Functional comparison of induced pluripotent stem cell-and blood-derived gpiibiiia deficient platelets
Orban M, Goedel A, Haas J, Sandrock-Lang K, Gaertner F, Jung CB, Zieger B, Parrotta E, Kurnik K, Sinnecker D, Wanner G, Laugwitz KL, Massberg S, Moretti A (2015)
Publication Type: Journal article
Publication year: 2015
Journal
Book Volume: 10
Article Number: 0115978
Journal Issue: 1
DOI: 10.1371/journal.pone.0115978
Abstract
Human induced pluripotent stem cells (hiPSCs) represent a versatile tool to model genetic diseases and are a potential source for cell transfusion therapies. However, it remains elusive to which extent patient-specific hiPSC-derived cells functionally resemble their native counterparts. Here, we generated a hiPSC model of the primary platelet disease Glanzmann thrombasthenia (GT), characterized by dysfunction of the integrin receptor GPIIbIIIa, and compared side-by-side healthy and diseased hiPSC-derived platelets with peripheral blood platelets. Both GT-hiPSC-derived platelets and their peripheral blood equivalents showed absence of membrane expression of GPIIbIIIa, a reduction of PAC-1 binding, surface spreading and adherence to fibrinogen. We demonstrated that GT-hiPSCderived platelets recapitulate molecular and functional aspects of the disease and show comparable behavior to their native counterparts encouraging the further use of hiPSCbased disease models as well as the transition towards a clinical application.
Involved external institutions
Ludwig-Maximilians-Universität (LMU)
Germany (DE)
Technische Universität München (TUM)
Germany (DE)
Universitätsklinikum Freiburg
Germany (DE)
Germany (DE)
Technische Universität München (TUM)
Germany (DE)
Universitätsklinikum Freiburg
Germany (DE)
How to cite
APA:
Orban, M., Goedel, A., Haas, J., Sandrock-Lang, K., Gaertner, F., Jung, C.B.,... Moretti, A. (2015). Functional comparison of induced pluripotent stem cell-and blood-derived gpiibiiia deficient platelets. PLoS ONE, 10(1). https://doi.org/10.1371/journal.pone.0115978
MLA:
Orban, Mathias, et al. "Functional comparison of induced pluripotent stem cell-and blood-derived gpiibiiia deficient platelets." PLoS ONE 10.1 (2015).
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