Gene therapy is a new way of doing medicine that centres on using genes to treat or prevent disease. Genes are at the heart of how our bodies develop and function, and genes that don’t work properly are the cause of, or contribute to the development of, many diseases. In the case of Cystic Fibrosis, a malfunctioning gene called CFTR results in the disease.

The principle behind gene therapy is that diseases like Cystic Fibrosis can be stopped, or able to be better combated by the body, through the replacement of the faulty gene or the adding-in of a properly functioning version of the gene.

There are different methods that scientists and medical researchers are developing to bring the corrected or properly functioning version of a gene into the body. The gene therapy research team in Adelaide is exploring and refining the use of an especially modified virus to transport the corrective gene into the body’s airway.

Gene therapy is not without risks, and as it is a new way of doing medicine it may take longer to assess the safety and the effectiveness of gene transfer treatments. However it is a fast evolving area of research, and one that holds great promise for those living with Cystic Fibrosis.

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“I want these kids to have the chance to live as long as I have”

– the late Allan Scott, AO OAM, Cure4CF Foundation Major Sponsor

“Gene therapy is a new way of doing medicine and we are making exciting progress in this innovative research aimed at correcting the basic defect which causes Cystic Fibrosis lung disease.

While we have much confidence it will be an effective long-lived treatment, the exciting hope is that it may become a cure for the lung disease in Cystic Fibrosis.”

– Dr. D. Parsons, Chief Medical Scientist, Respiratory Medicine, WCH