Study Rationale: Gene mutations are one of several cause of Parkinson’s disease (PD). Developing an efficient method for introducing functional versions of these mutated genes into cells that are affected by PD could provide an effective way to treat, or even cure, the disease. Our study is designed to determine a more efficient way to introduce genes into the parts of the brain affected in PD.
Hypothesis: We hypothesize that our technology for brain gene therapy will be a safer and more effective way to introduce curative genes for PD than other technologies.
Study Design: Our technology introduces entire genes into cells using RNA, which makes the process much safer than other methods. Our study is designed to optimize all of the components of the process to make each step much more efficient and to show that the method can be applied first to cells in a laboratory and then to the brains of animals. By doing so, we are paving the way to introduce a gene therapy that may be useful for treating or curing PD.
Impact on Diagnosis/Treatment of Parkinson’s disease: This project has the potential to fundamentally alter the treatment of PD. A single injection of a new gene into the brain of people with PD could prevent worsening of symptoms or potentially restore previously lost function and thus cure the disease.
Next Steps for Development: If our study is successful, the next step would be to start preclinical studies that would allow us to bring the therapy to people with PD in the context of a clinical trial.