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Treatment of Adenosine Deaminase Deficiency

Authors
  • Hoogerbrugge, Peter M.1
  • Bleesing, Jack J.H.1
  • Vossen, Jaak M.1
  • Valerio, Dinko2, 3
  • 1 University Hospital Leiden, Department of Paediatrics, Leiden, 2300 RC, The Netherlands , Leiden (Netherlands)
  • 2 University of Leiden, Department of Molecular Cell Biology, Leiden, The Netherlands , Leiden (Netherlands)
  • 3 IntroGene BV, Leiden, The Netherlands , Leiden (Netherlands)
Type
Published Article
Journal
BioDrugs
Publisher
Springer International Publishing
Publication Date
Feb 01, 1998
Volume
9
Issue
2
Pages
87–93
Identifiers
DOI: 10.2165/00063030-199809020-00001
Source
Springer Nature
Keywords
License
Yellow

Abstract

Severe combined immune deficiency disease due to a deficiency of the enzyme adenosine deaminase is a rare disease. However, it has been used as a prototype disease for the development of a variety of treatment modalities that are currently applied in more frequent diseases. For example, allogeneic bone marrow transplantation and stem cell gene therapy have been used for adenosine deaminase deficiency before being applied in other more frequent diseases. In the present paper, the development of bone marrow transplantation and stem cell gene therapy, as well as treatment with purified enzyme, for adenosine deaminase deficiency are discussed.

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