Funded Studies
Study Rationale:
Two main hallmarks of Parkinson’s disease (PD) are neurodegeneration and Lewy bodies, protein clumps comprising mainly of the protein alpha-synuclein. In diseases such as Parkinson’s, toxic alpha-synuclein clumps spread throughout the brain, damaging brain cells and causing neurodegeneration.
Hypothesis:
We hypothesize that stopping alpha-synuclein clumps from spreading or destroying them can prevent or stop neurodegeneration.
Study Design:
In this study, we will use a viral vector, a research tool that makes cells produce miniature proteins called antibodies. These proteins can stick to alpha-synuclein clumps and destroy them. Our viral vector will be injected into the spinal canal inside the spinal cord, where the mini-antibodies will begin their spread throughout the brain. We expect our approach to block toxic effects of alpha-synuclein and prevent neurodegeneration and resulting motor symptoms of PD.
Impact on Diagnosis/Treatment of Parkinson’s Disease:
This study will reveal the ability of our therapeutic approach to stop the progression of Parkinson’s in pre-clinical models.
Next Steps for Development:
If successful, our approach could be optimized and tested in clinical trials and potentially become a new treatment for Parkinson’s disease.