Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets.
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 gen...
| Main Authors: | , , , , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Public Library of Science (PLoS)
2015-01-01
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| Series: | PLoS ONE |
| Online Access: | http://europepmc.org/articles/PMC4301811?pdf=render |
| _version_ | 1818040845265272832 |
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| author | Mathias Orban Alexander Goedel Jessica Haas Kirstin Sandrock-Lang Florian Gärtner Christian Billy Jung Barbara Zieger Elvira Parrotta Karin Kurnik Daniel Sinnecker Gerhard Wanner Karl-Ludwig Laugwitz Steffen Massberg Alessandra Moretti |
| author_facet | Mathias Orban Alexander Goedel Jessica Haas Kirstin Sandrock-Lang Florian Gärtner Christian Billy Jung Barbara Zieger Elvira Parrotta Karin Kurnik Daniel Sinnecker Gerhard Wanner Karl-Ludwig Laugwitz Steffen Massberg Alessandra Moretti |
| author_sort | Mathias Orban |
| collection | DOAJ |
| description | 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-hiPSC-derived platelets recapitulate molecular and functional aspects of the disease and show comparable behavior to their native counterparts encouraging the further use of hiPSC-based disease models as well as the transition towards a clinical application. |
| first_indexed | 2024-12-10T08:21:00Z |
| format | Article |
| id | doaj.art-42e6caf4c10b4525940b5760c90e00bc |
| institution | Directory Open Access Journal |
| issn | 1932-6203 |
| language | English |
| last_indexed | 2024-12-10T08:21:00Z |
| publishDate | 2015-01-01 |
| publisher | Public Library of Science (PLoS) |
| record_format | Article |
| series | PLoS ONE |
| spelling | doaj.art-42e6caf4c10b4525940b5760c90e00bc2022-12-22T01:56:21ZengPublic Library of Science (PLoS)PLoS ONE1932-62032015-01-01101e011597810.1371/journal.pone.0115978Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets.Mathias OrbanAlexander GoedelJessica HaasKirstin Sandrock-LangFlorian GärtnerChristian Billy JungBarbara ZiegerElvira ParrottaKarin KurnikDaniel SinneckerGerhard WannerKarl-Ludwig LaugwitzSteffen MassbergAlessandra MorettiHuman 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-hiPSC-derived platelets recapitulate molecular and functional aspects of the disease and show comparable behavior to their native counterparts encouraging the further use of hiPSC-based disease models as well as the transition towards a clinical application.http://europepmc.org/articles/PMC4301811?pdf=render |
| spellingShingle | Mathias Orban Alexander Goedel Jessica Haas Kirstin Sandrock-Lang Florian Gärtner Christian Billy Jung Barbara Zieger Elvira Parrotta Karin Kurnik Daniel Sinnecker Gerhard Wanner Karl-Ludwig Laugwitz Steffen Massberg Alessandra Moretti Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. PLoS ONE |
| title | Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. |
| title_full | Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. |
| title_fullStr | Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. |
| title_full_unstemmed | Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. |
| title_short | Functional comparison of induced pluripotent stem cell- and blood-derived GPIIbIIIa deficient platelets. |
| title_sort | functional comparison of induced pluripotent stem cell and blood derived gpiibiiia deficient platelets |
| url | http://europepmc.org/articles/PMC4301811?pdf=render |
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