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Gene therapy of hepatic diseases: prospects for the new millennium

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Footnotes

  • *Note added in proof: A troubling development has occurred in a clinical trial of adenoviral gene therapy for ornithine transcarbamylase deficiency. The unexplained death of one of the trial patients has resulted in the indefinite suspension of the trial.

  • Abbreviations used in this review:
    AAV
    adeno-associated virus
    AsGPr
    asialoglycoprotein receptor
    BUGT
    bilirubin-UDP- glucuronyltransferase
    GSD
    glycogen storage disease
    HCV
    hepatitis C virus
    HSV-tk
    herpes simplex virus thymidine kinase
    hGAA
    human acid α-glucosidase
    IL
    interleukin
    LDL
    low density lipoprotein
    OTC
    ornithine transcarbamylase
    SV-40
    Simian virus 40