Approximately half of those who suffer from cystic fibrosis in Australia will die from lung disease by their late thirties. This project aims to test the effectiveness of airway gene therapy as a treatment for lung disease caused by cystic fibrosis, and hopefully provide an effective and safe cure for this devastating illness.
Gene therapy involves inserting healthy and correctly functioning cystic fibrosis genes into cells that line the airways. Once established, it is hoped this new genetic information will remedy the basic cellular defect that causes cystic fibrosis and lung disease.
Our group has already demonstrated successful gene transfer therapy in mice using a modified virus that carries the healthy genes to their destination. We have found that this type of gene transfer has corrected the defective cystic fibrosis genetic information in the airways of our mice models for more than a year. This discovery has the potential to transform the lives of children with cystic fibrosis and other respiratory diseases.
Started in 2010, this research project is being carried out in the Department of Respiratory and Sleep Medicine at the Women's and Children's Hospital, in collaboration with researchers from the Centre for Stem Cell Research at theRobinson Institute, Monash University, and the SPring-8 Synchrotron in Japan. It is funded by a $892,000 project grant awarded from the National Health and Medical Research Council.
This research projects aims to monitor the surface behaviour of inhaled particles and pollutants such as dust, lead, asbestos and fibreglass on live air passages using synchrotron x-rays. Synchrotron machines are the latest in radiation technology, able to produce extremely intense x-rays of particles that were previously considered too small to view. They represent a landmark first in being able to observe particle behaviour without the need for surgery or biopsy.
Since airborne pollutants have the potential to affect lung health if they are not cleared by our natural airway defences, this project will teach us more about the behaviour of ingested pollutants at their first point of contact with the airways, and the potential of synchrotron technology to measure the success of treatment for respiratory diseases such as cystic fibrosis.
Begun in 2007, this research project has involved up to six researchers led by Dr David Parsons, leader of the Cystic Fibrosis Stream at the Children's Research Centre and Chief Medical Scientist in the Department of Respiratory and Sleep Medicine at the Women's and Children's Hospital. Dr Parsons works in partnership with experts in the use of synchrotron x-rays at Monash University and in Japan. The project is funded by a $55,000 grant from the Women's and Children's Hospital Foundation and a grant of $94,000 from the USA Cystic Fibrosis Foundation.
Our cystic fibrosis researchers are using synchrotron x-rays to examine the surface behaviour of inhaled pollutants on live air passages, in order to learn more about the movement of ingested particles and measure the success of current cystic fibrosis treatments.