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16 Headlines Alzheimer’s the target for virus ‘vector’ Dr Bruce Mockett from the University of Otago has just embarked on an exciting three-year project investigating the use of viruses to treat Alzheimer’s disease. He has selected a specific virus that he will use as a vector, or transport mechanism, to deliver gene therapy across the blood-brain barrier. The virus is harmless and will not cause disease. “Diseases of the brain are immensely difficult to treat because of a protective mechanism known as the blood-brain barrier,” Dr Mockett explains. “This virus is special for two reasons. First, unlike many similar viruses, it has been shown to cross the blood- brain barrier of marmosets, a non- human primate, in overseas research. This is particularly important for this work if we are to go on to perform clinical trials in human subjects. Second, it has a very low risk of inducing cancer formation in liver cells. This is important as it is a risk of all virus treatments.” The virus will be modified to carry a protein known to restore neurological damage (sAPPa). Viruses are highly evolved and many, like this one, are able to ‘trick’ the brain into letting it through, where it will deliver sAPPa to specific cells. “Increasingly, we have the means to treat neurological diseases, but need to develop delivery mechanisms such as the one proposed in this grant.” Many clinical trials are in progress that use viruses as a delivery mechanism for gene therapy. If successful, the method could be used to treat other dementias or to boost brain repair after traumatic injuries and stroke. “We still don’t fully understand what initiates Alzheimer’s disease. However, our understanding of the molecular mechanisms underlying the disease has advanced enormously over the last 30 years. I think we are getting much closer to being able to stop its progression and possibly reverse some of the cognitive loss.” GOING VIRAL The researchers using common viruses to fight brain disease Viruses have wreaked havoc on humans since the dawn of our species. The COVID-19 pandemic reminded the world of the devastation a virus can cause. I n medicine, viruses can also be used for good. In neuroscience specifically they can be a valuable tool to develop treatments for disease. We talk to two neuroscientists who are using viruses in their research. Dr Bruce Mockett is a Senior Research Fellow in the Neural Mechanisms of Memory Lab at the University of Otago. He has nearly 30 years' experience as a neuroscience researcher. His project, titled A new peripheral gene therapy approach for treating Alzheimer’s disease , has received a grant from the Neurological Foundation totalling $247,000. Dr Mockett will work with Associate Professor Stephanie Hughes and Professor Cliff Abraham on the project, who are both highly respected brain health researchers at the University of Otago. “This approach is certainly cutting edge and holds great potential for the treatment of diseases where geneticmutations, and hence dysfunctional proteins, are an underlying cause.”
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