Table of Contents
I
230
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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