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Updated: Jul 30, 2026
07:04
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
Gene therapy for cystic fibrosis.
J C Davies1, D M Geddes, E W Alton
1Department of Gene Therapy, Imperial College at the National Heart and Lung Institute, London, UK. j.c.davies@ic.ac.uk
The Journal of Gene Medicine
|October 17, 2001
Summary
Gene therapy offers a promising approach to treating cystic fibrosis (CF) by targeting the cystic fibrosis transmembrane conductance regulator (CFTR) gene. This review examines gene transfer progress and challenges in CF clinical trials.
Area of Science:
- Molecular biology and genetics
- Medical research and clinical trials
- Gene therapy applications
Background:
- Cystic fibrosis (CF) remains a significant cause of morbidity and mortality.
- Conventional treatments have limitations despite advancements.
- The cloning of the cystic fibrosis transmembrane conductance regulator (CFTR) gene spurred gene therapy research.
Purpose of the Study:
- To review the molecular defect underlying cystic fibrosis.
- To discuss the progress of gene transfer studies in CF.
- To evaluate the development of endpoints for assessing gene therapy efficacy in clinical trials.
Main Methods:
- Review of existing literature on CF molecular defect.
- Analysis of gene transfer studies from in vitro to clinical trials.
- Examination of established efficacy endpoints for CF gene therapy.
Main Results:
- Significant progress has been made in understanding CF molecular pathology.
- Gene transfer studies have advanced from laboratory research to human clinical trials.
- Development of specific endpoints is crucial for evaluating gene therapy effectiveness.
Conclusions:
- Gene therapy holds potential for treating cystic fibrosis.
- Challenges in gene transfer and delivery persist.
- Future research directions are critical for overcoming current limitations and advancing CF treatment.
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