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Title : Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use
link : Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use
Major Success for Gene Therapy for Factor IX Deficiency: near elimination of bleeding and factor use
Hemophilia B Gene Therapy with a High-Specific-Activity Factor IX Variant: the researchers infused a single-stranded adeno-associated viral (AAV) vector consisting of a bioengineered capsid, liver-specific promoter and factor IX Padua (factor IX–R338L) transgene in 10 men with hemophilia B who had factor IX coagulant activity of 2% or less of the normal value.They found sustained therapeutic expression of factor IX coagulant activity after gene transfer in the 10 participants with hemophilia who received the same vector dose. Transgene-derived factor IX coagulant activity enabled the termination of baseline prophylaxis and the near elimination of bleeding and factor use.
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