This is the beginning of a new frontier in the treatment of complex and rare diseases that affect millions of Australians. By donating today, you are supporting this world-first genomics research, where Garvan scientists are combining key technologies.
Gene editing
Garvan is now one of the leading hubs for genomics research, where our scientists are combining key technologies to treat complex and rare diseases. Associate Professor Rob Weatheritt is using a gene editing tool called CRISPR, which acts like ‘genetic scissors’. He explains:
“CRISPR enables us to go in with forensic specificity. We can give it a set of instructions and the CRISPR machinery will go to a particular part of the genome and edit it with high precision.”
Cellular genomics
We have trillions of cells in our body, but just one single cell can lead to disease. Co-lead Professor Joseph Powell, Pillar Director of the Garvan laboratory that developed this breakthrough program, is using his expertise in cellular genomics – also known as single cell sequencing – to unlock how our cells work individually, and how they function together.
Population genetics
A pioneering Garvan study called OneK1K – which analysed genetic samples from 1,000 people – demonstrated how our genetics contribute to the risk of disease at a cellular level.
Professor Joseph Powell and his team are planning to build on the success of this study by expanding the cohort to several thousand more donors – including a significant number of people living with Parkinson’s Disease – to give us unprecedented insights into disease and immune function.