Peter van Mourik

15 General Introduction REFERENCES 1. Clevers, H. Modeling Development and Disease with Organoids. Cell 165, 1586–1597 (2016). 2. Sachs, N. et al. A Living Biobank of Breast Cancer Organoids Captures Disease Heterogeneity. Cell 172, 373–386 (2018). 3. Drost, J. & Clevers, H. Organoids in cancer research. Nat. Rev. Cancer 18, 407–418 (2018). 4. Sato, T. et al. Long-term expansion of epithelial organoids from human colon, adenoma, adenocarcinoma, and Barrett’s epithelium. Gastroenterology 141, 1762–72 (2011). 5. Friedmacher, F. & Puri, P. Rectal suction biopsy for the diagnosis of Hirschsprung’s disease: a systematic review of diagnostic accuracy and complications. Pediatr. Surg. Int. 31, 821–30 (2015). 6. Servidoni, M. F. et al. Rectal forceps biopsy procedure in cystic fibrosis: technical aspects and patients perspective for clinical trials feasibility. BMC Gastroenterol. 13, 91 (2013). 7. Dekkers, J. F. et al. Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis. Sci. Transl. Med. 8, 344ra84 (2016). 8. Wallach, T. E. & Bayrer, J. R. Intestinal Organoids: New Frontiers in the Study of Intestinal Disease and Physiology. J. Pediatr. Gastroenterol. Nutr. 64, 180–185 (2017). 9. Thiagarajah, J. R., Broadbent, T., Hsieh, E. & Verkman, A. S. Prevention of toxin- induced intestinal ion and fluid secretion by a small-molecule CFTR inhibitor. Gastroenterology 126, 511–9 (2004). 10. Dekkers, J. F. et al. A functional CFTR assay using primary cystic fibrosis intestinal organoids. Nat. Med. 19, 939–45 (2013). 11. Boj, S. F. et al. Forskolin-induced Swelling in Intestinal Organoids: An <em>In Vitro</em> Assay for Assessing Drug Response in Cystic Fibrosis Patients. J. Vis. Exp. 1–12 (2017). doi:10.3791/55159 12. Riordan, J. R. et al. Identification of the cystic fibrosis gene: cloning and characterization of complementary DNA. Science 245, 1066–73 (1989). 13. Foulke-Abel, J. et al. Human Enteroids as a Model of Upper Small Intestinal Ion Transport Physiology and Pathophysiology. Gastroenterology 150, 638–649.e8 (2016). 14. Sachs, N. et al. Long-term expanding human airway organoids for disease modeling. EMBO J. 38, e100300 (2019). 15. de Winter-de Groot, K. M. et al. Stratifying infants with cystic fibrosis for disease severity using intestinal organoid swelling as a biomarker of CFTR function. Eur. Respir. J. 52, 1702529 (2018). 16. Zomer-van Ommen, D. D. et al. Comparison of ex vivo and in vitro intestinal cystic fibrosis models to measure CFTR-dependent ion channel activity. J. Cyst. Fibros. 17, 316–324 (2018). 1

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