Journal of Inherited Metabolic Disease

, Volume 16, Issue 5, pp 881–885 | Cite as

Correction of enzyme deficiency in metachromatic leukodystrophy fibroblasts by retroviral-mediated transfer of the human arylsulphtase A gene

  • T. Ohashi
  • Y. Eto
  • R. Learish
  • J. A. Barranger
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Keywords

Public Health Internal Medicine Leukodystrophy Enzyme Deficiency Metachromatic Leukodystrophy 

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References

  1. Baum H, Dodgson KS, Spencer B (1959) The assay of arylsulfatase A and B in human urine.Clin Chim Acta 4: 453–455.Google Scholar
  2. Chen C, Okayama H (1987) High efficiency transformation of mammalian cells by plasmid DNA.Mol Cell Biol 7: 2745–2752.Google Scholar
  3. Kolodny EH (1989) Metachromatic leukodystrophy and multiple sulfatase deficiency. In Scriver CR, Beaudet AL, Sly WS, Valle D, eds.The Metabolic Basis of Inherited Disease, 6th edn. New York: McGraw-Hill, 1721–1750.Google Scholar
  4. Krivit W, Shapiro E, Kennedy W et al (1990) Treatment of late infantile metachromatic leukodystrophy by bone marrow transplantation.N Engl J Med 322: 28–32.Google Scholar
  5. Ohashi T, Boggs S, Robbins P et al (1992) Efficient and sustained high expression of the human glucocerebrosidase gene in mice and their functional macrophages following transplantation of bone marrow transduced by a retroviral vector.Proc Natl Acad Sci USA 89: 11332–11336.Google Scholar
  6. Rommerskirch W, Fluharty AL, Perters C, von Figura K, Gieselmann V (1991) Restoration of arylsulphatase A activity in human-metachromatic leukodystrophy fibroblasts via retroviral-vector-mediated gene transfer.Biochem J 280: 459–461.Google Scholar

Copyright information

© SSIEM and Kluwer Academic Publishers 1993

Authors and Affiliations

  • T. Ohashi
    • 1
  • Y. Eto
    • 1
  • R. Learish
    • 2
  • J. A. Barranger
    • 2
  1. 1.Department of PediatricsJikei University School of MedicineTokyoJapan
  2. 2.Department of Human Genetics, E 1650 BSTUniversity of PittsburghPittsburghUSA

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