Globin gene transfer for the treatment of severe hemoglobinopathies: A paradigm for stem cell-based gene therapy Journal Article


Author: Sadelain, M.
Article Title: Globin gene transfer for the treatment of severe hemoglobinopathies: A paradigm for stem cell-based gene therapy
Abstract: The prospect of treating blood disorders with genetically modified stem cells is highly promising. This therapeutic approach, however, raises a number of fundamental biological questions, spanning several research fields. Further investigation is required to better understand how to isolate and efficiently transduce hematopoietic stem cells (HSCs), while preserving optimal homing and self-renewing properties; how to design safe vectors permitting controlled expression of the transgene products; and how to promote host repopulation by engrafted HSCs. This article addresses basic issues in stem cell-based gene therapy from the perspective of regulating transgene expression, taking globin gene transfer for the treatment of severe hemoglobinopathies as a paradigm. Copyright © 2002 John Wiley & Sons, Ltd.
Keywords: genetics; hydroxyurea; review; nonhuman; side effect; methodology; mouse; animal; cytology; animals; mice; gene expression; biological model; gene transfer; transgenic mouse; viral gene delivery system; gene vector; genetic transduction; genetic vectors; mice, transgenic; transduction, genetic; stem cell; disease severity; gene therapy; lentivirus; globin; hemoglobin beta chain; lentivirus vector; retrovirus vector; beta thalassemia; transgene; beta-thalassemia; stem cells; allogeneic hematopoietic stem cell transplantation; models, genetic; hematopoietic stem cell; virus genome; genetic code; oxygen transport; transgenes; lentivirinae; globins; sickle cell disease; nonviral gene therapy; gene transfer techniques; beta globin; β-thalassemia; gene regulation; hemoglobinopathy; sickle cell anemia; hemoglobinopathies; gag protein; lentiviral vector; viral gene therapy; hemoglobin f; cell based gene therapy; deoxyribonuclease i; virus envelope protein; locus control region; hemoglobin a; human; priority journal; hemoglobin alpha chain; pol protein; erythroleukemia cell
Journal Title: Journal of Gene Medicine
Volume: 4
Issue: 2
ISSN: 1099-498X
Publisher: John Wiley & Sons  
Date Published: 2002-03-01
Start Page: 113
End Page: 121
Language: English
DOI: 10.1002/jgm.266
PUBMED: 11933212
PROVIDER: scopus
DOI/URL:
Notes: Export Date: 14 November 2014 -- Source: Scopus
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MSK Authors
  1. Michel W J Sadelain
    456 Sadelain