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Updated: Aug 9, 2026
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
Cystic fibrosis clinical trials
1Ion Transport Unit, National Heart and Lung Institute, Emmanuel Kaye Building, Manresa Road, London SW3 6LR, UK
Abstract:
The ion transport abnormalities in cystic fibrosis are becoming increasingly well defined, although how these lead to lung pathology is still speculation. Correction of these defects could theoretically be achieved either through pharmacological means or via gene therapy. Pharmacological approaches include increasing the amount of CFTR protein that reaches its correct localisation in epithelial cells. Secondly, approaches have been suggested which could increase the function of the protein already present at this correct localisation. Finally, it may be possible to identify alternative channels which could subserve the function of CFTR. Gene therapy is theoretically an attractive proposition as it should circumvent each of the identified abnormalities in cystic fibrosis. The principal difficulty at present relates to delivering sufficient copies of the normal CFTR gene into the appropriate cell population in vivo. A number of clinical trials have now been undertaken and steady and encouraging progress has been made in moving this approach from theory to practice.
Insights
Cystic Fibrosis (CF) involves ion transport defects. Research explores correcting these through drugs or gene therapy, with gene therapy showing promising progress in clinical trials for CF treatment.
Area of Science:
- Biochemistry
- Molecular Biology
- Medical Science
Background:
- Cystic Fibrosis (CF) is characterized by ion transport abnormalities.
- The precise mechanisms linking these defects to lung pathology remain under investigation.
- Understanding these mechanisms is crucial for developing effective treatments.
Purpose of the Study:
- To review current strategies for correcting ion transport defects in CF.
- To evaluate the potential of pharmacological and gene therapy approaches.
- To highlight the progress and challenges in translating these therapies into practice.
Main Methods:
- Review of existing literature on CFTR protein function and dysfunction.
- Analysis of pharmacological strategies targeting CFTR protein localization and function.
- Assessment of gene therapy approaches, including delivery methods and clinical trial outcomes.
Main Results:
- Pharmacological strategies aim to increase CFTR protein levels at the correct site or enhance its function.
- Alternative ion channels are being explored to compensate for CFTR dysfunction.
- Gene therapy offers a theoretical solution by addressing all identified CF defects.
Conclusions:
- Significant progress has been made in developing both pharmacological and gene therapy for CF.
- The primary challenge for gene therapy lies in efficient in vivo delivery of the CFTR gene.
- Clinical trials indicate encouraging advancements in moving CFTR-targeted therapies from theory to practice.
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