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Transduction of CD34+ enriched cord-blood and Gaucher bone-marrow cells by a retroviral vector carrying the glucocerebrosidase gene

Academic Article
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Overview

authors

  • Bahnson, A. B.
  • Nimgaonkar, M.
  • Fei, Y.
  • Boggs, S. S.
  • Robbins, Paul D.
  • Ohashi, T.
  • Dunigan, J.
  • Li, J.
  • Ball, E. D.
  • Barranger, J. A.

publication date

  • May 1994

journal

  • Gene Therapy  Journal

abstract

  • One promising strategy for gene therapy of Gaucher disease involves ex vivo retroviral transduction of autologous hematopoietic stem cells. Studies in small animals have demonstrated that this approach provides a life-long supply of the glucocerebrosidase (GC) enzyme. Human application has developed to the stage of a clinical trial. In this study, we describe development of a high titer amphotropic producer line for the vector, MFG-GC, and explore transduction of CD34+ cells from various human sources. Higher than three times the normal levels of glucocerebrosidase activity in non-transduced cells were achieved following transduction of CD34+ cells obtained from bone marrow or cord blood from normal donors. The improvement in enzyme activity in Gaucher marrow was about 40-fold above deficient levels. We examined the timing and stepwise effect of multiple rounds of infection and evaluated post-infection expansion of cells in two different cytokine mixtures. Transduction efficiency was determined using immunocytochemistry and Southern blot hybridization.

subject areas

  • Antigens, CD34
  • Bone Marrow
  • Fetal Blood
  • Gaucher Disease
  • Genetic Therapy
  • Genetic Vectors
  • Glucosylceramidase
  • Humans
  • In Vitro Techniques
  • Infant, Newborn
  • Retroviridae
  • Transduction, Genetic
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Identity

International Standard Serial Number (ISSN)

  • 0969-7128

PubMed ID

  • 7584079
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Additional Document Info

start page

  • 176

end page

  • 184

volume

  • 1

issue

  • 3

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