Research Project

The Cure4CF Foundation aims to find a Cure for Cystic Fibrosis by suppoting promising areas of scientific research. To date, this has seen the Foundation support the research being undertaken by the Adelaide CF Gene Therapy Research Group, which is focussed on a gene therapy approach to Cystic Fibrosis (CF) Airway Disease. If the techniques and strategies being developed by the team are successful, this research will improve and save the lives of the 3,000 people living with CF in Australia and more than 70,000 people worldwide.

The group employs a world-leading gene transfer approach. They use an especially modified virus to take a correcting gene into the airway, supplementing the defective gene that causes Cystic Fibrosis Airway Disease. The cell then takes over the job of producing the normal airway physiology.

The most exciting aspect of the work is that although this approach should produce long term gene therapy treatments, it is also a potential cure for the airway disease in Cystic Fibrosis. It is designed to correct airway stem cells, meaning that as new airway cells are produced by the body, they will have the corrected gene operating within them. Success in this research project means that people living with Cystic Fibrosis Airway Disease will, for the first time, be able to be free of this disease.

Research undertaken by the team has shown that their gene transfer approach is successful in several animal models. The next challenge is to continue to develop their work to the point where they can commence trials of the gene transfer approach in Cystic Fibrosis patients.

Recent Research Highlights

During 2010 the research team has made a number of significant advances, particularly around the use of the actual gene transfer approach, bringing them closer to starting trials with Cystic Fibrosis patients.

Of particular importance was their work that showed the ability of the research team to produce a properly functioning expression of the gene that corrects the Cystic Fibrosis defect for more than 12 months. No other research group worldwide had been able to attain such extended gene correction.

Select Research Publications

Through the publication of their research the team shares their techniques and developments with the scientific community. Below is a selection of the research publications produced by the team, many of which have been published by highly regarded academic journals.

2010

Liu, C, Wong, E, Miller, D, Smith, G, Anson, D & Parsons, D 2010, 'Lentiviral airway gene transfer in lungs of mice and sheep: successes and challenges', The Journal of Gene Medicine, vol. 12, no. 8, pp. 647-658.
Summary available at: http://onlinelibrary.wiley.com/doi/10.1002/jgm.1481/abstract.

Donnelley, M, Siu, KK, Morgan, KS, Skinner, W, Suzuki, Y, Takeuchi, A, Uesugi, K, Yagi, N & Parsons, DW 2010, 'A new technique to examine individual pollutant particle and fibre deposition and transit behaviour in live mouse trachea', Journal of Synchrotron Radiation, vol. 17, no. 6, Nov, pp. 719-729.
Full article freely available at: http://scripts.iucr.org/cgi-bin/paper?S0909049510028451.

Donnelley, M, Parsons, D, Morgan, K & Siu, K 2010, 'Animals In Synchrotrons: Overcoming Challenges For High-Resolution, Live, Small-Animal Imaging', AIP Conference Proceedings, vol. 1266, no. 1, pp. 30-34.
Summary available at: http://link.aip.org/link/?APCPCS/1266/30/1
 
Cmielewski, P, Anson, DS & Parsons, DW 2010, 'Lysophosphatidylcholine as an adjuvant for lentiviral vector mediated gene transfer to airway epithelium: effect of acyl chain length', Respiratory Research, vol. 11, p. 84.
Full article freely available at: http://respiratory-research.com/content/11/1/84.