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Gene therapy Progress and Prospects: Gene therapy for the hemophilias

Gene Therapy volume 10, pages 999–1003 (2003)Cite this article

Abstract

Recent gene transfer trials for hemophilia A and B, bleeding disorders lacking either functional factor VIII or IX, respectively, have produced tantalizing results, suggesting that the potential to correct these bleeding disorders at a molecular level may be at hand. Genetic correction of the hemophilias represents a model system to develop a basic understanding of how gene therapy will be achieved. The goals for hemophilia gene transfer require the long-term therapeutic production of the coagulant protein without stimulating an immune response to the transgene product or the vector. Based on a scientific understanding of the molecular and cellular defects, leading to the bleeding phenotype, impressive strides have been made in the last 2 years.

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  1. Mt Sinai School of Medicine, One Gustave Levy Pl, New York City, NY, USA

    Christopher E Walsh MD, Ph,D

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  1. Christopher E Walsh MD, Ph,D
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Walsh, C. Gene therapy Progress and Prospects: Gene therapy for the hemophilias. Gene Ther 10, 999–1003 (2003). https://doi.org/10.1038/sj.gt.3302024

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