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Australia; International; NSW
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Expert Reaction
These comments have been collated by the Science Media Centre to provide a variety of expert perspectives on this issue. Feel free to use these quotes in your stories. Views expressed are the personal opinions of the experts named. They do not represent the views of the SMC or any other organisation unless specifically stated.
Dr Bryce Vissel is a Professor in the School of Clinical Medicine at UNSW and Director of the Centre for Neuroscience and Regenerative Medicine at St Vincent's Hospital Sydney
This study potentially heralds the start of a new era of treatments for diseases of the brain and spinal cord where a specific underlying genetic cause is known.
In humans the gene mutation that causes Huntington’s disease is well known. The mutation has devastating impact following disease onset. In this study the mutant gene was effectively knocked down which has potential to stop or even maybe reverse the devastating disease.
Much more work is needed, but the indications from this study are that we are well into the journey now of developing treatments for devastating genetic disorders that affect the brain and spinal cord.
