (1994) Lack of expression from a retroviral vector after transduction of murine hematopoietic stem cells is associated with methylation in vivo. Proc Natl Acad Sci USA 91: 2567–2571

    Article  PubMed  CAS  Google Scholar 

  • Chatterjee S, Wong KK (1993) Adeno-associated virus vectors for the delivery of antisense RNA Methods (a companion to Methods in Enzymology) 5: 51–59

    CAS  Google Scholar 

  • Chatterjee S, Johnson PR, Wong KK (1992) Dual target inhibition of HIV-1 in vitro with an adeno-associated virus-based antisense vector. Science 258: 1485–1488

    Article  PubMed  CAS  Google Scholar 

  • Chatterjee S, Wong KK, Lu D, Permana P, Podsakoff GM (1995) Novel approaches for efficient gene transfer into hematopoietic progenitor cells: the use of adeno-associated virus vectors. Bone Marrow Transplant 15: 5309–5313

    Google Scholar 

  • Dexter TM, Allen TD, Lajtha LG (1977) Conditions controlling the proliferation of haemopoietic stem cells in vitro. J Cell Physiol 91: 335–344

    Article  PubMed  CAS  Google Scholar 

  • Dunbar CE, Cottier-Fox M, O’Shaughnessy JA, Doren S, Carter C, Berenson R, Brown S, Moen RC, Greenblatt J, Stewart FM, Leitman SF, Wilson WH, Cowan K, Young NS, Nienhuis AW (1995) Retrovirally marked CD34-enriched peripheral blood and bone marrow cells contribute to long-term engraftment after autologous transplantation. Blood 85: 3048–3057

    PubMed  CAS  Google Scholar 

  • Fisher-Adams G, Wong KK, Podsakoff GM, Forman S, Chatterjee S (1996) Integration of adeno-associated virus vector genomes in human CD34 cells following transduction. Blood 88 (in press)

    Google Scholar 

  • Flotte TR, Afione SA, Conrad C, McGrath SA, Solow R, Oka H, Zeitlin PL, Guggino WB, Carter BJ Stable in vivo expression of the cystic fibrosis transmembrane conductance regulator with an adeno-associated virus vector. Proc Natl Acad Sci USA 90: 10613

    Google Scholar 

  • Flotte TR, Afione SA, Zeitlin PL (1994) Adeno-associated virus vector gene expression occurs in nondividing cells in the absence of vector DNA integration. Am J Respir Cell Mol Biol 11: 517–521

    PubMed  CAS  Google Scholar 

  • Forman SJ, Blume KG, Thomas ED (eds) (1994) Bone marrow transplantation. Blackwell Scientific, Cambridge

    Google Scholar 

  • Goodman S, Xiao X, Donahue RE, Moulton A, Miller J, Walsh C, Young NS, Samulski RJ, Nienhuis AW (1994) Recombinant adeno-associated virus-mediated gene transfer into hematopoietic progenitor cells. Blood 84: 1492–1500

    PubMed  CAS  Google Scholar 

  • Halbert CL, Alexander IE, Wolgamot GM, Miller AD (1995) Adeno-associated virus vectors transduce primary cells much less efficiently than immortalized cells. J Virol 69: 1473–1479

    PubMed  CAS  Google Scholar 

  • Janik JE, Huston MM, Rose JA (1981) Locations of adenovirus genes required for the replication of adeno-associated virus. Proc Natl Acad Sci USA 78: 1925–1930

    Article  PubMed  CAS  Google Scholar 

  • Kantoff PW, Gillio AP, McKachlin JR, Bordignon C, Eglitis MA, Kernan NA, Moen RC, Kohn DB, Yu SF, Karson E, Karlsson S, Zwiebel JA, Gilboa E, Blaese RM, Nienhuis A, O’Reilly RJ, Anderson WF (1987) Expression of human adenosine deaminase in nonhuman primates after retrovirusmediated gene transfer. J Exp Med 166: 219–234

    Article  PubMed  CAS  Google Scholar 

  • Kaplitt MG, Leone P, Samulski RJ, Xiao X, Pfaff DW, O’Malley KL, During MJ (1994) Long-term gene expression and phenotypic correction using adeno-associated virus vectors in the mammalian brain. Nature Genet 8: 148–153

    Article  PubMed  CAS  Google Scholar 

  • Kotin RM, Siniscalco M, Samulski RJ, Zhu XD, Hunter L, Laughlin CA, Mclaughlin S, Muzyczka N, Rocchi M, Berns KI (1990) Site-specific integration by adeno-associated virus. Proc Natl Acad Sci USA 87: 2211–2215

