Supplementary Materials

Supplementary Material for:

Characterizing responses to CFTR-modulating drugs using rectal organoids derived from subjects with cystic fibrosis

Johanna F. Dekkers, Gitte Berkers, Evelien Kruisselbrink, Annelotte Vonk, Hugo R. de Jonge, Hettie M. Janssens, Inez Bronsveld, Eduard A. van de Graaf, Edward E. S. Nieuwenhuis, Roderick H. J. Houwen, Frank P. Vleggaar, Johanna C. Escher, Yolanda B. de Rijke, Christof J. Majoor, Harry G. M. Heijerman, Karin M. de Winter–de Groot, Hans Clevers, Cornelis K. van der Ent, Jeffrey M. Beekman*

*Corresponding author. Email: j.beekman{at}

Published 22 June 2016, Sci. Transl. Med. 8, 344ra84 (2016)
DOI: 10.1126/scitranslmed.aad8278

This PDF file includes:

  • Materials and Methods
  • Fig. S1. Characteristics of the SLA assay in organoids.
  • Fig. S2. Detectable residual CFTR function in several forskolin-stimulated organoids expressing severe CFTR mutations.
  • Fig. S3. Quantification of forskolin-induced organoid swelling.
  • Fig. S4. Genotype-specific profiles of residual and drug-corrected CFTR function in forskolin-stimulated mutant CFTR organoids.
  • Fig. S5. Data overview of clinical trials with CFTR-repairing treatment in subjects expressing different mutated CFTR genotypes.
  • Fig. S6. Pearson correlations of residual CFTR function and response to therapy measured by the FIS assay.
  • Fig. S7. CFTR correction in F508del homozygous organoids.
  • Fig. S8. In vivo indications for organoid-based selection of a clinical nonresponder to VX-770.
  • Table S1. Overview of all CFTR genotypes and their corresponding mutation classes and gender.

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