Our research in cystic fibrosis

Cystic fibrosis is the most common life-threatening, hereditary disease affecting Australian children. It is caused by a mutation in the gene responsible for regulating sweat, digestive juices, and mucus. When this gene is faulty, vital organs such as the gut, the lungs and the pancreas become blocked by mucous and cysts. In the lungs, serious chest infections and respiratory distress can develop early in childhood, and this can be accompanied by poor growth, digestive disorders, diarrhoea and infertility.

Our research in cystic fibrosis is focused on developing a safe and effective form of gene therapy to cure the lung disease caused by cystic fibrosis, the most common cause of early death among those who suffer from this condition. By inserting healthy genes into the defective, cystic fibrosis cells lining the airways in the lungs, we hope to halt or overcome the damaging effects of the disease on the respiratory system.

Our group has already demonstrated successful gene transfer in mice, and proven that faulty genetic information can be rectified with the insertion of normal DNA. It is hoped that as our research progresses we will be in a position to facilitate new and improved methods of gene transfer and application protocols, and eventually trial our gene technology on an international scale.