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Evaluating a Gene Therapy that Restores Mitochondrial Function as a Treatment for Parkinson’s Disease

Study Rationale: Currently, all treatments for Parkinson’s disease (PD) aim to improve symptoms by restoring or maintaining the levels of dopamine in the brain. No treatment on the market slows or stops disease progression. Although the mechanism underlying neurodegeneration in PD remains elusive, growing evidence suggests that an accumulation of damaged mitochondria can play an important role. In this study, we will assess whether introducing a gene involved in mitochondrial quality control into cells and preclinical PD models will improve mitochondrial function and thereby act as a neuroprotective treatment.

Hypothesis: We hypothesize that Nix, a protein that restores mitochondrial function by facilitating mitochondrial quality control, will protect dopamine-producing neurons in PD.

Study Design: We will develop a new gene therapy to increase Nix levels in the brain cells of people with PD. We will evaluate this treatment in cells and in preclinical models of PD. This novel therapy works by improving the health of mitochondria, structures that supply cells with energy; increasing energy supply to vulnerable brain cells should boost their survival and enhance their ability to function.

Impact on Diagnosis/Treatment of Parkinson’s disease: There are currently no neuroprotective treatments for PD. This project will assess whether Nix gene therapy improves mitochondrial function, restores energy levels, and promotes survival in dopamine-producing neurons.

Next Steps for Development: If successful, the next step towards clinical application would be to undertake larger, preclinical studies prior to conducting a clinical trial in people with PD.


  • Carolyn M. Sue, MBBS, PhD, FRACP

    Sydney NSW Australia

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