Platelets as delivery systems for disease treatments.
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.
Shi
Qizhen
Q
Blood Research Institute, BloodCenter of Wisconsin, Departments of Pediatrics, Medical College of Wisconsin, Children's Hospital of Wisconsin, Milwaukee, USA. qizhen.shi@bcw.edu
Montgomery
Robert R
RR
D000818:Animals; D001777:Blood Coagulation; D001778:Blood Coagulation Disorders / Q000097:blood / Q000378:metabolism / Q000628:therapy*; D001792:Blood Platelets / Q000378:metabolism; D003594:Cytoplasmic Granules; D005169:Factor VIII / Q000235:genetics / Q000378:metabolism / Q000627:therapeutic use*; D015316:Genetic Therapy / Q000009:adverse effects; D006467:Hemophilia A / Q000097:blood / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D002836:Hemophilia B / Q000097:blood / Q000235:genetics / Q000378:metabolism / Q000628:therapy*; D006487:Hemostasis; D006801:Humans; D008533:Megakaryocytes / Q000502:physiology; D010976:Platelet Count
D005169:Factor VIII