Related Experiment Video
Updated: Jan 28, 2026
08:00
Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
11.1K
Functional, Cohort-Level Assessment of CFTR Modulator Responses Using Biobanked Nasal Epithelial Cells from
Bente L Aalbers1, Gimano D Amatngalim2, Ellen M Aarts2
1Department of Pulmonology, University Medical Center Utrecht, 3584 EA Utrecht, The Netherlands.
Journal of Personalized Medicine
|January 27, 2026
Summary
Biobanked nasal cells from cystic fibrosis patients retain CFTR modulator responses. These functional assays correlate with clinical outcomes like sweat chloride and lung function, enabling broader theratyping.
Area of Science:
- Pulmonology
- Genetics
- Cell Biology
Background:
- Individual responses to CFTR modulators in cystic fibrosis (CF) vary.
- Nasal epithelial cells offer a personalized model for CFTR modulator theratyping.
- Current methods using fresh cells limit repeated testing and long-term use.
Purpose of the Study:
- To assess if CFTR modulator responses in biobanked nasal cells correlate with clinical outcomes in people with CF (pwCF).
- To evaluate the utility of cryopreserved nasal epithelial cultures for functional CFTR modulator testing.
Main Methods:
- Cryopreserved nasal epithelial cells from 23 pwCF were cultured at the air-liquid interface.
- CFTR modulator-responsive ion transport was measured using Ussing chambers.
- In vitro responses were correlated with changes in sweat chloride concentration, FEV1, and BMI.
Main Results:
- Cryopreserved cultures maintained donor-specific CFTR modulator responsiveness.
- Modulator-induced currents correlated with changes in sweat chloride concentration (R = -0.512).
- CFTR inhibitor-sensitive currents correlated with FEV1 (R = 0.564).
Conclusions:
- Biobanked nasal epithelial cultures preserve clinically relevant CFTR modulator responses at a cohort level.
- This cryopreservation strategy supports using functional assays for population-level CF assessment.
- It enables repeated testing and expands access to theratyping beyond fresh samples.
Related Concept Videos
01:23Cystic Fibrosis: Pathogenesis
864
Cystic fibrosis (CF), an autosomal recessive disorder, significantly affects the function of exocrine glands. This genetically inherited disease is characterized by the production of thick and sticky mucus, which can severely affect various organs and systems in the body.
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
CF is primarily caused by a genetic mutation in a chromosome 7 gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. The most common gene mutation leading to CF is the ΔF508 mutation,...
864
01:24Cystic Fibrosis: Management
516
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.
Sinus disease and chronic...
Sinus disease and chronic...
516
01:23Epithelial Tissues and Their Functions
38.6K
Epithelial tissues are large sheets of cells covering all of the surfaces of the body. These surfaces can be internal or external, for example, skin, airways, the digestive tract, the urinary system, and the reproductive system. Hollow organs and body cavities that do not connect to the body's exterior, including blood vessels and serous membranes, are lined by epithelial tissue known as the endothelium.
Epithelial tissues provide the body's first line of protection from physical,...
Epithelial tissues provide the body's first line of protection from physical,...
38.6K
01:30Impact of Individuals on Individuals
372
Human behavior is intricately shaped by social influences that arise from interactions with others in diverse contexts. These influences not only mold beliefs and attitudes but also drive the regulation of behaviors through both direct communication and observational learning. The study of these processes falls within the domain of social psychology, which seeks to understand how individuals are affected by and affect those around them.Mechanisms of Social InfluenceDirect social influence...
372
01:24Nose and Nasal Cavity
11.5K
The nose is composed of an observable exterior segment (external nose) and an internal segment within the skull known as the nasal cavity (internal nose). The external nose, visible on the face, consists of a framework of bone and hyaline cartilage enveloped in skin and muscle and lined with a mucous membrane. This structure is supported by the frontal bone, nasal bones, and maxillary bone and is supplemented by a cartilaginous framework comprising the septal nasal cartilage, lateral nasal...
11.5K
01:09Levels of Organization
140.3K
Biological organization is the classification of biological structures, ranging from atoms at the bottom of the hierarchy to the Earth's biosphere. Each level of the hierarchy represents an increase in complexity that builds upon the previous level.
Molecules Are Composed of Atoms, and Biomolecules Are Assembled from Molecules:
The most basic levels include atoms, molecules, and biomolecules. Atoms, the smallest unit of ordinary matter, are composed of a nucleus and electrons. Molecules...
Molecules Are Composed of Atoms, and Biomolecules Are Assembled from Molecules:
The most basic levels include atoms, molecules, and biomolecules. Atoms, the smallest unit of ordinary matter, are composed of a nucleus and electrons. Molecules...
140.3K