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  • Title: Therapeutic globin gene delivery using lentiviral vectors.
    Author: Rivella S, Sadelain M.
    Journal: Curr Opin Mol Ther; 2002 Oct; 4(5):505-14. PubMed ID: 12435053.
    Abstract:
    The severe hemoglobinopathies, including beta-thalassemia major and sickle cell anemia, are candidate diseases for a genetic treatment based on the transfer of a regulated globin gene in autologous hematopoietic stem cells. Two years ago, May et al reported that an optimized beta-globin transcription unit containing multiple proximal and distal regulatory elements harbored by a recombinant lentiviral vector could efficiently integrate into murine hematopoietic stem cells and express therapeutic levels of the human beta-globin gene. Here, we review the advantages afforded by lentivirus-mediated globin gene transfer and recent studies based on this strategy.
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