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Human Gene Expression in Murine Hemopoietic Cells In Vivo

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Molecular Biology of Hemopoiesis

Part of the book series: Advances in Experimental Medicine and Biology ((AEMB,volume 34))

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Abstract

Somatic gene transfer offers the possibility of a new approach in the treatment of human genetic disease. Defects affecting the blood and blood forming tissues are candidates for therapies involving transfer of new genetic information into hemopoietic stem cells. One such defect, adenosine deaminase (ADA) deficiency, is being used as a model in which hemopoietic gene transfer techniques can be developed and evaluated. In this model, gene transfer is mediated by a retroviral vector. Retroviral vectors have been used extensively to deliver information to hemopoietic cells1–14. We have previously reported delivery and expression of human ADA (hADA) sequences in murine hemopoietic progenitors in vitro15 and in vivo16, but were not able to demonstrate long term stability of expression. We describe here the construction and testing of four new vectors, representing the first demonstration of efficient transfer and long term in vivo expression of hADA in murine hemopoietic cells.

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Authors and Affiliations

  1. Institute for Molecular Genetics, Baylor College of Medicine, Houston, Texas, 77030, USA

    Frederick A. Fletcher, Kateri A. Moore, Grant R. MacGregor, John W. Belmont & C. Thomas Caskey

  2. Howard Hughes Medical Institute, Baylor College of Medicine, Houston, Texas, 77030, USA

    Frederick A. Fletcher, Kateri A. Moore, Grant R. MacGregor, John W. Belmont & C. Thomas Caskey

Authors
  1. Frederick A. Fletcher
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  2. Kateri A. Moore
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  3. Grant R. MacGregor
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  4. John W. Belmont
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  5. C. Thomas Caskey
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Editor information

Editors and Affiliations

  1. Veterans Administration Medical Center, University of Mississippi, Jackson, Mississippi, USA

    Mehdi Tavassoli

  2. Veterans Administration Medical Center, University of Nevada, Reno, Nevada, USA

    Esmail D. Zanjani

  3. New York Medical College, Valhalla, New York, USA

    Joao L. Ascensao  & Nader G. Abraham  & 

  4. Division of Blood Diseases and Resources, National Heart, Lung, and Blood Institute, Bethesda, Maryland, USA

    Alan S. Levine

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© 1988 Plenum Press, New York

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Fletcher, F.A., Moore, K.A., MacGregor, G.R., Belmont, J.W., Caskey, C.T. (1988). Human Gene Expression in Murine Hemopoietic Cells In Vivo. In: Tavassoli, M., Zanjani, E.D., Ascensao, J.L., Abraham, N.G., Levine, A.S. (eds) Molecular Biology of Hemopoiesis. Advances in Experimental Medicine and Biology, vol 34. Springer, Boston, MA. https://doi.org/10.1007/978-1-4684-5571-7_15

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  • DOI: https://doi.org/10.1007/978-1-4684-5571-7_15

  • Publisher Name: Springer, Boston, MA

  • Print ISBN: 978-1-4684-5573-1

  • Online ISBN: 978-1-4684-5571-7

  • eBook Packages: Springer Book Archive

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