Fig. 7. Patient specific mesenchyme-free-derived HIOs are suitable for disease modeling.

a Schematic overview of experiment to measure CFTR function in ΔF508, ΔF508-corrected and WT HIOs (n = 3 independent differentiations per iPSC line). b Representative micrograph of whole mount of distally patterned HIOs generated from the C17 iPSC line at day 54 of differentiation, sorted for NKX2-1^GFP− at day 15, and cultured in CK + DCI (scale bar = 50 μm, representative of n = 2 differentiations). c qRT-PCR for CFTR expression in HIOs cultured in both IM + CK and CK + DCI at day 54 of differentiation (2^−ΔΔCT, technical triplicates normalized to GAPDH; n = 3). d Average baseline organoid size of WT, ΔF508 and ΔF508-corrected HIOs (n = 192 mean spheres analyzed per iPSC line). e Quantification of the steady-state lumen area (SLA) in ΔF508 and ΔF508-corrected HIOs (see also Supplementary Fig. 7b). f Representative micrographs of HIOs pre- and post-24-h forskolin treatment (representative scale bar = 200 μm in upper left image, images represent n = 3 biological replicates per cell line and an average of n = 8 wells per replicate). g Quantification of change in whole well CSA in response to 24-h forskolin treatment (normalized to T = 0 h whole well CSA). Statistical significance where indicated determined by unpaired Student’s t-test, *p < 0.05, **p < 0.005 (n = 3 independent differentiations per cell line, error bars represent the s.d.).