Our lab is interested in understanding the cellular and molecular processes involved in the regulation of epithelial cell biology and how the breakdown of this regulation leads to pathogenesis of disease such as Cystic Fibrosis.
We have developed an Australian national biobank of stem-cell-derived airway and gut organoids, and have built a platform for high-throughput therapy-testing on patients' organoids. We combine our strengths in organoid disease modelling, multi-omic molecular profiling and computational research with clinical data to improve individualised outcomes for patients with CF.
Cystic Fibrosis (CF) is the most common inherited disease in Australia. Patients endure a life of debilitating symptoms resulting from pathology which begins prior to birth. This is despite advances in therapies which have improved symptom relief by targeting the underlying molecular defect – the CF Transmembrane Regulator (CFTR) protein. We have implemented a state-of-the-art organoid-based functional precision drug screening platform and established CF airway and gut organoid biobank resource (from >200 CF patients). This ground-breaking platform has allowed the full characterisation patients with common CFTR mutations and those with ultra-rare CFTR variants, acting as a personalised care model and identifying those that would otherwise not have access to life-saving targeted drugs.