Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation

The study of biliary disease has been constrained by a lack of primary human cholangiocytes. Here we present an efficient, serum-free protocol for directed differentiation of human induced pluripotent stem cells into cholangiocyte-like cells (CLCs). CLCs show functional characteristics of cholangioc...

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Main Authors: Sampaziotis, Fotios, Cardoso de Brito, Miguel, Madrigal, Pedro, Bertero, Alessandro, Saeb-Parsy, Kourosh, Soares, Filipa A.C., Schrumpf, Elisabeth, Melum, Espen, Karlsen, Tom H., Bradley, J. Andrew, Gelson, William T.H., Davies, Susan, Baker, Alastair, Kaser, Arthur, Alexander, Graeme J., Hannan, Nicholas R.F., Vallier, Ludovic
Format: Article
Published: Nature Publishing Group 2015
Online Access:https://eprints.nottingham.ac.uk/43037/
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author Sampaziotis, Fotios
Cardoso de Brito, Miguel
Madrigal, Pedro
Bertero, Alessandro
Saeb-Parsy, Kourosh
Soares, Filipa A.C.
Schrumpf, Elisabeth
Melum, Espen
Karlsen, Tom H.
Bradley, J. Andrew
Gelson, William T.H.
Davies, Susan
Baker, Alastair
Kaser, Arthur
Alexander, Graeme J.
Hannan, Nicholas R.F.
Vallier, Ludovic
author_facet Sampaziotis, Fotios
Cardoso de Brito, Miguel
Madrigal, Pedro
Bertero, Alessandro
Saeb-Parsy, Kourosh
Soares, Filipa A.C.
Schrumpf, Elisabeth
Melum, Espen
Karlsen, Tom H.
Bradley, J. Andrew
Gelson, William T.H.
Davies, Susan
Baker, Alastair
Kaser, Arthur
Alexander, Graeme J.
Hannan, Nicholas R.F.
Vallier, Ludovic
author_sort Sampaziotis, Fotios
building Nottingham Research Data Repository
collection Online Access
description The study of biliary disease has been constrained by a lack of primary human cholangiocytes. Here we present an efficient, serum-free protocol for directed differentiation of human induced pluripotent stem cells into cholangiocyte-like cells (CLCs). CLCs show functional characteristics of cholangiocytes, including bile acids transfer, alkaline phosphatase activity, γ-glutamyl-transpeptidase activity and physiological responses to secretin, somatostatin and vascular endothelial growth factor. We use CLCs to model in vitro key features of Alagille syndrome, polycystic liver disease and cystic fibrosis (CF)-associated cholangiopathy. Furthermore, we use CLCs generated from healthy individuals and patients with polycystic liver disease to reproduce the effects of the drugs verapamil and octreotide, and we show that the experimental CF drug VX809 rescues the disease phenotype of CF cholangiopathy in vitro. Our differentiation protocol will facilitate the study of biological mechanisms controlling biliary development, as well as disease modeling and drug screening.
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spelling nottingham-430372020-05-04T17:10:48Z https://eprints.nottingham.ac.uk/43037/ Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation Sampaziotis, Fotios Cardoso de Brito, Miguel Madrigal, Pedro Bertero, Alessandro Saeb-Parsy, Kourosh Soares, Filipa A.C. Schrumpf, Elisabeth Melum, Espen Karlsen, Tom H. Bradley, J. Andrew Gelson, William T.H. Davies, Susan Baker, Alastair Kaser, Arthur Alexander, Graeme J. Hannan, Nicholas R.F. Vallier, Ludovic The study of biliary disease has been constrained by a lack of primary human cholangiocytes. Here we present an efficient, serum-free protocol for directed differentiation of human induced pluripotent stem cells into cholangiocyte-like cells (CLCs). CLCs show functional characteristics of cholangiocytes, including bile acids transfer, alkaline phosphatase activity, γ-glutamyl-transpeptidase activity and physiological responses to secretin, somatostatin and vascular endothelial growth factor. We use CLCs to model in vitro key features of Alagille syndrome, polycystic liver disease and cystic fibrosis (CF)-associated cholangiopathy. Furthermore, we use CLCs generated from healthy individuals and patients with polycystic liver disease to reproduce the effects of the drugs verapamil and octreotide, and we show that the experimental CF drug VX809 rescues the disease phenotype of CF cholangiopathy in vitro. Our differentiation protocol will facilitate the study of biological mechanisms controlling biliary development, as well as disease modeling and drug screening. Nature Publishing Group 2015-06-13 Article PeerReviewed Sampaziotis, Fotios, Cardoso de Brito, Miguel, Madrigal, Pedro, Bertero, Alessandro, Saeb-Parsy, Kourosh, Soares, Filipa A.C., Schrumpf, Elisabeth, Melum, Espen, Karlsen, Tom H., Bradley, J. Andrew, Gelson, William T.H., Davies, Susan, Baker, Alastair, Kaser, Arthur, Alexander, Graeme J., Hannan, Nicholas R.F. and Vallier, Ludovic (2015) Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation. Nature Biotechnology, 33 (8). pp. 845-852. ISSN 1546-1696 https://www.nature.com/nbt/journal/v33/n8/full/nbt.3275.html doi:10.1038/nbt.3275 doi:10.1038/nbt.3275
spellingShingle Sampaziotis, Fotios
Cardoso de Brito, Miguel
Madrigal, Pedro
Bertero, Alessandro
Saeb-Parsy, Kourosh
Soares, Filipa A.C.
Schrumpf, Elisabeth
Melum, Espen
Karlsen, Tom H.
Bradley, J. Andrew
Gelson, William T.H.
Davies, Susan
Baker, Alastair
Kaser, Arthur
Alexander, Graeme J.
Hannan, Nicholas R.F.
Vallier, Ludovic
Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title_full Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title_fullStr Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title_full_unstemmed Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title_short Cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
title_sort cholangiocytes derived from human induced pluripotent stem cells for disease modeling and drug validation
url https://eprints.nottingham.ac.uk/43037/
https://eprints.nottingham.ac.uk/43037/
https://eprints.nottingham.ac.uk/43037/