Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

@article{Jiang2006EvidenceOM,
  title={Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.},
  author={Haiyan Jiang and G F Pierce and Margareth Castro Ozelo and E V De Paula and Joseph A Vargas and Peter J. Smith and J{\"u}rg M Sommer and Alvin Y. Luk and Catherine S. Manno and K. A. High and Valder R Arruda},
  journal={Molecular therapy : the journal of the American Society of Gene Therapy},
  year={2006},
  volume={14 3},
  pages={452-5}
}
In a phase I study, administration of an AAV2-FIX vector into the skeletal muscle of eight hemophilia B subjects proved safe and achieved local gene transfer and FIX expression for at least 10 months after vector injection, the last time point assessed by muscle biopsy. In hemophilia B dogs we have demonstrated FIX in both muscle biopsies and circulation >4… CONTINUE READING