Gene Therapy sounds simple enough.
You give someone a healthy gene, to replace a faulty one.
But it's not such a simple process. It involves the use of new technology, a combination of gene therapy and cell research, specifically targeting the root genetic cause of inherited diseases.
Cell and gene therapy involves delivering a healthy gene into the patient's target cells via a harmless virus vector. Once the virus binds to the cell it then injects the DNA that it contains into the cell and that cell then takes up that DNA, makes it its own and then produces whatever the cell is told to make, thereby eliminating the faulty 'disease-causing' gene.
What's so amazing about cell and gene therapy is that it has the almost incomprehensible potential of finding cures for thousands of genetically inherited diseases that affect us and our families everyday, like breast cancer, heart disease, diabetes, prostate cancer, haemophilia and leukaemia just to name a few.
At the forefront of this pioneering research is Professor John Rasko. He is a world-renowned gene therapy scientist and practising haematologist based at Royal Prince Alfred Hospital, chairman of the federal committee GTTAC (The Gene Technology Technical Advisory Committee) and amongst other things, he is a board director of Cure The Future - a foundation specifically set up to fund cell and gene therapy research in Australia.
Along with international collaborators, Professor Rasko was the first-in-the-world to run a clinical gene therapy trial with the aim of curing Haemophilia. As recently reported on the ABC Television program Catalyst, this trial involved a single injection of a modified, safe virus carrying the therapeutic haemophilia gene. This was delivered into the liver of the patient. In one of the two Australian patients we saw an increase in the previously-absent haemophilia factor was experienced.
While this success was only temporary, this trial offered proof to the principle that by treating the root genetic cause of a disease it can be cured.
Cure The Future is committed to funding research which might one day lead to finding the cures for thousands of genetically inherited diseases.
In decades to come, we will look back on this time as a pioneering era when our contributions to human health and well-being were rewarded by discoveries which led to the cure of many diseases.
who are Cure The Future?
Cure The Future comprises passionate individuals who believe that by funding research designed to facilitate these innovative cell and gene therapies, we can cure genetic diseases and, in so doing, bring an end to the future suffering of those struck by illness.