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. Author manuscript; available in PMC: 2020 Jan 1.
Published in final edited form as: J Cyst Fibros. 2018 Jun 20;18(1):22–34. doi: 10.1016/j.jcf.2018.05.004

Table 1: Preclinical model systems for theratyping.

 Model system  Source  Level of advancement  Most common uses
Transient CFTR expression
in heterologous cell lines
Established
cell lines
high Characterize CFTR variants
and CFTR biology
Stable CFTR transduction
in cell lines
Established
cell lines

high
HTS screening, evaluate
common and rare CFTR
variants in standardized system

Human bronchial airway
epithelial (HBE) planar cultures

Lung explant

high
Validation of CFTR modulation
in primary human cells, assess
downstream effects (e.g.
mucocilary clearance, airway
surface liquid height)

Rectal organoids

Rectal biopsy

moderate
Validation of CFTR modulation
in primary human cells, and
patient-specific responses to
modulators (fluid secretion)
Duodenal enteroids Duodenal
biopsy
low Similar to rectal organoids
Above

HBE planar cultures from
brush

Bronchial brush

low
Similar to HBEs from lung
explant, and patient-specific
responses to modulators (ion
transport)
Human nasal epithelial
(HNE) planar cultures from
brush

Nasal brush

low
Similar to HBEs from lung explant,
and patient-specific responses to
modulators (ion
transport)

HNE spheres

Nasal brush

low
Similar to HBEs from lung
explant, and patient-specific
responses to modulators (fluid
transport)
Induced pluripotent stem cells (iPSCs)
Blood

low
Differentiation into CFTR- expressing epithelial cells and
tissues