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United European Gastroenterol J. 2017 Aug;5(5):609-624. doi: 10.1177/2050640616670565. Epub 2016 Sep 21.

Stem cell-derived organoids to model gastrointestinal facets of cystic fibrosis.

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Department of Internal Medicine 1, University Medical Centre Ulm, Ulm, Germany.
Institute of Neuroanatomy, Eberhard Karls University Tuebingen, Oesterbergstr. 3, 72074 Tuebingen, Germany.


Cystic fibrosis (CF) is one of the most frequently occurring inherited human diseases caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) which lead to ample defects in anion transport and epithelial fluid secretion. Existing models lack both access to early stages of CF development and a coeval focus on the gastrointestinal CF phenotypes, which become increasingly important due increased life span of the affected individuals. Here, we provide a comprehensive overview of gastrointestinal facets of CF and the opportunity to model these in various systems in an attempt to understand and treat CF. A particular focus is given on forward-leading organoid cultures, which may circumvent current limitations of existing models and thereby provide a platform for drug testing and understanding of disease pathophysiology in gastrointestinal organs.


Pancreas development; cystic fibrosis; induced pluripotent stem cells; organoids; stem cells

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