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Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency

Induced pluripotent stem cells (iPSCs) represent an innovative source for the standardized in vitro generation of macrophages (Mφ). We here describe a robust and efficient protocol to obtain mature and functional Mφ from healthy as well as disease-specific murine iPSCs. With regard to morphology, su...

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Autores principales: Mucci, Adele, Kunkiel, Jessica, Suzuki, Takuji, Brennig, Sebastian, Glage, Silke, Kühnel, Mark P., Ackermann, Mania, Happle, Christine, Kuhn, Alexandra, Schambach, Axel, Trapnell, Bruce C., Hansen, Gesine, Moritz, Thomas, Lachmann, Nico
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2016
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4982988/
https://www.ncbi.nlm.nih.gov/pubmed/27453007
http://dx.doi.org/10.1016/j.stemcr.2016.06.011
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author Mucci, Adele
Kunkiel, Jessica
Suzuki, Takuji
Brennig, Sebastian
Glage, Silke
Kühnel, Mark P.
Ackermann, Mania
Happle, Christine
Kuhn, Alexandra
Schambach, Axel
Trapnell, Bruce C.
Hansen, Gesine
Moritz, Thomas
Lachmann, Nico
author_facet Mucci, Adele
Kunkiel, Jessica
Suzuki, Takuji
Brennig, Sebastian
Glage, Silke
Kühnel, Mark P.
Ackermann, Mania
Happle, Christine
Kuhn, Alexandra
Schambach, Axel
Trapnell, Bruce C.
Hansen, Gesine
Moritz, Thomas
Lachmann, Nico
author_sort Mucci, Adele
collection PubMed
description Induced pluripotent stem cells (iPSCs) represent an innovative source for the standardized in vitro generation of macrophages (Mφ). We here describe a robust and efficient protocol to obtain mature and functional Mφ from healthy as well as disease-specific murine iPSCs. With regard to morphology, surface phenotype, and function, our iPSC-derived Mφ (iPSC-Mφ) closely resemble their counterparts generated in vitro from bone marrow cells. Moreover, when we investigated the feasibility of our differentiation system to serve as a model for rare congenital diseases associated with Mφ malfunction, we were able to faithfully recapitulate the pathognomonic defects in GM-CSF signaling and Mφ function present in hereditary pulmonary alveolar proteinosis (herPAP). Thus, our studies may help to overcome the limitations placed on research into certain rare disease entities by the lack of an adequate supply of disease-specific primary cells, and may aid the development of novel therapeutic approaches for herPAP patients.
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spelling pubmed-49829882016-08-19 Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency Mucci, Adele Kunkiel, Jessica Suzuki, Takuji Brennig, Sebastian Glage, Silke Kühnel, Mark P. Ackermann, Mania Happle, Christine Kuhn, Alexandra Schambach, Axel Trapnell, Bruce C. Hansen, Gesine Moritz, Thomas Lachmann, Nico Stem Cell Reports Resource Induced pluripotent stem cells (iPSCs) represent an innovative source for the standardized in vitro generation of macrophages (Mφ). We here describe a robust and efficient protocol to obtain mature and functional Mφ from healthy as well as disease-specific murine iPSCs. With regard to morphology, surface phenotype, and function, our iPSC-derived Mφ (iPSC-Mφ) closely resemble their counterparts generated in vitro from bone marrow cells. Moreover, when we investigated the feasibility of our differentiation system to serve as a model for rare congenital diseases associated with Mφ malfunction, we were able to faithfully recapitulate the pathognomonic defects in GM-CSF signaling and Mφ function present in hereditary pulmonary alveolar proteinosis (herPAP). Thus, our studies may help to overcome the limitations placed on research into certain rare disease entities by the lack of an adequate supply of disease-specific primary cells, and may aid the development of novel therapeutic approaches for herPAP patients. Elsevier 2016-07-21 /pmc/articles/PMC4982988/ /pubmed/27453007 http://dx.doi.org/10.1016/j.stemcr.2016.06.011 Text en © 2016 The Author(s) http://creativecommons.org/licenses/by-nc-nd/4.0/ This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/).
spellingShingle Resource
Mucci, Adele
Kunkiel, Jessica
Suzuki, Takuji
Brennig, Sebastian
Glage, Silke
Kühnel, Mark P.
Ackermann, Mania
Happle, Christine
Kuhn, Alexandra
Schambach, Axel
Trapnell, Bruce C.
Hansen, Gesine
Moritz, Thomas
Lachmann, Nico
Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title_full Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title_fullStr Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title_full_unstemmed Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title_short Murine iPSC-Derived Macrophages as a Tool for Disease Modeling of Hereditary Pulmonary Alveolar Proteinosis due to Csf2rb Deficiency
title_sort murine ipsc-derived macrophages as a tool for disease modeling of hereditary pulmonary alveolar proteinosis due to csf2rb deficiency
topic Resource
url https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4982988/
https://www.ncbi.nlm.nih.gov/pubmed/27453007
http://dx.doi.org/10.1016/j.stemcr.2016.06.011
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