Peter van Mourik

Chapter 1 General Introduction 7
Chapter 2 R117H-CFTR function and response to VX-770 correlate with mRNA and protein expression in intestinal organoids 19
Chapter 3 Potentiator synergy in rectal organoids carrying S1251N, G551D or F508del CFTR mutations 39
Chapter 4 Protocol for application, standardization and validation of the forskolin induced swelling assay in Cystic Fibrosis human colon organoids 67
Chapter 5 Centralized intestinal organoid generation is a feasible and safe approach for personalized medicine as demonstrated in the European HIT-CF study 119
Chapter 6 Comparison of organoid swelling and in vivo biomarkers of CFTR function to determine effects of lumacaftor-ivacaftor in patients with Cystic Fibrosis homozygous for the F508del mutation 129
Chapter 7 Rectal organoids enable personalized treatment of Cystic Fibrosis 139
Chapter 8 Rationale and design of the HIT-CF organoid study: stratifying Cystic Fibrosis patients based on intestinal organoid response to different CFTRmodulators 163
Chapter 9 General discussion 179
Chapter 10 Nederlandse samenvatting 203
Chapter 11 Addendum 213

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