    Article  PubMed  CAS  Google Scholar 

  • Laughlin CA, Tratschin J-D, Coon H, Carter BJ (1983) Cloning of infectious adeno-associated virus genomes in bacterial plasmids. Gene 23: 65–73

    Article  PubMed  CAS  Google Scholar 

  • Lebkowski JS, Mally MM, Okarma TB, Lerch LB (1988) Adeno-associated virus: a vector system for efficient introduction and integration of DNA into a variety of mammalian cell types. Mol Cell Biol 8: 3988–3996

    PubMed  CAS  Google Scholar 

  • Lu D, Chatterjee S, Brar D, Wong KK Jr (1994a) High efficiency in vitro cleavage of transcripts arising from the major transforming genes of human papillomavirus type 16 mediated by ribozymes transcribed from an adeno-associated virus-based vector. Cancer Gene Therapy. 1: 267–277

    PubMed  CAS  Google Scholar 

  • Lu D, Wong KK Jr, Podsakoff G, Chatterjee S (1994b) Stable and efficient adeno-associated virusmediated gene transfer into primitive human marrow-derived hematopoietic cells in long term culture. Blood 84 [Suppl 1]: 360a

    Google Scholar 

  • Luskey BD, Rosenblatt M, Zsebo K, Williams DA (1992) Stem cell factor, interleukin-3, and interleukin-6 promote retroviral-mediated gene transfer into murine hematopoietic stem cells. Blood 80: 396–402

    PubMed  CAS  Google Scholar 

  • McLaughlin SK, Collis P, Hermonat PL, Muzyczka N (1988) Adeno-associated virus general transduction vectors: analysis of proviral structures. J Virol 62: 1963–1973

    PubMed  CAS  Google Scholar 

  • Miller AD (1990) Retrovirus packaging cells. Hum Gene Ther 1: 5–14

    Article  PubMed  CAS  Google Scholar 

  • Miller DG, Adam MA, Miller AD (1990) Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection. Mol Cell Biol 10: 4239–4242

    PubMed  CAS  Google Scholar 

  • Miller AD, Miller DG, Garcia JV, Lynch CM (1993) Use of retroviral vectors for gene transfer and expression. Methods Enzymol 217: 581–599

    Article  PubMed  CAS  Google Scholar 

  • Miller JL, Donahue RE, Sellers SE, Samulski RJ, Young NS, Nienhuis AW (1994) Recombinant adeno-associated virus (AV)-mediated expression of a human gamma-globin gene in human progenitorderived erythroid cells. Proc Natl Acad Sci USA 91: 10183–10187

    Article  PubMed  CAS  Google Scholar 

  • Muzyczka N (1992) Use of AAV as a general transduction vector for mammalian cells. In: Muzyczka N (ed) Viral expression vectors. Springer, Berlin Heidelberg New York, pp 97–129 (Current topics in microbiology and immunology, vol 158)

    Chapter  Google Scholar 

  • Nolta JA, Crooks GM, Overell RW, Williams DE, Kohn DB (1992) Retroviral vector-mediated gene transfer into primitive human hematopoietic progenitor cells: effects of mast cell growth factor (MGF) combined with other cytokines. Exp Hematol 20: 1065–1071

    PubMed  CAS  Google Scholar 

  • Ogawa M (1993) Differentiation and proliferation of hematopoietic stem cells. Blood 81: 2844–2853

    PubMed  CAS  Google Scholar 

  • Podsakoff G, Wong KK Jr, Chatterjee S (1994a) Stable and efficient gene transfer into non-dividing cells by adeno-associated virus (AAV)-based vectors. J Virol 68: 656–5666

    Google Scholar 

  • Podsakoff G, Shaughnessy EA, Lu D, Wong KK Jr, Chatterjee S (1994b) Long term in vivo reconstitution with murine marrow cells transduced with an adeno-associated virus vector. Blood 84 [Suppl]: 256a

    Google Scholar 

  • Roe TY, Reynolds TC, Yu G, Brown PO (1993) Integration of murine leukemia virus DNA depends on mitosis. EMBO J 12: 2099–2108

    PubMed  CAS  Google Scholar 

  • Russell DW, Miller AD, Alexander IE (1994) Adeno-associated virus vectors preferentially transduce cells in S phase. Proc Natl Acad Sci USA 91: 8915–8919

    Article  PubMed  CAS  Google Scholar 

  • Samulski RJ, Berns KI, Tan M, Muzyczka N (1982) Cloning of adeno-associated virus into PBR322: rescue of intact virus from recombinant plasmid in human cells. Proc Natl Acad Sci USA 79: 2077–2081

