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Gene Therapy Approach to Reduce Alpha‐synuclein Aggregation in a Model of Parkinson's

Study Rationale:
A hallmark of Parkinson’s disease is the accumulation of an abnormal form of the protein alpha-synuclein inside brain cells. The team plans to use a gene therapy approach to insert a gene into a model of Parkinson's. This gene produces a factor that reduces the accumulation of abnormal alpha-synuclein in brain cells. Successful reduction of abnormal alpha-synuclein should lead to healthier brain cells that function normally.

This study will determine whether a viral vector gene therapy approach can be used to express a factor in brain cells that reduces abnormal alpha-synuclein accumulation and subsequently reduces or halts associated damage in those cells.

Study Design:
The investigators plan to use a model with accumulated abnormal alpha-synuclein inside brain cells. In order to stop the progressive accumulation of alpha-synuclein, they will inject a viral vector that inserts a gene into brain cells. This gene will express a factor that plays a role in reducing and clearing abnormal alpha-synuclein from brain cells. The team plans to use histological, molecular, protein and behavioral measures to examine how this treatment affects the accumulation of alpha-synuclein in the brain.

Impact on Diagnosis/Treatment of Parkinson’s Disease:
Gene therapy approaches can be used for long-term expression of therapeutic genes in cells. This study may identify a factor that is effective in reducing levels of abnormal alpha-synuclein and ultimately in reducing or halting damage and degeneration in brain cells affected by Parkinson's.

Next Steps for Development:
Positive results from this study could result in the development of clinical grade viral vectors for testing in clinical trials.


  • David Yurek, PhD

    Lexington, KY United States

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