Elsevier

Pathology

Volume 30, Issue 4, 1998, Pages 335-347
Pathology

Gene transfer: A review of methods and applications

https://doi.org/10.1080/00313029800169606Get rights and content

Summary

Gene transfer is a potentially powerful tool for the treatment of a wide variety of diseases. The transfer of these genes is achieved by utilizing a variety of vectors, including retroviral, adenoviral, adeno-associated virus (AAV) and a number of non-viral mechanisms. Numerous studies have successfully demonstrated transduction of genes into target cells with a variety of vectors, and have provided 'proof-in-principle' that gene transfer can result in prolonged in vivo expression of transduced genes, albeit at low quantities. Furthermore, gene marking studies in acute myeloblastic leukemia (AML), chronic myeloid leukemia (CML) and neuroblastoma have elegantly demonstrated that gene-marked tumor cells contribute to relapse following autologous transplantation. However none of the studies examining the therapeutic benefit of gene therapy has definitively demonstrated a clinically meaningful benefit. Nonetheless, the results of studies involving gene transfer for severe combined immunodeficiency (SCID), chronic granulomatous disease (CGD), melanoma and lung cancer highlight the potential benefit of this strategy. This review will discuss mechanisms of achieving gene transfer into target cells. It will examine some of the pre-clinical and clinical results to date and will discuss some of the potential uses of gene transfer for therapeutic purposes.

References (102)

  • L. Chen et al.

    Costimulation of T cells for tumor immunity

    Immunol Today

    (1993)
  • E.C. Guinan et al.

    Pivotal role of the B7:CD28 pathway in transplantation tolerance and tumor immunity

    Blood

    (1994)
  • L. Chen et al.

    Costimulation of antitumor immunity by the B7 counter-receptor for the T Iymphocyte molecules CD28 and CTLA-4

    Cell

    (1992)
  • U.A. Matulonis et al.

    Role of B7-1 in mediating an immune response to myeloid leukemia cells

    Blood

    (1995)
  • M.S. Dilber et al.

    Suicide gene therapy for plasma cell tumours

    Blood

    (1996)
  • E. Sporeno et al.

    Human Interleukin-6 receptor super-antagonists with high potency and wide spectrum on multiple myeloma cells

    Blood

    (1996)
  • S.H. Orkin et al.

    NIH Proceedings-Dec 95 Report and recommendations of the panel to assess the NIH investment in research on gene therapy

    (1995)
  • N.A. Wivel et al.

    Germ-line gene modification and disease prevention: some medical and ethical perspectives

    Science

    (1993)
  • P.H. Raven et al.
  • O.T. Avery et al.

    Studies on the chemical nature of the substance inducing transformation of pneumococcal types

    J Exp Med

    (1944)
  • J.D. Watson et al.
  • H.M. Temin

    The Retroviradae

    (1992)
  • J.M. Coffin

    Virology

    (1990)
  • M.P. Kavanangh et al.

    Cell-surface receptors for gibbon ape leukemia virus and amphotropic murine retrovirus are inducible sodium-dependent phosphate symporters

    Proc Natl Acad Sci USA

    (1994)
  • M. Van Zeul et al.

    A human amphotrophic retrovirus receptor is a second member of the gibbon ape leukemia virus receptor family

    Proc Natl Acad Sci USA

    (1994)
  • D.G. Miller et al.

    Gene transfer by retrovirus vectors occurs only in cells that are actively replicating at the time of infection

    Mol Cell Biol

    (1990)
  • C.D. Porter et al.

    Comparison of efficiency of infection of human gene therapy target cells via four different retroviral receptors

    Hum Gene Ther

    (1996)
  • C. Gelinas et al.

    Nondefective spleen necrosis virus-derived vectors define the upper size limit for packaging reticuloendotheliosis viruses

    Proc Natl Acad Sci USA

    (1986)
  • R.E. Donahue et al.

    Helper virus induced T cell lymphoma in nonhuman primates after retroviral mediated gene transfer

    J Exp Med

    (1992)
  • E.J. Kremer et al.

    Adenovirus and adeno-associated virus mediated gene transfer

    Br Med Bull

    (1995)
  • H.S. Ginsberg

    The Adenoviruses

    (1984)
  • F.L. Graham et al.

    Characteristics of a human cell line transformed by DNA from human adenovirus type 5.

    J Gen Virol

    (1977)
  • A.J. Bert et al.

    Packaging capacity and stability of human adenovirus type 5 vectors

    J Virol

    (1993)
  • A.J. Bett et al.

    An efficient and flexible system for construction of adenovirus vectors with insertions and deletions in early regions 1 and 3

    Proc Natl Acad Sci USA

    (1994)
  • R.J. Parks et al.

    A helper-dependent adenovirus vector system: removal of the helper virus by Cre-mediated excision of the viral packaging signal

    Proc ?Cad Acad Sci USA

    (1996)
  • S.I. Michael et al.

    Strategies to accomplish targeted gene delivery implying tropism-modified recombinant adenovirat vectors

    Cancer Gene Ther

    (1995)
  • T.J. Wickham et al.

    Targeted adenovirus-mediated gene delivery to T cells via CD3

    J Virol

    (1997)
  • T.J. Wickham et al.

    Targeting of adenovirus penton base to new receptors through replacement of its RGD motif with other receptor-specific peptide motifs

    Gene Ther

    (1995)
  • R.L. Neve et al.

    A defective herpes simplex virus vector system for gene delivery into the brain: comparison with alternative gene delivery systems and usefulness for gene therapy

    Clin Neurosci

    (1996)
  • S.L. Turner et al.

    The roles of herpes simplex virus in neuroscience

    J Neurovirol

    (1997)
  • D.S. Latchman

    Herpes simplex virus vector for gene therapy

    Mol Biotechnol

    (1994)
  • F. Toneguzzo et al.

    Stable expression of selectable genes introduced into human hematopoietic stem cells by electric field mediated DNA transfer

    Proc Natl Acad Sci USA

    (1986)
  • D. Wu et al.

    Engraftment of donor-derived bone marrow stromal cells

    Exp Hematol

    (1991)
  • M. Fechheimer et al.

    Transfection of mammalian cells with plasmid DNA by scrape loading and sonicating loading

    Proc Natl Acad Sci USA

    (1987)
  • P.L. MacNeil et al.

    Glass beads load macromolecules into living cells

    J Cell Sci

    (1987)
  • K.E. Mathews et al.

    Bead transfection: rapid and efficient gene transfer into marrow stromal and other adherent mammalian cells

    Exp Hematol

    (1993)
  • K.E. Mathews et al.

    Gene therapy with physical methods of gene transfer

    Transfusion Sci

    (1996)
  • M.Z. Ratajczak et al.

    The biology of hematopoietic stem cells

    Semin Oncol

    (1995)
  • B. Salmons et al.

    Targeting retroviral vectors for gene therapy

    Hum Gene Ther

    (1993)
  • R.F. Carter et al.

    Autologous transplantation of canine long-term marrow culture cells genetically marked by retroviral vectors

    Blood

    (1992)
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