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Using Genomics to Save Lives
Genetics, the study of our genes, is making it possible to predict, diagnose, and treat diseases more precisely and personally than ever before. Scientists at Garvan are now harnessing this knowledge to research personalised treatments for some of the world’s most rare and complex diseases.
Drug development is notoriously difficult, with over 85% of drugs that enter clinical trials failing in phase one. Garvan hopes to fast-track this process with its drug-discovery program, which combines world-leading genetic technologies in a totally unique way.
Associate Professor Rob Weatheritt, an expert in RNA therapies, is co-leading this world-first genomics-led drug discovery program.
“Our goal is to develop new, personalised treatments by leveraging the genetic diversity between patients to identify the mechanisms that cause disease.”
– Associate Professor Rob Weatheritt, Co-Lead.
The life-changing power of genomics
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.
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.”
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.
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.
Treating complex diseases with Genomics is the future of personalised medicine.
Parkinson’s is a debilitating, progressive disorder of the brain that causes shaking and stiffness, as well as walking, balance and coordination problems. There are no current treatments for the causes of Parkinson’s, in part because there is a poor understanding of how and why the disease develops.
However, we do know that genetics play a key role in this disease. Associate Professor Weatheritt is co-leading a team who is using the latest in genetic technology to understand the genetic differences between people, and the underlying causes of complex diseases like Parkinson’s.
“We can go from looking at the genetic changes in a Parkinson’s patient to a targeted treatment for them.” – Associate Professor Rob Weatheritt.
Your support will not only accelerate our ability to treat complex and rare diseases, but make a lasting impact on the health and lives of future generations.