Therapeutic globin gene delivery using lentiviral vectors Journal Article

  


Authors: Rivella, S.; Sadelain, M.
Article Title: Therapeutic globin gene delivery using lentiviral vectors
Abstract: The severe hemoglobinopathies, including β-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 β-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 β-globin gene. Here, we review the advantages afforded by lentivirus-mediated globin gene transfer and recent studies based on this strategy.
Keywords: promoter region; hydroxyurea; review; cytotoxic agent; drug efficacy; drug safety; nonhuman; cd34 antigen; gene expression; green fluorescent protein; immunoglobulin; genetic transcription; gene transfer; viral gene delivery system; genetic transduction; genetic vectors; rna; disease severity; genetic transfection; messenger rna; virus rna; transactivator protein; gene therapy; lentivirus; lentivirus vector; retrovirus vector; beta thalassemia; erythroid cell; transgene; murinae; genetic stability; gene control; human immunodeficiency virus; bone marrow cell; hematopoietic stem cell; virus recombinant; virus replication; plasmid vector; human immunodeficiency virus 1; transcription factor gata 1; virus protein; globins; sickle cell disease; tissue specificity; beta globin; thrombospondin; β-thalassemia; hemoglobinopathy; sickle cell anemia; gag protein; globin gene regulation; lentiviral vector; regulator gene; deoxyribonuclease i; ankyrin; spectrin; unidentified retrovirus; humans; human; transcription factor nf e2; oncovirinae; 5 aminolevulinate synthase; alpha globulin; rev protein; drug packaging; hepatitis virus
Journal Title: Current Opinion in Molecular Therapeutics
Volume: 4
Issue: 5
ISSN: 1464-8431
Publisher: Thomson Reuters  
Date Published: 2002-01-01
Start Page: 505
End Page: 514
Language: English
PUBMED: 12435053
PROVIDER: scopus
DOI/URL:

http://www.scopus.com/inward/record.url?eid=2-s2.0-0036819745&partnerID=40&md5=0f1cb6035d4cb6f2086d074103d6a7d2
Notes: Export Date: 14 November 2014 -- Source: Scopus
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MSK Authors
  1. Stefano Rivella
    16 Rivella
  2. Michel W J Sadelain
    583 Sadelain
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