Research from the CBR has made the international news with advances in genetic therapies.
The transgenic Huntington’s sheep developed by Professors Richard Faull and Russell Snell has been featured in the prestigious magazine Scientific American. The article highlights how the sheep model was developed over many years of painstaking research, supported by the Freemasons of New Zealand.
Now 5 years on, the sheep is being used to study Huntington’s disease, which causes devastating movement and psychological symptoms which get worse as patients age. The sheep will hopefully be used to develop new drugs for this debilitating disease. The article can be viewed here.
Research into another genetic disease, Canavan disease, has also been published in the top-class journal Science Translational Medicine. The gene therapy breakthrough was developed by Associate Professor Debbie Young and Professor Matt During’s team, who research viral vectors as a way to alter disease progression in humans.
Canavan disease is a rare hereditary disorder which affects children, causing their entire nervous system to stop working. Long-term follow-up after the gene therapy has shown that patients had less seizures and slower brain shrinkage. The exciting research may mean that the children have better health as they grow up.
These important clinical developments both came about through painstaking preclinical research, and highlight just how important support for basic research is. You can read more about the need for basic research here.