The Complete Dependence of Factor IX Padua (R338L) Hyperactivity on Factor VIIIa Cofactor Activity Supports Its Safety As a Transgene for Hemophilia B Gene Therapy
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DOI: 10.1182/blood-2018-99-119270
Abstract: Factor IX (FIX) Padua (R338L) has been described as a game changer for hemophilia B (HB) gene therapy. The ~8-fold increased specific activity compared to wild-type FIX (FIX WT) in aPTT-based clotting assay has recently… read more here.
Keywords: activity; gene therapy; fix padua; padua ... See more keywords