    Article  PubMed  CAS  Google Scholar 

  • Samulski RJ, Chang L-S, Shenk T (1989) Helper-free stocks of recombinant adeno-associated viruses: normal integration does not require viral gene expression. J Virol 63: 3822–3828

    PubMed  CAS  Google Scholar 

  • Samulski RJ, Zhu X, Xiao X, Brook JD, Housman DE, Epstein N, Hunter LA (1991) Targeted integration of adeno-associated virus (AAV) into human chromosome 19. EMBO J 10: 3941–3950

    PubMed  CAS  Google Scholar 

  • Sullenger BA, Gallardo HF, Ungers GE, Gilboa E (1990) Overexpression of TAR sequences renders cells resistant to human immunodeficiency virus replication. Cell 63: 601–608

    Article  PubMed  CAS  Google Scholar 

  • Sutherland JH, Lansdorp PM, Kenkelman DH, Eaves AC, Eaves CJ (1990) Functional characterization of individual human hematopoietic stem cells cultured at limiting dilution on supportive marrow stormal layers. Proc Natl Acad Sci USA 87: 3584–3588

    Article  PubMed  CAS  Google Scholar 

  • Sutherland HJ, Eaves CJ, Lansdorp PM, Thacker JD, Hogge DE (1991) Differential regulation of primitive human hematopoietic cells in long-term cultures maintained on genetically engineered murine stromal cells. Blood 78: 666–672

    PubMed  CAS  Google Scholar 

  • Walsh CE, Nienhuis AW, Samulski RJ, Brown MG, Miller JL, Young NS, Liu JM (1994) Phenotypic correction of Fanconi anemia in human hematopoietic cells with a recombinant adeno-associated virus vector. J Clin Invest 94: 1440–1448

    Article  PubMed  CAS  Google Scholar 

  • Wong KK Jr, Rose JA, Chatterjee S (1991) Restriction of HSV-1 production in cell lines transduced with an antisense viral vector targeting the HSV-1 ICP4 gene. Vaccine 91: 183–189

    Google Scholar 

  • Yu M, Poeschla E, Wong-Staal F (1994) Progress towards gene therapy for HIV infection. Gene Ther 1: 13–26

    PubMed  CAS  Google Scholar 

  • Zhou SZ, Broxmeyer HE, Cooper S, Harrington M, Srivastava A (1993) Adeno-associated virus 2-mediated gene transfer in murine hematopoietic progenitor cells. Exp Hematol 21: 928–933

    PubMed  CAS  Google Scholar 

  • Zhou SZ, Cooper S, Kang LY, Ruggieri L, Heimfeld S, Srivastava A, Broxmeyer HE (1994) Adeno-associated virus 2-mediated high efficiency gene transfer into immature and mature subsets of hematopoietic progenitor cells in human umbilical cord blood. J Exp Med 179: 1867–1875

    Article  PubMed  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Division of Pediatrics, City of Hope National Medical Center, 1500 E. Duarte Road, 91010, Duarte, CA, USA

    S. Chatterjee

  2. Department of Hematology and Bone Marrow Transplantation, City of Hope National Medical Center, 1500 E. Duarte Road, 91010, Duarte, CA, USA

    K. K. Wong Jr.

Authors
  1. S. Chatterjee
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. K. K. Wong Jr.
    View author publications

    You can also search for this author in PubMed Google Scholar

Editor information

Editors and Affiliations

  1. Hearst Microbiology Research Center, Department of Microbiology, Cornell University Medical College, 1300 York Avenue, Box 62, 10021, New York, NY, USA

    Kenneth I. Berns M.D., Ph.D.  & Catherine Giraud Ph.D.  & 

Rights and permissions

Reprints and permissions

Copyright information

© 1996 Springer-Verlag Berlin Heidelberg

About this chapter

Cite this chapter

Chatterjee, S., Wong, K.K. (1996). Adeno-associated Virus Vectors for Gene Therapy of the Hematopoietic System. In: Berns, K.I., Giraud, C. (eds) Adeno-Associated Virus (AAV) Vectors in Gene Therapy. Current Topics in Microbiology and Immunology, vol 218. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-80207-2_5

Download citation

  • DOI: https://doi.org/10.1007/978-3-642-80207-2_5

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-80209-6

  • Online ISBN: 978-3-642-80207-2

  • eBook Packages: Springer Book Archive

Publish with us

Policies and ethics

Search

Navigation