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Updated: Jan 2, 2026
Author Spotlight: Generation and Manipulation of Rat Intestinal Organoids
Published on: June 23, 2023
Intestinal organoids for Cystic Fibrosis research
E de Poel1, J W Lefferts1, J M Beekman1
1Department of Pediatric Respiratory Medicine, Wilhelmina Children's Hospital, University Medical Center, Utrecht University, 3584 EA Utrecht, the Netherlands; Regenerative Medicine Utrecht, University Medical Center, Utrecht University, 3584 CT Utrecht, the Netherlands.
Abstract:
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.
Insights
Patient-derived intestinal organoids offer a promising model for cystic fibrosis (CF) research. These models can help classify CF disease, develop new drugs, and personalize treatments for individual patients with CFTR mutations.
Area of Science:
- Biomedical Engineering
- Genetics
- Pharmacology
Background:
- Current Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulator therapies benefit only a fraction of CF patients.
- Variability in treatment response among patients with homozygous F508del-CFTR highlights the need for personalized therapy prediction.
Purpose of the Study:
- To review the utility of intestinal organoids as in vitro models for Cystic Fibrosis (CF).
- To explore the potential of these models for CF disease classification, drug development, and personalized treatment optimization.
Main Methods:
- Focus on patient-derived intestinal organoids as 3D in vitro models.
- Analysis of how endogenous CFTR function is influenced by patient-specific mutations and other factors.
Main Results:
- Intestinal organoids capture patient-specific CFTR mutation effects and genetic/environmental influences.
- These models allow for the study of rare CFTR mutations and clinical heterogeneity.
Conclusions:
- Intestinal organoids represent a valuable tool for advancing personalized medicine in CF.
- These models facilitate CF disease classification, drug discovery, and tailored treatment strategies for diverse patient populations.
