Corpus ID: 80523603

New hemophilia B gene therapy appears effective

@article{Freedman2018NewHB,
  title={New hemophilia B gene therapy appears effective},
  author={Marian Freedman and Michael G Burke},
  journal={Contemporary pediatrics},
  year={2018}
}
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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It seems that, given the high potential of gene Therapy and the fact that this experimental technique is a candidate for the treatment of pulmonary diseases, further study of genes, vectors and gene transfer systems can draw a very positive perspective of gene therapy in near future. Expand