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Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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Related Experiment Video

Updated: Feb 23, 2026

Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
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Toward inclusive therapy with CFTR modulators: Progress and challenges.

Jennifer Guimbellot1,2, Jyoti Sharma2,3, Steven M Rowe1,2,3,4

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PubMed
Summary
This summary is machine-generated.

Cystic fibrosis treatments, known as CFTR modulators, target the underlying protein defect. Further research into biomarkers and preclinical tools is needed to personalize these therapies for all patients.

Keywords:
biomarkersclinical trialscystic fibrosis (CF)pharmacology

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Area of Science:

  • Biochemistry
  • Genetics
  • Pharmacology

Background:

  • Cystic fibrosis (CF) arises from mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene, leading to defective CFTR protein.
  • CFTR modulators represent a therapeutic class designed to correct the molecular defects underlying CF.

Purpose of the Study:

  • To review the current landscape of CFTR modulators in clinical development.
  • To discuss the role of biomarkers and preclinical tools in optimizing CFTR modulator therapy.
  • To explore pharmacometric approaches for personalized CF treatment strategies.

Main Methods:

  • Literature review of CFTR modulator classes (potentiators, correctors, readthrough agents).
  • Analysis of current clinical development status for CFTR modulators.
  • Examination of existing biomarkers and preclinical models for CFTR modulator efficacy.
  • Discussion of pharmacometric strategies for treatment personalization.

Main Results:

  • CFTR potentiators have shown significant patient benefits, but other modulators and treatments for rare mutations are still evolving.
  • A critical gap exists in validated biomarkers for individual efficacy assessment and preclinical tools for predicting response to specific modulator regimens.
  • Pharmacometrics offers a framework for developing tailored therapeutic strategies.

Conclusions:

  • While CFTR modulators have advanced CF care, challenges remain in broad patient applicability and personalized treatment.
  • Development of robust biomarkers and predictive preclinical models is crucial for expanding the reach of CFTR modulator therapies.
  • Personalized therapeutic strategies, informed by pharmacometrics, are essential to improve outcomes for all individuals with cystic fibrosis, irrespective of their specific mutation.