Nix Gene Therapy as a Treatment for Parkinson’s Disease

Professor Sue and her team have discovered that the Nix protein restores mitophagy and mitochondrial function in people with the PINK1/PARKIN gene mutation and the team are now working on the theory that overexpressing the Nix protein using gene therapy will stop the progression of Parkinson’s.

Study Rationale:

Currently, all Parkinson’s disease treatments aim to improve symptoms by restoring or maintaining levels of dopamine in dopaminergic neurons. No treatment on the market slows or stops the progression of disease. This research addresses this gap by showing that Nix gene therapy is effective in improving mitochondrial function and can be used as a neuroprotective treatment in Parkinson’s disease.


We hypothesise that Nix, a mitophagic protein can restore mitochondrial function by mediating an alternative method of mitophagy to maintain mitochondrial quality and function in Parkinson’s disease.

Study Design:

We will develop a new gene therapy to increase Nix expression in the brain cells of patients with Parkinson’s disease. This treatment will be tested in cell and animal- based models of Parkinson’s disease This novel treatment works by improving the energy supply to the brains cells so that they can function for longer.

Impact on Diagnosis/Treatment of Parkinson’s disease:

There are currently no neuroprotective treatments for Parkinson’s disease. This project will test the treatment, a form of gene therapy, to see if it is effective in restoring neuronal energy levels.

Next Steps for Development:

If we successfully show that Nix gene therapy can improve mitochondrial function and restore energy levels in neurons, then the next step towards clinical application is to undertake large animal studies prior to developing an in human clinical trial


About Professor Carolyn Sue AM

Professor Carolyn Sue AM is the inaugural Kinghorn Chair, Neurodegeneration.

As an international expert in mitochondrial disease and movement disorders. Professor Sue has been running Australia’s largest specialised clinic dedicated to the diagnosis, assessment and treatment of patients with mitochondrial disease since 1994.

She is a medicine alum of UNSW who has trained at Prince of Wales Hospital and at Columbia University in the United States.

Prof Sue is a Fellow of the Australian Academy of Health and Medical Sciences and serves on the Council of the National Health and Medical Research Council. She also holds leadership roles at Movement Disorder Society of Australia and New Zealand, International Parkinson’s disease and Movement Disorder Society and Australian Mitochondrial Disease Medical Network.

In her new position as the inaugural Kinghorn Chair, Neurodegeneration at NeuRA, Prof Sue also seeks to establish clinical trials centres for both Parkinson’s disease and mitochondrial disease patients and create a training centre for clinicians and scientists in the neurosciences who will become future leaders in neuroscience research, expanding on the collective achievements of NeuRA.