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Gene Therapy with Cord Blood Hematopoietic Stem Cells for Adenosine Deaminase Deficiency: An Update

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Transplantation in Hematology and Oncology

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Abstract

Gene therapy for ADA deficiency has been a model disease in which to evaluate hematopoietic stem cells for clinical gene therapy [1]. ADA deficiency has been a candidate disease because

  1. 1.

    ADA is a single polypeptide protein,

  2. 2.

    a clinically normal phenotype is associated with a wide range of enzymatic function (5–500% of normal), and

  3. 3.

    ADA is widely expressed so that tissue specific expression is not required.

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Author information

Authors and Affiliations

  1. Division of Research Immunology/Bone Marrow Transplantation Childrens Hospital, Molecular Microbiology and Immunology University of Southern California School Medicine, Los Angeles, USA

    D. B. Kohn (Professor of Pediatrics)

  2. Division of Research Immunology/Bone Marrow Transplantation Childrens Hospital, Pediatrics, Molecular Microbiology and Immunology University of Southern California School of Medicine, Los Angeles, USA

    K. Weinberg (Associate Professor)

  3. Division of Research Immunology/Bone Marrow Transplantation Childrens Hospital, Craniofacial and Molecular Biology University of Southern California School of Medicine, Los Angeles, USA

    J. Nolta (Assistant Professor of Pediatrics)

  4. Division of Research Immunology/Bone Marrow Transplantation Childrens Hospital, University of Southern California School Medicine, Los Angeles, USA

    G. Crooks (Assistant Professor of Pediatrics)

  5. Division of Research Immunology/Bone Marrow Transplantation Childrens Hospital, Molecular Microbiology and Immunology University of Southern California School of Medicine, Los Angeles, USA

    R. Parkman (Professor of Pediatrics)

Authors
  1. D. B. Kohn
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  2. K. Weinberg
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  3. J. Nolta
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  4. G. Crooks
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  5. R. Parkman
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Editor information

Editors and Affiliations

  1. Department of Internal Medicine Hematology/Oncology, University of Muenster, Albert-Schweitzer-Straße 33, 48129, Muenster, Germany

    W. E. Berdel (Prof. Dr. med.), Th. Büchner (Prof. Dr. med.), J. Kienast  & J. Van De Loo (Prof. Dr. med.) (Prof. Dr. med.),  (Prof. Dr. med.),  &  (Prof. Dr. med.)

  2. Department of Pediatrics, University of Münster, Albert-Schweitzer-Straße 33, 48129, Muenster, Germany

    H. Jürgens (Prof. Dr. med.), J. Ritter (Prof. Dr. med.) & J. Vormoor (Dr. med.) (Prof. Dr. med.),  (Prof. Dr. med.) &  (Dr. med.)

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© 2000 Springer-Verlag Berlin Heidelberg

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Kohn, D.B., Weinberg, K., Nolta, J., Crooks, G., Parkman, R. (2000). Gene Therapy with Cord Blood Hematopoietic Stem Cells for Adenosine Deaminase Deficiency: An Update. In: Berdel, W.E., et al. Transplantation in Hematology and Oncology. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-59592-9_35

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  • DOI: https://doi.org/10.1007/978-3-642-59592-9_35

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-64041-4

  • Online ISBN: 978-3-642-59592-9

  • eBook Packages: Springer Book Archive

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