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

作者：Hohwieler Meike; Perkhofer Lukas; Liebau Stefan; Seufferlein Thomas; Mueller Martin; Illing Anett; Kleger Alexander

来源：United European Gastroenterology Journal, 2017, 5(5): 609-624.

DOI：10.1177/2050640616670565

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

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.

• 出版日期2017-8