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 …

Gene therapy for hemophilia B aims to ameliorate bleeding risk and provide endogenous
factor IX (FIX) activity/synthesis through a single treatment, eliminating the requirement for …

Background Moderate-to-severe hemophilia B is treated with lifelong, continuous
coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims …

Background In patients with severe hemophilia B, gene therapy that is mediated by a novel
self-complementary adeno-associated virus serotype 8 (AAV8) vector has been shown to …

Background Hemophilia B, an X-linked disorder, is ideally suited for gene therapy. We
investigated the use of a new gene therapy in patients with the disorder. Methods We …

Gene therapy offers the potential for a cure for patients with hemophilia by establishing
continuous endogenous expression of factor VIII or factor IX (FIX) following transfer of a …

Gene therapy has the potential to maintain therapeutic blood clotting factor IX (FIX) levels in
patients with hemophilia B by delivering a functional human F9 gene into liver cells. This …

Hemophilia B is an X-linked coagulopathy caused by absence of functional coagulation
factor IX (FIX). Using adeno-associated virus (AAV)–mediated, liver-directed gene therapy …

Background Prophylactic factor replacement in patients with hemophilia B improves
outcomes but requires frequent injections. A recombinant factor IX Fc fusion protein (rFIXFc) …

The safety of several gene therapy approaches for treatment of the severe, X-linked
bleeding disorder hemophilia is currently being evaluated in early phase clinical trials. One …