RESEARCH Role of the vector genom e H

(F.IX), resulting in a deficiency in the ability of blood to clot. In addition to increased propensity for bleeding disease on demand. Gene therapy represents an attrac-tive alternative to protein replacement therapy, as it Rogers et al. Journal of Translational Medicine 2014, 12:25 http://www.translational-medicine.com/content/12/1/25packaging around the transgenic construct. The variousUniversity of Florida, Gainesville, Florida, USA Full list of author information is available at the end of the articleafter trauma or injury, spontaneous bleeds can occur in capillaries, particularly in the joints, resulting in tissue damage over time. Bleeds into critical closed spaces can be life-threatening. Currently, hemophilia B is treated by intravenous administration of F.IX concentrate, either plasma-derived or recombinant, in order to would involve a single injection to provide long-term in-trinsic production of F.IX. Among potential gene therapies for hemophilia B, th