Corpus ID: 80523603

New hemophilia B gene therapy appears effective

  title={New hemophilia B gene therapy appears effective},
  author={Marian Freedman and Michael G Burke},
  journal={Contemporary pediatrics},
A recent trial showed that a single intravenous infusion of a vector expressing a high specific-activity factor IX gene safely produces a sustained, effective level of factor IX coagulant activity in patients with hemophilia B. 
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