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Article

Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant

6 Dec 2017

Abstract excerpt

BACKGROUND: The prevention of bleeding with adequately sustained levels of clotting factor, after a single therapeutic intervention and without the need for further medical intervention, represents an important goal in the treatment of hemophilia. METHODS: vector genomes per kilogram of body weight in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value. Laboratory...

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Share to Blood Coagulation and Thrombosis Mechanisms
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PMID: 29211678
DOI: 10.1056/nejmoa1708538

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