The Cystic Fibrosis Airway Research Group
Our goal is to develop an effective genetic therapy for prevention or treatment of Cystic Fibrosis airway disease.
The group’s research themes are currently focussed on several complementary areas; achieving effective lentiviral CFTR vector gene delivery, lentiviral vector development, upscaling vector production, transducing airway stem cells in situ to enable extended gene expression, developing new delivery methods, and developing rapid and accurate outcome measures for assessment of airway disease using X-ray imaging.
The lentiviral gene therapy we are developing and testing has three clear advantages:
- The only rational way to prevent or halt CF airway disease is by overcoming the fundamental problem – the defective CF gene – by adding a correct copy of the CF gene to airway cells.
- Successful correction will be effective for all CF mutations (of which there are more than 2,000), in contrast to new and expensive drugs that depend on specific CF mutation type, this cure would be suitable for all CF patients.
- Effective gene treatment of airway stem cells – the cells naturally present in the airway that are responsible for repair and renewal of airway tissue – will provide very long-lived correction after the initial dosing is completed, with a goal of seeking lifelong correction for the patient.
The need for fast, reliable, and non-invasive methods to pre-clinically test for correction of CF airway physiological function has led to rapid progress using novel synchrotron X-ray imaging approaches in live mice in collaboration with physicists from Monash University and the Australian Synchrotron. These techniques quantify airflow in the lungs during breathing, as well as the effects of treatments on airway surface function.
Research into translation of methods for potential human application in a non-synchrotron diagnostic setting has recently begun and has been assisted by the 2013 opening of the Imaging and Medical Beamline at the Australian Synchrotron and the Monash Dynamic X-ray Imaging Facility in the Laboratory for Dynamic Imaging.