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Updated: Jun 13, 2026
Generation of Human Nasal Epithelial Cell Spheroids for Individualized Cystic Fibrosis Transmembrane Conductance Regulator Study
Published on: April 11, 2018
Cystic fibrosis gene therapy: successes, failures and hopes for the future
Uta Griesenbach1, Eric W F W Alton
1Department of Gene Therapy, Faculty of Medicine at the National Heart and Lung Institute, Imperial College London, Manresa Road, London SW3 6LR, UK. u.griesenbach@imperial.ac.uk
Abstract:
Cystic fibrosis (CF) is a single-gene disorder with insufficient treatment options and a target organ, the lung that is relatively easily accessible. Thus, it is not surprising that in the early years of gene therapy, CF was at the forefront of this field. Since cloning of the CF gene in 1989, 25 Phase I/II clinical trials involving approximately 420 CF patients have been carried out using a variety of viral and nonviral gene transfer agents. Most early trials focused on the nasal epithelium as a surrogate for the lung to allow for easy access and sampling, and, importantly, to ensure safety. Once an acceptable safety profile had been established, gene transfer agents were administered directly into the lung. Although many of these trials established proof-of-principle for gene transfer in the airways, a gene therapy-based treatment has not yet been developed. Here, we will summarize the key findings of these clinical studies and describe current preclinical and clinical research aimed at further developing gene therapy for CF.
Insights
Gene therapy for cystic fibrosis (CF) has been explored since 1989, with numerous clinical trials demonstrating proof-of-principle. However, a widely available gene therapy treatment for CF lung disease is still under development.
Area of Science:
- Medical research
- Genetics
- Pulmonology
Background:
- Cystic fibrosis (CF) is a genetic disorder impacting the lungs, with limited treatment options.
- The lung's accessibility makes it a prime target for gene therapy interventions.
- Gene therapy for CF has been a focus since the CF gene's cloning in 1989.
Purpose of the Study:
- To summarize key findings from Phase I/II clinical trials of gene therapy for CF.
- To review current preclinical and clinical research in CF gene therapy development.
Main Methods:
- Analysis of 25 Phase I/II clinical trials involving approximately 420 CF patients.
- Review of viral and nonviral gene transfer agents used in CF gene therapy.
- Examination of studies using nasal epithelium and direct lung administration.
Main Results:
- Early trials established safety and proof-of-principle for gene transfer in CF airways.
- Gene transfer agents were tested in both nasal epithelium and directly in the lungs.
- Despite progress, a clinically approved gene therapy for CF has not yet been achieved.
Conclusions:
- Gene therapy for cystic fibrosis has shown promise but requires further development.
- Ongoing research focuses on advancing gene transfer technologies for effective CF treatment.
- Translating gene therapy into a viable treatment for CF remains an active area of investigation.
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