A promising genetic approach to the treatment of β-thalassemia Journal Article


Authors: May, C.; Sadelain, M.
Article Title: A promising genetic approach to the treatment of β-thalassemia
Abstract: The stable introduction of a functional gene into autologous stem cells is a potentially powerful approach to treat a number of inherited or acquired diseases. One challenge facing this approach is to express adequate levels of the therapeutic transgene in a regulated and sustained fashion, eventually restricting expression to a single lineage developing from the transduced stem cells. Until now, low-level expression, position effects, and transcriptional silencing have hampered the effectiveness of retroviral-mediated gene transfer. In an effort to overcome these obstacles, we have systematically investigated vectors encoding the human β-globin gene linked to selected combinations of proximal and distal genetic regulatory elements. Our results demonstrate that with thoughtful vector design one can successfully express long-term, therapeutic levels of virally encoded human β-globin in the erythroid progeny of hematopoietic stem cells. © 2001, Elsevier Science Inc.
Keywords: promoter region; nonhuman; animals; mice; gene expression; bone marrow; hematopoietic stem cell transplantation; gene transfer; chimera; drug mechanism; gene therapy; beta thalassemia; transgene; beta-thalassemia; short survey; hematopoietic stem cell; gene silencing; lentivirinae; globins; virus vector; gene transfer techniques; enhancer region; beta globin; virus gene; regulator gene; murine leukemia virus; humans; human; priority journal; erythroleukemia cell
Journal Title: Trends in Cardiovascular Medicine
Volume: 11
Issue: 7
ISSN: 1050-1738
Publisher: Elsevier Science, Inc.  
Date Published: 2001-10-01
Start Page: 276
End Page: 280
Language: English
DOI: 10.1016/s1050-1738(01)00125-6
PUBMED: 11709281
PROVIDER: scopus
DOI/URL:
Notes: Export Date: 21 May 2015 -- Source: Scopus
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MSK Authors
  1. Chad May
    21 May
  2. Michel W J Sadelain
    492 Sadelain