Cystic Fibrosis Gene Therapy

Research Leaders: Dr David Parsons

The goal of the Cystic Fibrosis Gene Therapy group is to produce a safe and effective treatment for cystic fibrosis airway disease; ultimately our hope is to produce a cure. To reach this goal the group uses a modified virus to transport the correcting gene directly into the affected airway surfaces, and our unique method is designed to target the adult stem cells that are normally present in the airway cell surface.

When effective, those corrected airway stem cells can automatically generate corrected daughter cells that populate the airway surface. In parallel, the group is also developing new methods using the special X-rays produced by a synchrotron - at facilities in Japan and Melbourne, to improve early detection of the effectiveness of our gene therapy. The group's studies include the use of laboratory mice, and special CF mice, to develop and test these methods to help us translate our findings into realistic therapies for testing in future clinical trials.

Research Priorities:

• Safe and effective airway gene transfer
• Early and non-invasive detection of success or failure of gene transfer in living airways