Viral vector-mediated gene therapy for hemophilia B

Autor: R C, Eisensmith, S L, Woo
Rok vydání: 1997
Předmět:
Zdroj: Thrombosis and haemostasis. 78(1)
ISSN: 0340-6245
Popis: 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.
Databáze: OpenAIRE