Journal article
Solving Cystic Fibrosis: From lab Bench to Bedside
Frontiers for young minds, Vol.13, 1650140
10/06/2025
DOI: 10.3389/frym.2025.1650140
Abstract
Cystic fibrosis (CF) is a serious genetic disease that causes thick, sticky mucus to build up in the lungs and digestive system. For many years, treatments could only manage the symptoms. That began to change with the discovery that CF is caused by problems with a protein called CFTR, which normally helps keep mucus thin and slippery. When CFTR is missing or does not work properly, mucus becomes too thick, leading to serious health problems. After scientists discovered how CFTR works and what goes wrong in CF, another team began developing medicines that help the faulty protein fold correctly, reach the cell surface, and open to let chloride pass through. Today, powerful triple therapies that combine these drugs work for about 90% of people with CF. This work has changed what it means to live with the disease—and has shown how understanding the root of a problem can lead to real solutions.
Details
- Title: Subtitle
- Solving Cystic Fibrosis: From lab Bench to Bedside
- Creators
- Michael J. WelshPaul Negulescu
- Resource Type
- Journal article
- Publication Details
- Frontiers for young minds, Vol.13, 1650140
- DOI
- 10.3389/frym.2025.1650140
- ISSN
- 2296-6846
- eISSN
- 2296-6846
- Language
- English
- Date published
- 10/06/2025
- Academic Unit
- Neurology; Molecular Physiology and Biophysics; Pulmonary, Critical Care, and Occupational Medicine; Fraternal Order of Eagles Diabetes Research Center; Neurosurgery; Internal Medicine
- Record Identifier
- 9985014874502771
Metrics
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