期刊
ADVANCED DRUG DELIVERY REVIEWS
卷 62, 期 12, 页码 1196-1203出版社
ELSEVIER SCIENCE BV
DOI: 10.1016/j.addr.2010.06.007
关键词
Gene therapy; Platelet-specific promoter; Platelet; Hemostasis
资金
- American Heart Association [0730183N]
- National Hemophilia Foundation
- Hemophilia Association of New York
- National Institutes of Health [HL-102035, HL-44612]
- Goerlich Foundation
- Bayer Hemophilia Award
Platelets are small, anucleate, discoid shaped blood cells that play a fundamental role in hemostasis. Platelets contain a large number of biologically active molecules within cytoplasmic granules that are critical to normal platelet function. Because platelets circulate in blood through out the body, release biological molecules and mediators on demand and participate in hemostasis as well as many other pathophysiologic processes, targeting expression of proteins of interest to platelets and utilizing platelets as delivery systems for disease treatment would be a logical approach. This paper reviews the genetic therapy for inherited bleeding disorders utilizing platelets as delivery system, with a particular focus on platelet-derived FVIII for hemophilia A treatment. (c) 2010 Elsevier B.V. All rights reserved.
作者
我是这篇论文的作者
点击您的名字以认领此论文并将其添加到您的个人资料中。
推荐
暂无数据