Related Experiment Videos
Gene therapy for cystic fibrosis
1Department of Medicine, University of North Carolina at Chapel Hill.
Chest
|February 1, 1995
Summary
Gene therapy for cystic fibrosis (CF) requires in vivo delivery of gene transfer vectors to the airways. Adenovirus and liposome vectors are being evaluated in clinical trials for CF gene therapy safety and efficacy.
Area of Science:
- Biotechnology
- Medical Genetics
- Pulmonology
Background:
- Cystic Fibrosis (CF) is a genetic disease.
- Gene therapy offers a potential treatment for CF.
- In vitro studies confirmed the feasibility of CF gene therapy.
Purpose of the Study:
- To review the rationale and laboratory studies for CF gene therapy.
- To evaluate currently available gene transfer vectors for CF.
- To discuss the promises and pitfalls of CF gene therapy, focusing on adenovirus vectors.
Main Methods:
- Review of laboratory studies and clinical trial data.
- Evaluation of adenovirus-mediated and liposome-mediated gene transfer vectors.
- Focus on in vivo gene delivery to the airways for CF patients.
Main Results:
- Several gene transfer vectors have advanced to clinical trials in the US and UK.
- Adenovirus vectors are used in US trials; liposome vectors in UK trials.
- Initial safety and efficacy data from these trials are being gathered.
Conclusions:
- In vivo gene therapy is the likely approach for CF.
- Development of safe and efficient gene transfer vectors is crucial.
- Ongoing clinical trials will determine the future of CF gene therapy.