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Human Hematopoietic Stem Cells, Progenitors, and Peripheral Blood Lymphocytes as Targets for the Correction of Immune System Disorders via Gene Therapy

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Book cover Molecular Biology of B-Cell and T-Cell Development

Part of the book series: Contemporary Immunology ((CONTIM))

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Abstract

As genetic defects responsible for congenital disorders are delineated at the DNA level, it is possible to design strategies to express the corrected gene in autologousderived cells (1–3). A large number of gene therapy clinical trials encompassing a broad spectrum of human diseases are in progress (4,5). Therapies to prevent and treat human immunodeficiency virus (HIV) (6), adenosine deaminase deficiency associated with severe combined immunodeficiency disease (ADA− SCID) (7–11); lysosomal storage diseases (12,13), autoimmune diseases (3,14), as well as a variety of cancers (15), are ongoing. In this chapter, the authors will review the current status on gene transfer technology and discuss current findings on treatment of ADA− SCID by gene therapy. In addition, five primary immunodeficiency diseases that affect the B- and T-cell lineages at various stages of differentiation will be highlighted, and the potential treatment of these diseases by gene therapy will be discussed.

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Authors
  1. Karen E. Pollok
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  2. David A. Williams
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Editors and Affiliations

  1. University of Pennsylvania, Philadelphia, PA, USA

    John G. Monroe

  2. California Institute of Technology, Pasadena, CA, USA

    Ellen V. Rothenberg

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Pollok, K.E., Williams, D.A. (1998). Human Hematopoietic Stem Cells, Progenitors, and Peripheral Blood Lymphocytes as Targets for the Correction of Immune System Disorders via Gene Therapy. In: Monroe, J.G., Rothenberg, E.V. (eds) Molecular Biology of B-Cell and T-Cell Development. Contemporary Immunology. Humana Press, Totowa, NJ. https://doi.org/10.1007/978-1-4757-2778-4_27

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  • DOI: https://doi.org/10.1007/978-1-4757-2778-4_27

  • Publisher Name: Humana Press, Totowa, NJ

  • Print ISBN: 978-1-61737-065-6

  • Online ISBN: 978-1-4757-2778-4

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