Article
Complete and sustained phenotypic correction of hemophilia B in mice following hepatic gene transfer of a high-expressing human factor IX plasmid.
Journal of thrombosis and haemostasis : JTH - 1 Jan 2003
Ye X, Loeb K R, Stafford D W, Thompson A R, Miao C H
Abstract excerpt
Therapeutic correction of hemophilia B was achieved by rapid infusion of a large-volume solution containing a high-expressing human factor IX (hFIX) plasmid into the tail vein of hemophilia B mice. hFIX circulated at therapeutic levels (1-5 micro g mL-1) in all animals for more than 1 year as determined by both species-specific antigen assay and an activated partial thromboplastin time (APTT)-based clotting...
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