Article
Phenotypic correction of hemophilia A by ectopic expression of activated factor VII in platelets.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 Aug 2008
Ohmori Tsukasa, Ishiwata Akira, Kashiwakura Yuji, Madoiwa Seiji, Mitomo Katsuyuki, Suzuki Hidenori, Hasegawa Mamoru, Mimuro Jun, Sakata Yoichi
Abstract excerpt
Platelets are receiving much attention as novel target cells to secrete a coagulation factor for hemophilia gene therapy. In order to extend the application of platelet-directed gene therapy, we examined whether ectopic expression of activated factor VII (FVIIa) in platelets would result in an efficient bypass therapy to induce sufficient thrombin generation on platelet surfaces in mice with hemophilia A....
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.