Induced Pluripotent Stem Cell-Derived Megakaryocytes and Platelets for Disease Modeling and Clinical Use

• Published in: Arteriosclerosis, thrombosis, and vascular biology Vol. 37; no. 11; pp. 2007 - 2013
• Main Authors: Borst, Sara, Sim, Xiuli, Poncz, Mortimer, French, Deborah L., Gadue, Paul
• Format: Journal Article
• Language: English
• Published: 05.10.2017
• ISSN: 1079-5642; 1524-4636
• DOI: 10.1161/ATVBAHA.117.309197

Platelets, derived from megakaryocytes, are anucleate cytoplasmic discs that circulate in the blood stream and play major roles in hemostasis, inflammation, and vascular biology. Platelet transfusions are utilized in a variety of medical settings to prevent life-threatening thrombocytopenia due to cancer therapy, other causes of acquired or inherited thrombocytopenia, and trauma. Currently, platelets used for transfusion purposes are donor-derived. However, there is a drive to generate non-donor sources of platelets to help supplement donor-derived platelets. Efforts have been made by many laboratories to generate in vitro platelets and optimize their production and quality. In vitro-derived platelets have the potential to be a safer, more uniform product, and genetic manipulation could allow for better treatment of patients that become refractory to donor-derived units. This review focuses on potential clinical applications of in vitro-derived megakaryocytes and platelets, current methods to generate and expand megakaryocytes from pluripotent stem cell sources, and the use of these cells for disease modeling.