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Gene & Stem Cell Therapy

Our Gene and Stem Cell Therapy program is focused on better understanding regenerative medicines to develop effective treatments for cancer, heart disease and genetic diseases. Regenerative medicine is the process of replacing or regenerating human cells, tissues or organs to restore or establish normal function.

Understanding Disease

We are focused on understanding how cancer cells work. Cancer is caused by the accumulation of mutations (errors) in our DNA. Cancer causing mutations activate oncogenes or inactivate tumour suppressor genes. Multiple DNA mutations lead to the development of cancer.

One tumour suppressor gene called CTCF is a DNA binding protein that is important for normal organisation of the chromatin, found in our chromosomes. Mutations and deletions of the CTCF gene occur in many cancer types including blood cancer. We are working to understand how CTCF functions in normal cells, and how changes in the CTCF gene lead to cancer development.

Finding a Cure

In the laboratory, we are focused on identifying the triggers that switch
genes on and off in cancer cells with the long-term goal of developing new cancer therapies. By integrating Centenary’s bioinformatics expertise into all of our research areas, we have significantly increased the outcomes of our research in the lab.

Our research has discovered new ways to target blood cancer. It has also identified key nutrient pumps, which are vital to the growth of prostrate cancer cells. Using these discoveries and our knowledge of how cancer cells work, we are striving towards better therapeutics for the treatment of cancer.

Track Record

In 2014 The Gene & Stem Cell Therapy Program published two seminal papers in the stem cell field arising from a global collaboration spanning five years’ of research (Nature and Nature Communications). The work provided the most detailed study to date of how specialised body cells can revert to an ‘undifferentiated’ state – akin to the cells in the early embryo which can give rise to all cells in the adult body. These discoveries accelerate our understanding of how such cells could be used as therapeutics to target many different diseases and regenerating tissues.

One day patients’ tissues and organs might be repaired using transplantation of ‘spare parts’ grown in the laboratory from a small sample of their own cells. The discovery that body cells can in principle be coaxed to become induced pluripotent stem cells (known as iPS cells) was awarded a Nobel Prize in 2012 to Drs Yamanaka and Gurdon.

With tongue-in-cheek humour the ambitious international consortium was known as “Project Grandiose”, so-called by Andras Nagy who co-ordinated the effort. The results provide a detailed map of molecular processes of iPS cell generation with the Gene & Stem Cell Therapy Program contributing data and analysis of diverse RNA molecules. The work was especially important as it extended and confirmed results on splicing of RNA molecules we had published in 2013 in Cell. The vast datasets are now publicly available online for all scientists working on reprogramming stem cells,

Professor John Rasko AO, Head of Program

Phone: +61 2 9565 6156
Email: j.rasko@centenary.org.au

Head, Gene and Stem Cell Therapy Program

In addition to leading the Gene and Stem Cell Therapy Program at The Centenary Institute, Professor John Rasko AO is a clinical hematologist, pathologist and scientist with a productive track record in gene and stem cell therapy, experimental haematology and molecular biology. In over 150 publications he has contributed to the understanding of stem cells and haemopoiesis, gene transfer technologies, oncogenesis, human aminoacidurias and non-coding RNAs.

He serves on Hospital, state and national bodies including Chair of GTTAC, Office of the Gene Technology Regulator – responsible for regulating all genetically-modified organisms in Australia – and Chair of the Advisory Committee on Biologicals, Therapeutic Goods Administration. Contributions to scientific organisations include co-founding (2000) and past-President (2003-5) of the Australasian Gene Therapy Society; Vice President, International Society for Cellular Therapy (2008-12) and founder (2009) ISCT-Australia; Scientific Advisory Committees and Board member for philanthropic foundations; and several Ethics Committees. He is the recipient of national (RCPA, RACP, ASBMB) and international awards in recognition of his commitment to excellence in medical research, including appointment as an Officer of the Order of Australia.