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Hemophilia as a Model Disease for Gene Therapy of Genetic Disorders

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Abstract

Gene therapy offers the potential for cure of hemophilia, a sex-linked genetic bleeding disorder caused by deficiency of either coagulation factor VIII or coagulation factor IX. The features of hemophilia that make it a leading candidate for gene therapy include the fact that the factor VIII and factor IX genes have been identified and cloned, therapeutic benefit would result from achieving expression at plasma levels as low as 1% of normal, and a wide variety of gene transfer vectors and cell target types could be useful. The challenge is to obtain long-term gene expression at levels sufficient to prevent spontaneous bleeding, while avoiding unwanted toxicity or immune responses to the expressed clotting factor.

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Authors and Affiliations

  1. Center for Biologics Evaluation and Research, Food and Drug Administration, Rockville, MD

    Jay Lozier

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  1. Jay Lozier
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  1. Department of Medicine, Division of Cardiology Carolina Cardiovascular Biology Center, University of North Carolina School of Medicine, Chapel Hill, NC

    Marschall S. Runge MD, PhD  & Cam Patterson MD  & 

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Lozier, J. (2006). Hemophilia as a Model Disease for Gene Therapy of Genetic Disorders. In: Runge, M.S., Patterson, C. (eds) Principles of Molecular Medicine. Humana Press. https://doi.org/10.1007/978-1-59259-963-9_6

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  • DOI: https://doi.org/10.1007/978-1-59259-963-9_6

  • Publisher Name: Humana Press

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