Professor Carolyn Sue AM
Kinghorn Chair, Neurodegeneration
Our group aims to translate research discoveries into clinical practice. Our primary focus is to understand how mitochondrial function, necessary for neuronal survival, becomes impaired in patients with Parkinson’s. This allows us to develop strategies to restore mitochondrial function and prevent neuronal degeneration and cell death thus slowing or stopping disease progression.
Our Parkinson’s Disease Centre actively recruits to clinical trials and offers our patients the latest opportunities to participate in research to improve their health outcomes.
By studying the genetic basis of disease in Parkinson’s patients we have identified new ways to improve mitochondrial quality and function by enhancing mitochondrial clearance (mitophagy). Damaged or senescent mitochondria are unable to provide the cell with adequate amounts of vital energy and can lead to cell degeneration. When mitophagic proteins are experimentally enhanced in cultured patient-derived cells mitochondrial recycling and function is restored.
Our research is now focused on developing ways to enhance mitophagy, including gene therapy approaches, in preclinical studies with patient-derived neuronal cells and animal models, that will pave the way to the treatment of patients.
Our preclinical studies will demonstrate the most effective gene therapy vectors in enhancing Nix expression in dopamine-producing cells for testing in a clinical trial. When Nix over-expression is developed into a robust in-human treatment, it has the potential to revolutionise the field by providing the first treatment to stop the progression of Parkinson’s.
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