title: Platelet-delivered therapeutics.
abstract: We have proposed that modified platelets could potentially be used to correct intrinsic platelet defects as well as for targeted delivery of therapeutic molecules to sights of vascular injury. Ectopic expression of proteins within α-granules prior to platelet activation has been achieved for several proteins, including urokinase, factor (F) VIII, and partially for FIX. Potential uses of platelet-directed therapeutics will be discussed, focusing on targeted delivery of urokinase as a thromboprophylactic agent and FVIII for the treatment of hemophilia A patients with intractable inhibitors. This presentation will discuss new strategies that may be useful in the care of patients with vascular injury as well as remaining challenges and limitations of these approaches.
authors.affiliation: Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Department of Pharmacology, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: R
authors.initials: R
authors.lastname: Lyde
authors.affiliation: Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: D
authors.initials: D
authors.lastname: Sabatino
authors.affiliation: Division of Pediatric Hematology/Oncology, Department of Pediatrics, University of Mississippi Medical Center, Jackson, MS, USA.
authors.forename: S K
authors.initials: SK
authors.lastname: Sullivan
authors.affiliation: Division of Hematology, Department of Pediatrics, Children's Hospital of Philadelphia, Philadelphia, PA, USA.|Department of Pediatrics, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA, USA.
authors.forename: M
authors.initials: M
authors.lastname: Poncz
mesh_terms: D000818:Animals; D001778:Blood Coagulation Disorders / Q000097:blood / Q000235:genetics / Q000628:therapy*; D001779:Blood Coagulation Factors / Q000096:biosynthesis / Q000235:genetics; D001791:Blood Platelet Disorders / Q000097:blood / Q000235:genetics / Q000628:therapy*; D001792:Blood Platelets / Q000378:metabolism*; D005166:Factor V Deficiency / Q000097:blood / Q000235:genetics / Q000628:therapy; D018014:Gene Transfer Techniques; D020022:Genetic Predisposition to Disease; D015316:Genetic Therapy / Q000379:methods*; D006467:Hemophilia A / Q000097:blood / Q000235:genetics / Q000628:therapy; D006801:Humans; D017713:Platelet Transfusion; D016896:Treatment Outcome; D014568:Urokinase-Type Plasminogen Activator / Q000096:biosynthesis / Q000097:blood / Q000235:genetics
chemical_list: D001779:Blood Coagulation Factors; D014568:Urokinase-Type Plasminogen Activator
keywords: factor VIII; fibrinolysis; gene therapy; hemophilia; platelet; urokinase