Viral Vector-Mediated Gene Therapy for Hemophilia B
- 1 June 1997
- journal article
- review article
- Published by Georg Thieme Verlag KG in Thrombosis and Haemostasis
- Vol. 78 (01) , 024-030
- https://doi.org/10.1055/s-0038-1657495
Abstract
Over the past five years, significant advances have been made in the development of novel viral vector systems for the treatment of hemophilia B by somatic gene therapy. At present, both a sustained but partial or a complete but transient correction of the hemophilia B phenotype have been observed in a clinically relevant animal model. Present efforts are being directed toward the development of safe, effective and persistent methods of virally-mediated gene transfer to achieve the complete restoration of normal hemostasis in individuals with hemophilia B.Keywords
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