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Novel therapeutic approach for hemophilia using gene delivery of an engineered secreted activated Factor VII

Hemophilia is a bleeding disorder caused by mutations in the genes encoding coagulation Factor VIII (FVIII) or FIX. Current treatment is through intravenous infusion of the missing protein. The major complication of treatment is the development of neutralizing Ab’s to the clotting factor. Infusion o...

Ausführliche Beschreibung

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Bibliographische Detailangaben
Hauptverfasser: Margaritis, Paris, Arruda, Valder R., Aljamali, Majed, Camire, Rodney M., Schlachterman, Alexander, High, Katherine A.
Format: Artigo
Sprache:Inglês
Veröffentlicht: American Society for Clinical Investigation 2004
Schlagworte:
Online Zugang:https://ncbi.nlm.nih.gov/pmc/articles/PMC379321/
https://ncbi.nlm.nih.gov/pubmed/15057309
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1172/JCI200420106
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