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Related Experiment Video

Updated: Mar 2, 2026

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients

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Published on: February 11, 2017

In Utero CFTR Modulator Therapy in Fetuses With Cystic Fibrosis.

Angela Metcalf¹, Jordana E Hoppe¹, Stacey L Martiniano¹

¹Department of Pediatrics, University of Colorado Anschutz Medical Campus and Children's Hospital Colorado, Aurora, Colorado.

|February 28, 2026

Summary

This summary is machine-generated.

CFTR modulator therapies show promise for treating fetal meconium ileus during pregnancy. However, potential benefits must be carefully weighed against risks for pregnant individuals and their offspring.

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Area of Science:

Medical Research
Pharmacology
Genetics

Background:

Cystic Fibrosis (CF) affects multiple organs due to CFTR protein dysfunction.
CFTR modulators are small molecule therapies that restore CFTR protein function.
Meconium ileus is a severe manifestation of CF in newborns.

Purpose of the Study:

To review current data on CFTR modulator therapy use during pregnancy.
To evaluate the potential of in utero CFTR modulator therapy for meconium ileus.
To discuss risks, ethical implications, and knowledge gaps of prenatal CFTR modulator use.

Main Methods:

Literature review of existing studies and case reports.
Analysis of reported effects in pregnant individuals and neonates.
Examination of potential therapeutic benefits and risks.

Main Results:

CFTR modulators may prevent meconium ileus and preserve exocrine pancreatic function in utero.
Data on safety and efficacy in pregnant populations is limited.
Potential risks to both mother and fetus require careful consideration.

Conclusions:

Prenatal CFTR modulator therapy presents potential benefits for fetal CF, including meconium ileus prevention.
Balancing potential benefits against known and unknown risks is crucial.
Further research is essential to establish best practices for prenatal CFTR modulator use.