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.
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.
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