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Cystic Fibrosis: Management

Cystic Fibrosis: Management

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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Cystic Fibrosis: Pathogenesis

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Related Experiment Video

Updated: Apr 7, 2026

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

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

Published on: February 11, 2017

Keeping up with CFTR modulator eligibility.

Rebecca Jackson¹, Siân Bentley², Jane C Davies³

¹National Heart and Lung Institute, Imperial College, London, UK; Department of Adult Cystic Fibrosis, Royal Brompton Hospital, London, UK; Department of Paediatric Cystic Fibrosis, Royal Brompton Hospital, London, UK.

Paediatric Respiratory Reviews

|April 5, 2026

Summary

This summary is machine-generated.

Highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators have transformed patient outcomes. Evolving eligibility criteria and personalized medicine strategies aim to optimize treatment response for more individuals with cystic fibrosis.

Keywords:

CFTR modulators Cystic fibrosis Elexacaftor Eligibility Ivacaftor Lumacaftor Organoids Tezacaftor Vanzacaftor

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Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

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Last Updated: Apr 7, 2026

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

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

Published on: February 11, 2017

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

Functional Reconstitution and Channel Activity Measurements of Purified Wildtype and Mutant CFTR Protein

Published on: March 9, 2015

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study

Published on: April 11, 2018

Area of Science:

Medical Science
Pharmacology
Genetics

Background:

Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have revolutionized treatment for cystic fibrosis.
Initial eligibility for CFTR modulators was based on clinical trial data for specific variants.

Purpose of the Study:

To outline the evolving approaches for determining eligibility for CFTR modulators.
To discuss strategies for improving prediction, verification, and monitoring of clinical response.
To highlight the shift towards personalized medicine in cystic fibrosis care.

Main Methods:

Review of regulatory approvals and evidence used for expanding CFTR modulator eligibility.
Analysis of factors influencing variable clinical response to CFTR modulators.
Discussion of advancements in in vitro 'theratyping' and real-world evidence utilization.

Main Results:

CFTR modulator eligibility has expanded to over 180 variants beyond initial approvals.
Regulatory bodies increasingly accept non-traditional evidence, such as cell-based assays.
Clinical response to CFTR modulators is variable and influenced by multiple factors.

Conclusions:

Personalized medicine approaches are crucial for optimizing CFTR modulator therapy.
Continued research is needed to refine methods for predicting and monitoring treatment response.
Advancements in evidence generation are broadening access to effective CFTR modulator therapies.