The leak stops here: platelets as delivery vehicles for coagulation factors
J. Clin. Invest. Katherine A. High, et al. 116:1840 doi:10.1172/JCI29193 [Go to this article.]

Figure 1
Strategies for achieving long-term protein expression in genetic disease. (A) Gene transfer strategies leading to long-term protein expression in canine hemophilia. At left, intravenous infusion of a retroviral vector expressing canine factor IX (FIX) into neonatal dogs results in transduction of hepatocytes with transmission of the integrated DNA into all daughter cells and continuous expression of factor IX in the growing animal (1). In the approach shown to the right, with portal vein infusion of adeno-associated virus–FVIII (AAV-FVIII) or –factor IX into an adult animal, the donated DNA is stabilized in an episomal form, with long-term expression of the clotting factor (2–4). (B) After gene transfer into hematopoietic stem cells (CD34⁺ cells), megakaryocytes carrying the donated gene give rise to platelets that contain FVIII packaged in the α-granules. Activation of platelets at the site of an injury leads to release of granule contents, including FVIII.