Faculty Collaboration Database

Platelets as delivery systems for disease treatments. Adv Drug Deliv Rev 2010 Sep 30;62(12):1196-203

Date

07/14/2010

Pubmed ID

20619307

Pubmed Central ID

PMC2991519

DOI

10.1016/j.addr.2010.06.007

Scopus ID

2-s2.0-78649321428 (requires institutional sign-in at Scopus site)   48 Citations

Abstract

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.

Author List

Shi Q, Montgomery RR

Authors

Robert R. Montgomery MD Adjunct Professor in the Pediatrics department at Medical College of Wisconsin
Qizhen Shi MD, PhD Professor in the Pediatrics department at Medical College of Wisconsin

MESH terms used to index this publication - Major topics in bold

Animals
Blood Coagulation
Blood Coagulation Disorders
Blood Platelets
Cytoplasmic Granules
Factor VIII
Genetic Therapy
Hemophilia A
Hemophilia B
Hemostasis
Humans
Megakaryocytes
Platelet Count