These studies support that targeting FVIII to platelets is unique in the hemophilia A gene therapy, because FVIII stored in platelets can be better sequestered compared to plasma FVIII as platelet-FVIII will be released together with its carrier protein VWF when it is needed, i.e., at the site of injury where platelets are activated. The gene discussed is F8; the disease is hemophilia A.