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Inherited Hepatic Enzyme Defects as Candidates for Liver-Directed Gene Therapy

  • Chapter
Hepadnaviruses

Part of the book series: Current Topics in Microbiology and Immunology ((CT MICROBIOLOGY,volume 168))

Abstract

There are a small number of inherited hepatic defects that are so devastating that virtually 100% of the affected infants either die or suffer severe developmental disability. For these defects, serious consideration must be given to any new treatment that offers the possibility of altering the extremely poor prognosis. In the desparate clinical setting of these single gene defects, two forms of therapy have recently been considered—hepatic transplantation and gene therapy. Here, I discuss two severe single gene disorders that might be candidates for such novel therapies, outline their natural histories, elaborate on the diagnostic tools available, and describe the result of hepatic transplantation in one case. I conclude that there remains a place for the development of cell or gene therapy for these severe disorders, speculate on potential strategies, and suggest that, if a safe and effective strategy could be developed, other less severe conditions of the liver could ultimately be considered for such therapy.

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Authors and Affiliations

  1. Howard Hughes Medical Institute and Department of Human Genetics, Yale University School of Medicine, 06510, New Haven, CT, USA

    A. L. Horwich

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  1. A. L. Horwich
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Editor information

Editors and Affiliations

  1. Fox Chase Cancer Center, 7701 Burholme Ave., Philadelphia, PA, 19111, USA

    William S. Mason  & Christoph Seeger  & 

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© 1991 Springer-Verlag Berlin·Heidelberg

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Horwich, A.L. (1991). Inherited Hepatic Enzyme Defects as Candidates for Liver-Directed Gene Therapy. In: Mason, W.S., Seeger, C. (eds) Hepadnaviruses. Current Topics in Microbiology and Immunology, vol 168. Springer, Berlin, Heidelberg. https://doi.org/10.1007/978-3-642-76015-0_9

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  • DOI: https://doi.org/10.1007/978-3-642-76015-0_9

  • Publisher Name: Springer, Berlin, Heidelberg

  • Print ISBN: 978-3-642-76017-4

  • Online ISBN: 978-3-642-76015-0

  • eBook Packages: Springer Book Archive

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