Centre for Rare Diseases & Gene Therapy
Approximately 8% of Australians live with a rare disease, that is around 2 million people, globally there are around 350 million people. Rare diseases are varied and life-threatening, yet most still remain undiagnosed and have no effective treatments.
About the Centre
Rare diseases are notoriously difficult to diagnose, and it can take years for people to discover the cause of their symptoms. Our focus is to understand the basic mechanisms of gene regulations to inform therapies for rare diseases. We aim to improve the lives of people with rare cardiovascular, blood and cancer disease through timely diagnosis and drug discovery and repurposing to improve the personalised treatments.
Centre Head
Laboratories
The laboratories operating within this centre are:
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Bailey
Gene regulation is frequently dysregulated in cancer. During cancer initiation, transcription factors can exhibit somatic mutations including point mutations in regulatory domains or undergo copy number variations due to genetic deletion. -
Francois
The David Richmond Laboratory for Cardiovascular Development: Gene Regulation and Editing is focused on identifying new and innovative therapeutic approaches targeting vascular disease. -
Rasko
Our Gene and Stem Cell Therapy program focuses it’s research on cancer and genetic diseases.
Get in touch
To get in touch and for all general enquiries relating to our work, please contact Adjunct Professor Mathias Francois.
Student opportunities
Postdoctoral and postgraduate students can work alongside world-leading medical researchers, with state-of-the-art facilities, in direct collaboration with the University of Sydney, UTS and Royal Prince Alfred Hospital.