Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine
Cystic fibrosis (CF) is an inherited disorder where individual disease etiology and response to therapeutic intervention is impacted by CF transmembrane regulator (CFTR) mutations and other genetic modifiers. CFTR regulates multiple mechanisms in a diverse range of epithelial tissues. In this Review...
| Main Authors: | , , , , , , |
|---|---|
| Format: | Journal Article |
| Language: | English |
| Published: |
FRONTIERS MEDIA SA
2018
|
| Subjects: | |
| Online Access: | http://hdl.handle.net/20.500.11937/76772 |
| _version_ | 1848763755060527104 |
|---|---|
| author | Awatade, N. Wong, S. Hewson, C. Fawcett, L. Kicic, Anthony Jaffe, A. Waters, S. |
| author_facet | Awatade, N. Wong, S. Hewson, C. Fawcett, L. Kicic, Anthony Jaffe, A. Waters, S. |
| author_sort | Awatade, N. |
| building | Curtin Institutional Repository |
| collection | Online Access |
| description | Cystic fibrosis (CF) is an inherited disorder where individual disease etiology and response to therapeutic intervention is impacted by CF transmembrane regulator (CFTR) mutations and other genetic modifiers. CFTR regulates multiple mechanisms in a diverse range of epithelial tissues. In this Review, we consolidate the latest updates in the development of primary epithelial cellular model systems relevant for CF. We discuss conventional two-dimensional (2-D) airway epithelial cell cultures, the backbone of in vitro cellular models to date, as well as improved expansion protocols to overcome finite supply of the cellular source. We highlight a range of strategies for establishment of three dimensional (3-D) airway and intestinal organoid models and evaluate the limitations and potential improvements in each system, focusing on their application in CF. The in vitro CFTR functional assays in patient-derived organoids allow for preclinical pharmacotherapy screening to identify responsive patients. It is likely that organoids will be an invaluable preclinical tool to unravel disease mechanisms, design novel treatments, and enable clinicians to provide personalized management for patients with CF. |
| first_indexed | 2025-11-14T11:08:30Z |
| format | Journal Article |
| id | curtin-20.500.11937-76772 |
| institution | Curtin University Malaysia |
| institution_category | Local University |
| language | English |
| last_indexed | 2025-11-14T11:08:30Z |
| publishDate | 2018 |
| publisher | FRONTIERS MEDIA SA |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | curtin-20.500.11937-767722019-11-11T03:30:42Z Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine Awatade, N. Wong, S. Hewson, C. Fawcett, L. Kicic, Anthony Jaffe, A. Waters, S. Science & Technology Life Sciences & Biomedicine Pharmacology & Pharmacy cystic fibrosis organoid personalized medicine CFTR drug development sweat chloride CFTR modulator INTESTINAL CURRENT MEASUREMENT IN-VITRO CFTR POTENTIATOR TEZACAFTOR-IVACAFTOR ION-TRANSPORT REGENERATION LUMACAFTOR ABSORPTION CORRECTORS SPHEROIDS Cystic fibrosis (CF) is an inherited disorder where individual disease etiology and response to therapeutic intervention is impacted by CF transmembrane regulator (CFTR) mutations and other genetic modifiers. CFTR regulates multiple mechanisms in a diverse range of epithelial tissues. In this Review, we consolidate the latest updates in the development of primary epithelial cellular model systems relevant for CF. We discuss conventional two-dimensional (2-D) airway epithelial cell cultures, the backbone of in vitro cellular models to date, as well as improved expansion protocols to overcome finite supply of the cellular source. We highlight a range of strategies for establishment of three dimensional (3-D) airway and intestinal organoid models and evaluate the limitations and potential improvements in each system, focusing on their application in CF. The in vitro CFTR functional assays in patient-derived organoids allow for preclinical pharmacotherapy screening to identify responsive patients. It is likely that organoids will be an invaluable preclinical tool to unravel disease mechanisms, design novel treatments, and enable clinicians to provide personalized management for patients with CF. 2018 Journal Article http://hdl.handle.net/20.500.11937/76772 10.3389/fphar.2018.01429 English http://creativecommons.org/licenses/by/4.0/ FRONTIERS MEDIA SA fulltext |
| spellingShingle | Science & Technology Life Sciences & Biomedicine Pharmacology & Pharmacy cystic fibrosis organoid personalized medicine CFTR drug development sweat chloride CFTR modulator INTESTINAL CURRENT MEASUREMENT IN-VITRO CFTR POTENTIATOR TEZACAFTOR-IVACAFTOR ION-TRANSPORT REGENERATION LUMACAFTOR ABSORPTION CORRECTORS SPHEROIDS Awatade, N. Wong, S. Hewson, C. Fawcett, L. Kicic, Anthony Jaffe, A. Waters, S. Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title | Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title_full | Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title_fullStr | Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title_full_unstemmed | Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title_short | Human Primary Epithelial Cell Models: Promising Tools in the Era of Cystic Fibrosis Personalized Medicine |
| title_sort | human primary epithelial cell models: promising tools in the era of cystic fibrosis personalized medicine |
| topic | Science & Technology Life Sciences & Biomedicine Pharmacology & Pharmacy cystic fibrosis organoid personalized medicine CFTR drug development sweat chloride CFTR modulator INTESTINAL CURRENT MEASUREMENT IN-VITRO CFTR POTENTIATOR TEZACAFTOR-IVACAFTOR ION-TRANSPORT REGENERATION LUMACAFTOR ABSORPTION CORRECTORS SPHEROIDS |
| url | http://hdl.handle.net/20.500.11937/76772 |