• Introduction to gene therapy
• Why use gene therapy for CF?
• Other CF gene therapy groups around the world
• Examples of successful gene therapy
• Gene Therapy Links

A diagram of the human large airways. One of the most compelling reasons to attempt gene therapy for CF was that the airways should present a relatively simple, easy to reach target. In truth the airways present many more barriers to successful gene therapy than most other organs.

Many diseases such as cystic fibrosis, muscular dystrophy, haemophilia and cancer are caused by faulty genes.

Gene therapy involves the addition of a healthy, working copy of the gene into appropriate cells in the body to replace or override the faulty copy present in the genome.

Unlike most conventional medicines, instead of treating the symptoms of a disease, gene therapy has the potential to correct the underlying cause.