Intestinal organoids for Cystic Fibrosis research

Significant progress has been made in the development of CFTR modulator therapy; however, current CFTR modulator therapies are only available for a minority of the CF-patient population. Additionally, heterogeneity in in vivo modulator response has been reported among individuals carrying homozygous...

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Bibliographic Details
Published in:Journal of cystic fibrosis 2020-03, Vol.19, p.S60-S64
Main Authors: de Poel, E., Lefferts, J.W., Beekman, J.M.
Format: Article
Language:English
Subjects:
CFTR modulators
Cystic Fibrosis
Drug development
In vitro model systems
Organoids
Personalized medicine
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Summary:Significant progress has been made in the development of CFTR modulator therapy; however, current CFTR modulator therapies are only available for a minority of the CF-patient population. Additionally, heterogeneity in in vivo modulator response has been reported among individuals carrying homozygous F508del-CFTR, adding to the desire for an optimal prediction of response-to-therapy on an individual level. In the last decade, a lot of progress has been made in the development of primary cell cultures into 3D patient-derived disease models. The advantage of these models is that the endogenous CFTR function is affected by the patient's mutation as well as other genetic or environmental factors. In this review we focus on intestinal organoids as in vitro model for CF, enabling for CF disease classification, drug development and treatment optimization in a personalized manner, taking into account rare CFTR mutations and clinical heterogeneity among individuals with CF.
ISSN:1569-1993
1873-5010
DOI:10.1016/j.jcf.2019.11.002