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. We infused a single dose of a ...
Nine-year follow-up of gene therapy in haemophilia B confirms long-term safety and reduced bleeding, though full capsid ...
Hemophilia B gene therapy reduced bleeding and supported lasting clotting levels, even in some patients with antibodies, new ...