Study Rationale: Parkinson's disease is a serious brain condition that causes problems with movement and other body functions. Scientists know that a protein called alpha-synuclein builds up in the brains of people with Parkinson's and harms important brain cells. To better understand this process, we are creating two different pre-clinical models where we add extra alpha-synuclein to their brains using a safe virus. This will help us study the early changes in the brain that happen in Parkinson's disease and develop better ways to treat it.
Hypothesis: We believe that putting extra alpha-synuclein into each model brains will cause movement problems and changes in brain chemicals, similar to what happens in people with Parkinson's disease. We will also assess virus batch variability and compare effects of delivering normal versus mutated human alpha-synuclein.
Study Design: We will inject a safe virus carrying the human alpha-synuclein gene into a specific part of each model brains called the substantia nigra. We will test the ability of each model to move before the injection and again several weeks after. After six weeks, we will study their brains carefully to look for chemical changes and signs of damage. This will help us understand how the protein affects brain health and movement.
Impact on Diagnosis/Treatment of Parkinson’s disease:This project could help scientists find early warning signs of Parkinson's disease and create new treatments that stop or slow the disease before major brain damage occurs.
Next Steps for Development: If our study works as we hope, we will use these two different pre-clinical models to test promising new medicines. These studies could lead to treatments that help people with Parkinson's live better and longer lives.