Does the presence of lung infection impact the efficacy of cystic fibrosis airway gene therapy?
Chief Investigator: Dr Alexandra McCarron (Early Career Researcher)
Funding Amount : $40,060
Recipient: The University of Adelaide
Overview: This project will evaluate a novel treatment for cystic fibrosis (CF), a chronic, progressive, and debilitating genetic illness. Unrelenting CF lung disease begins early in infancy, steadily deteriorating patient quality of life and ultimately leading to premature death. The development of effective therapies for lung disease will undoubtedly provide significant benefit. Airway gene therapy is one strategy that could cure CF lung disease in all children and works by correcting the fundamental genetic defect. Provided early in life, gene therapy will enable children to grow into adulthood without the burden of lung disease, dramatically improving longevity and quality of life.